RTI Headlines

July 3, 2019 New publication from the Zamore lab. High-Throughput Analysis Reveals Rules for Target RNA Binding and Cleavage by AGO2.  Becker WR, Ober-Reynolds B, Jouravleva J, Jolly SM, Zamore PD, Greenleaf WJ. Mol Cell. 2019 Jul 3. pii: S1097-2765(19)30445-9. doi: 10.1016/j.molcel.2019.06.012. [Epub ahead of print]PMID: 31324449

June 21, 2019 New publication from the Sontheimer lab. X-Tracting a New CRISPR-Cas Genome-Editing Platform from Metagenomic Data Sets.  Sontheimer EJ.CRISPR J. 2019 Jun;2:148-150. doi: 10.1089/crispr.2019.29061.ejs. No abstract available. PMID:31225753 

September 11, 2018 New publication from the Grunwald lab. Fluorescence Polarization Control for On-Off Switching of Single Molecules at Cryogenic Temperatures. Hulleman CN, Huisman M, Moerland RJ, Grünwald D, Stallinga S, Rieger B.Small Methods. 2018 Sep 11;2(9). pii: 1700323. doi: 10.1002/smtd.201700323. Epub 2018 Apr 30. PMID:31240238

June 12, 2019 New publication from the Xue lab.  Depletion of TRRAP induces p53‐independent senescence in liver cancer by downregulating mitotic genes.  Suet‐Yan Kwan,  Ankur Sheel, Chun‐Qing Song,  Xiao‐Ou Zhang,  Tingting Jiang,  Hien Dang,  Yueying Cao, Deniz M. Ozata,  Haiwei Mou,  Hao Yin,  Zhiping Weng,  Xin Wei Wang,  Wen Xue.  AASLD 2019 June 12 doi: 10.1002/hep.30807 

May 11, 2019 New publication from the Zamore lab. The RNA-Binding ATPase, Armitage, Couples piRNA Amplification in Nuage to Phased piRNA Production on Mitochondria. Ge DT, Wang W, Tipping C, Gainetdinov I, Weng Z, Zamore PD. Mol Cell. 2019 Apr 26. pii: S1097-2765(19)30276-X. doi: 10.1016/j.molcel.2019.04.006. [Epub ahead of print] PMID: 31076285 

May 8, 2019 New pre-print publication from the Sontheimer and Xue labs. 
Tissue-specific Genome Editing in vivo by MicroRNA-repressible Anti-CRISPR Proteins. Lee J,Mou H, Ibraheim R, Liang SQ, Xue W, Sontheimer EJ. BioRxiv. May 8, 2019. doi: https://doi.org/10.1101/631689 

April 29, 2019 New publication from Khvorova, Watts & Brown labs. Nucleic Acid Therapeutics for Neurological Diseases. Watts JK, Brown RH, Khvorova A. Neurotherapeutics. 2019 Apr 29. doi: 10.1007/s13311-019-00736-1.

April 16, 2019 New publication from the Xue lab. 
CRISPR-SONIC: targeted somatic oncogene knock-in enables rapid in vivo cancer modeling. Mou† H, Ozata† DM, Smith† JL, Sheel A, Kwan SY, Hough S, Kucukural A, Zachary Kennedy Z, Cao Y and Xue W. Genome Medicine 2019 11:21

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March 7, 2019 Congratulations, Alireza Edraki (Sontheimer lab) for receiving an F31 Fellowship!!

March 1, 2019 Congratulations, Jordan Smith (Xue lab) for receiving a NCI F30 fellowship!!

February 25, 2019 New publication from the Xue labAdenine base editing in an adult mouse model of tyrosinaemia. Chun-Qing Song, Tingting Jiang, Michelle Richter, Luke H. Rhym, Luke W. Koblan, Maria Paz Zafra, Emma M. Schatoff, Jordan L. Doman, Yueying Cao, Lukas E. Dow, Lihua Julie Zhu, Daniel G Anderson, David R. Liu, Hao Yin & Wen Xue. Nature BME. 25 February 2019

February 12, 2019 New publication from the Watts lab. Next-Generation Peptide Nucleic Acid Chimeras Exhibit High Affinity and Potent Gene Silencing. Debacker AJ, Sharma VK, Meda Krishnamurthy P, O'Reilly D, Greenhill R, Watts JK. Biochemistry. 2019 Feb 12;58(6):582-589. doi: 10.1021/acs.biochem.8b00827. Epub 2018 Dec 18. PMID: 30520300

February 12, 2019 New book chapter from the Khvorova lab. Conjugate-mediated Delivery of RNAi-based Therapeutics: Enhancing Pharmacokinetics–Pharmacodynamics Relationships of Medicinal Oligonucleotides. Godinho BMDC, Coles AH, and Khvorova K. Chapter 10. Advances in Nucleic Acid Therapeutics. 2019 Feb 11. http://dx.doi.org/10.1039/9781788015714

Jon Watts

February 6, 2019 Rett Syndrome Research Trust provides $2.7 million in new funding for research to Jonathan Watts and colleagues working to develop therapy to fix faulty gene in Rett syndrome

CRISPRJanuary 21, 2019 New publication from the Xue labCRISPR–Cas: a tool for cancer research and therapeutics. Yin H, Xue W, Anderson DG. Nat Rev Clin Oncol. 2019 Jan 21. doi: 10.1038/s41571-019-0166-8. [Epub ahead of print] Review. PMID: 30664678

January 17, 2019 New publication from the Zamore and Grunwald labs. An automated Bayesian pipeline for rapid analysis of single-molecule binding data. Smith CS, Jouravleva K, Huisman M, Jolly SM, Zamore PD, Grunwald D. Nat Commun. 2019 Jan 17;10(1):272. doi: 10.1038/s41467-018-08045-5. PMID: 30655518

December 19, 2018 New publication from the Aronin and Khvorova labs. 
Rac1 Activity Is Modulated by Huntingtin and Dysregulated in Models of Huntington's Disease. Tousley A, Iuliano M, Weisman E, Sapp E, Zhang N, Vodicka P, Alexander J, Aviolat H, Gatune L, Reeves P, Li X, Khvorova A, Ellerby ML, Aronin N, DiFiglia M, Kegel-Gleason BK. J Huntingtons Dis. 2018 Dec 28. doi: 10.3233/JHD-180311. [Epub ahead of print] PMID: 30594931

December 18, 2018. New publication from the Sontheimer lab. A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome Editing. Edraki A, Mir A, Ibraheim R, Gainetdinov I, Yoon Y, Song CQ, Cao Y, Gallant J, Xue W, Rivera-Pérez JA, Sontheimer EJ. Mol Cell. 2018 Dec 18. pii: S1097-2765(18)31033-5. doi: 10.1016/j.molcel.2018.12.003. [Epub ahead of print]
PMID: 30581144

Khvorova Liver TransplantationDecember 18, 2018 New publication from the Khvorova lab. Gene Silencing with siRNA (RNA Interference): A New Therapeutic Option during Ex-vivo Machine Liver Perfusion Preservation.  Thijssen MF, Brüggenwirth IMA, Gillooly A, Khvorova A, Kowalik TF, Martins PN.Liver Transpl. 2018 Dec 18. doi: 10.1002/lt.25383. [Epub ahead of print]PMID: 30561891

December 15, 2018. New publication from the Grunwald lab. Impact of optical aberrations on axial position determination by photometry. Thorsen RØ, Hulleman CN, Hammer M, Grünwald D, Stallinga S, Rieger B. Nat Methods. 2018 Dec;15(12):989-990. doi: 10.1038/s41592-018-0227-4. PMID: 30504865 

December 14, 2018 New publication from the Khvorova lab.  Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo. Biscans A, Coles A, Haraszti R, Echeverria D, Hassler M, Osborn M, Khvorova A. Nucleic Acids Res. 2018 Dec 14. doi: 10.1093/nar/gky1239. [Epub ahead of print] PMID: 30544191

December 8, 2018 New publication from the Khvorova lab. Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways. Osborn MF, Coles AH, Biscans A, Haraszti RA, Roux L, Davis S, Ly S, Echeverria D, Hassler MR, Godinho BMDC, Nikan M, Khvorova A.Nucleic Acids Res. 2018 Dec 8. doi: 10.1093/nar/gky1232. [Epub ahead of print] PMID: 30535404

reconstructionDecember 5, 2018. New publication from the Grunwald lab. Deconstructing transport-distribution reconstruction in the nuclear-pore complex. Tu LC, Huisman M, Chung YC, Smith CS, Grunwald D. Nat Struct Mol Biol. 2018 Dec 5. doi: 10.1038/s41594-018-0161-2. [Epub ahead of print] PMID: 30518848

Cas9December 5, 2018. New publication from the Sontheimer lab. NmeCas9 is an intrinsically high-fidelity genome-editing platform. Amrani N, Gao XD, Liu P, Edraki A, Mir A, Ibraheim R, Gupta A, Sasaki KE, Wu T, Donohoue PD, Settle AH, Lied AM, McGovern K, Fuller CK, Cameron P, Fazzio TG, Zhu LJ, Wolfe SA, Sontheimer EJ. Genome Biol. 2018 Dec 5;19(1):214. doi: 10.1186/s13059-018-1591-1. PMID: 30518407

December 4, 2018. New publication from the Sontheimer lab. Potent Cas9 Inhibition in Bacterial and Human Cells by AcrIIC4 and AcrIIC5 Anti-CRISPR Proteins. Lee J, Mir A, Edraki A, Garcia B, Amrani N, Lou HE, Gainetdinov I, Pawluk A, Ibraheim R, Gao XD, Liu P, Davidson AR, Maxwell KL, Sontheimer EJ.MBio. 2018 Dec 4;9(6). pii: e02321-18. doi: 10.1128/mBio.02321-18. PMID: 30514786

December 4, 2018. New publication from the Sontheimer lab.
 Adapting dCas9-APEX2 for subnuclear proteomic profiling. Gao XD, Rodríguez TC, Sontheimer E2. Methods Enzymol. 2019;616:365-383. doi: 10.1016/bs.mie.2018.10.030. Epub 2018 Dec 10.

preeclampsiaNovember 19, 2018 New publication from the Khvorova & Moore labs. RNAi modulation of placental sFLT1 for the treatment of preeclampsia. Turanov AA, Lo A, Hassler MR, Makris A, Ashar-Patel A, Alterman JF, Coles AH, Haraszti RA, Roux L, Godinho BMDC, Echeverria D, Pears S, Iliopoulos J, Shanmugalingam R, Ogle R, Zsengeller ZK, Hennessy A, Karumanchi SA, Moore MJ, Khvorova A. Nat Biotechnol. 2018 Nov 19. doi: 10.1038/nbt.4297. [Epub ahead of print]. PMID: 30451990

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November 19, 2018 New publication from the Wolfe and Sontheimer labs. Orthogonal Cas9-Cas9 chimeras provide a versatile platform for genome editing. Bolukbasi MF, Liu P, Luk K, Kwok SF, Gupta A, Amrani N, Sontheimer EJ, Zhu LJ, Wolfe SA. Nat Commun. 2018 Nov 19;9(1):4856. doi: 10.1038/s41467-018-07310-x. PMID: 30451839

SCGENovember 18, 2018 The RTI's Erik Sontheimer, Anastasia Khvorova, Jon Watts along with Scot Wolfe and the RTI's Wen Xue along with Guanping Gao all join the National Institutes of Health’s Somatic Cell Genome Editing Consortium after each receiving UG3 awards, major collaborative awards. The NIH Common Fund’s Somatic Cell Genome Editing program aims to develop quality tools to perform effective and safe genome editing in human patients.
Press Coverage:

piRNAsNovember 16, 2018 New review from the Zamore Lab. PIWI-interacting RNAs: small RNAs with big functions. Ozata DM, Gainetdinov I, Zoch A, O'Carroll D, Zamore PD. Nat Rev Genet. 2018 Nov 16. doi: 10.1038/s41576-018-0073-3. [Epub ahead of print] Review. PMID: 30446728

Craig MelloNovember 10, 2018 The RTI's Nobel Laureate, Craig Mello, honored in Fall River, MA. Event put on by the Portugese American community at the Cove restaurant in Fall River. 

Press coverage:

November 12, 2018 New publication from the Zamore lab. Maelstrom Represses Canonical Polymerase II Transcription within Bi-directional piRNA Clusters in Drosophila melanogaster. Chang TH, Mattei E, Gainetdinov I, Colpan C, Weng Z, Zamore PD. Mol Cell. 2018 Nov 12. pii: S1097-2765(18)30932-8. doi: 10.1016/j.molcel.2018.10.038. [Epub ahead of print] PMID: 30527661

November 8, 2018 New publication from the Dekker & Moore labs. Higher-Order Organization Principles of Pre-translational mRNPs. Mihir Metkar M, Ozadam H, Lajoie BR, Imakaev M, Mirny LA, Dekker J, Moore M. Mol Cell Nov 8, 2018 DOI:https://doi.org/10.1016/j.molcel.2018.09.012

November 7, 2018 New publication from Sena-Esteves, Flotte, Aronin & Gao labs. Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer. Wang D, Zhong L, Li M, Li J, Tran K, Ren L, He R, Xie J, Moser RP, Fraser C, Kuchel T, Sena-Esteves M, Flotte TR, Aronin N, Gao G. Mol Ther Methods Clin Dev. 2018 Oct 4;11:65-72. doi: 10.1016/j.omtm.2018.09.003. eCollection 2018 Dec 14. PMID: 30397628 [PubMed]

Grunwald figureOctober 30, 2018 New publication from the Grunwald & Pederson labs. CRISPR-Sirius: RNA scaffolds for signal amplification in genome imaging. Ma H, Tu LC, Naseri A, Chung YC, Grunwald D, Zhang S, Pederson T. Nat Methods. 2018 Nov;15(11):928-931. doi: 10.1038/s41592-018-0174-0. Epub 2018 Oct 30. PMID: 30377374 [PubMed - in process]

October 26, 2018 RTI Nobel Laureate, Craig Mello, to be honored in Fall River, MA. On Nov. 10, Mello will be recognized during a special brunch and birthday bash for the Portugese American community at the Cove restaurant in Fall River. The event will take place from 10am to 2pm.

October 19, 2018 New publication from the Khvorova & Aronin labs.
Exosomes Produced from 3D Cultures of MSCs by Tangential Flow Filtration Show Higher Yield and Improved Activity.
Haraszti RA, Miller R, Stoppato M, Sere YY, Coles A, Didiot MC, Wollacott R, Sapp E, Dubuke M, Li X, Shaffer S, DiFiglia M, Wang Y, Aronin N, Khvorova A. Mol Ther. 2018 Sep 22. pii: S1525-0016(18)30456-8. doi: 10.1016/j.ymthe.2018.09.015. [Epub ahead of print]

Julia AltermanOctober 18, 2018 Julia Alterman (Khvorova lab) presented a webinar for the Huntington's Disease Society of America (HDSA). RNAi-based huntingtin lowering in the central nervous system: a new potential HD therapeutic.

Bruno, Julia, Chantal, AntonOctober 1, 2018 Congratulations to Julia Alterman, Bruno Godhino, Chantal Ferguson and Anton Turanov, (all from the Khvorova lab) for winning poster prizes at the Oligonucleotide Therapeutics Society annual meeting!

Cansu, Zachary, ChantalOctober 1, 2018 Congratulations to Cansu Colpan (Zamore lab), Chantal Ferguson (Khvrova lab), and Zachary Kennedy (Xue lab) for receiving travel awards from the Oligonucleotide Therapeutics Society for the annual meeting!

 September 29, 2018 New publication from the Aronin lab. Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration. Mondo E, Moser R, Gao G, Mueller C, Sena-Esteves M, Sapp E, Pfister E, O'Connell D, Takle K, Erger KE, Liu W, Conlon TJ, DiFiglia M, Gounis MJ, Aronin N. J Huntingtons Dis. 2018 Sep 29. doi: 10.3233/JHD-180302. [Epub ahead of print]

Asthma review imageSeptember 14, 2018 New review from the Pai lab. Environmental influences on RNA processing: Biochemical, molecular and genetic regulators of cellular response. Pai AA, Luca F. WIREs RNA. 14 Sept 2018.

mello paperSeptember 13, 2018 New Publication from the Mello lab. Robust Genome Editing With Short Single-Stranded and Long, Partially Single-Stranded DNA Donors in Caenorhabditis elegans. Dokshin GA,  Ghanta KS, Piscopo KM, and Mello CC. GENETICS. Early online September 13, 2018; doi.org/10.1534/genetics.118.301532

Anastasia KhvorovaSeptember 12, 2018 Anastasia Khvorova presents a webinar for the Oligonucleotide Therapeutics Society. Presentation now online: 'Oligonucleotide Therapeutics: Technology Evolution for Clinically Relevant Tissue Delivery'

September 5, 2018 Congratulations to Enzhi Shen from the Mello Lab for being awarded a Charles A. King Trust Postdoctoral Fellowship in Basic Science!

Sen MarkeyAugust 14, 2018 Senator Markey visits UMass Medical to speak with RTI researchers, Dr. Craig Mello, Dr. Anastasia Khvorova and Chancellor Michael Collins

New genome-editing strategy developed at UMMS may lead to therapeutics Dan Wang, Guangping Gao and colleagues describe approach in Nature Biotechnology

August 13, 2018 New genome-editing strategy developed at UMMS may lead to therapeutics Dan Wang, Guangping Gao, Wen Xue and Phillip D. Zamore describe this approach in Nature Biotechnology.

Wen Xue Phillip Zamore, Guangping Gao Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in miceAugust 13, 2018 New publication from Xue, Zamore, and Gao labs. Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice.  Wang D, Li J, Song CQTran KMou HWu PH, Tai PWLMendonca CARen L,Wang BYSu QGessler DJZamore PDXue W & Gao G. Nat Biotechnol. 13 Aug 2018. doi:10.1038/nbt.4219

Craig Mello, Phillip ZamoreAugust 10, 2018  FDA approves first drug to use RNA interference, based on discoveries made at UMass Medical SchoolThe new drug, patisiran, approved on Aug. 10 by the FDA, is based on the discovery of RNAi made by Craig Mello, Ph.D, and Andrew Fire, Ph.D. It was developed by Alnylam, an RNAi-based drug development company co-founded by Phillip Zamore, Ph.D

Andrei Korostelev Mechanism of premature translation termination on a sense codon.August 10,2018 New publication from Korostelev lab. Mechanism of premature translation termination on a sense codon  Svidritskiy E, Demo G, Korostelev AA. J Biol Chem. 2018 Aug 10;293(32):12472-12479. doi: 10.1074/jbc.AW118.003232. Epub 2018 Jun 25. 

transvascular delivery of siRNAAugust 7, 2018 New publication from the Khvorova lab. Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier. Godinho BMDC, Henninger N, Bouley J, Alterman JF, Haraszti RA, Gilbert JW, Sapp E, Coles AH, Biscans A, Nikan M, Echeverria D, DiFiglia M, Aronin N, Khvorova A. Mol Therapy August 07, 2018 DOI:https://doi.org/10.1016/j.ymthe.2018.08.005

August 5, 2018 Congratulations to Raed Ibraheim from the Sontheimer Lab for being awarded an NIH F31 Fellowship!

August 4, 2018 Congratulations, Zack Kennedy, from the Xue lab, upon receiving an Oligonucleotide Therapeutics Society travel grant! 

Human Gene Therapy Wen Xue Terrence FlotteAugust 1,2018 New publication from Xue lab. In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency Chun-Qing Song, Dan Wang, Tingting Jiang, Kevin O'Connor, Qiushi Tang, Lingling Cai, Xiangrui Li, Zhiping Weng, Hao Yin, Guangping Gao, Christian Mueller, Terence R. Flotte, and Wen Xue. Human Gene Therapy. Aug 2018. http://doi.org/10.1089/hum.2017.225

Phil Zamore Small RNA Northern HybridizationAugust 1, 2018 New publication from Zamore lab. Analysis of Small RNAs by Northern Hybridization Li C, Zamore PD. Cold Spring Harb Protoc. 2018 Aug 1;2018(8):pdb.prot097493. doi: 10.1101/pdb.prot097493. 

Mihir Metkar, Melissa Moore, Job Dekker

July 31, 2018 Update from the Moore lab. Congratulations, Dr. Mihir Metkar PhD for a successful thesis defense with the guidance of Drs. Moore and Dekker! 

Elimination of PCR duplicates in RNA-seq and small RNA-seq using unique molecular identifiersJuly 13, 2018 New Publication from Zamore lab. Elimination of PCR duplicates in RNA-seq and small RNA-seq using unique molecular identifiers Yu Fu, Pei-Hsuan Wu, Timothy Beane, Phillip D. Zamore and Zhiping Weng. BMC Genomics. 2018 Jul 13;19(1):531. doi: 10.1186/s12864-018-4933-1.

Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing

July 6, 2018 New publication from Khvorova, Watts & Sontheimer labs. Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing Aamir Mir, Julia F. Alterman, Matthew R. Hassler, Alexandre J. Debacker, Edward Hudgens, Dimas Echeverria, Michael H. Brodsky, Anastasia Khvorova, Jonathan K. Watts & Erik J. Sontheimer. Nat Commun. 2018 Jul 6;9(1):2641. doi: 10.1038/s41467-018-05073-z.

July 5, 2018 Congratulations to Tomás Rodriguez from the Sontheimer Lab for being awarded Research Supplements to Promote Diversity in Health-Related Research from the NIDDK!

Ankur Sheel Xue Lab

July 5, 2018 Congratulations to Ankur Sheel from the Xue Lab for being awarded an NIH F30 fellowship! 


GalNAc conjugate deliveryJUNE 14, 2018 New publication from the Khvorova lab.  Novel Cluster and Monomer-Based GalNAc Structures Induce Effective Uptake of siRNAs in Vitro and in Vivo.  Sharma VK, Osborn MF, Hassler M, Echeverria D, Ly S, Ulashchik E, Martynenko-Makaev Y, Shmanai VV, Zatsepin TS, Khvorova A, Watts J.Bioconjug Chem. 2018 Jun 13. doi: 10.1021/acs.bioconjchem.8b00365.

siRNA DeliveryMAY 11, 2018 New publication from the Khvorova lab.  Improving Small Interfering RNA Delivery In Vivo Through Lipid ConjugationOsborn MF, and Khvorova A. doi.org/10.1089/nat.2018.0725

siRNA brain silencing MAY 10, 2018 New publication from the Khvorova labEfficient Gene Silencing in Brain Tumors with Hydrophobically Modified siRNAsOsborn MF, Coles AH, Golebiowski D, Echeverria D, Moazami MP, Watts JK, Sena-Esteves M, and Khvorova A. Molecular Cancer Therapeutics (2018)  10.1158/1535-7163.MCT-17-1144

C-BERST telomere identificationMAY 7, 2018 New publication from the Sontheimer labC-BERST: defining subnuclear proteomic landscapes at genomic elements with dCas9–APEX2. Gao XD, Tu LC, Mir A, Rodriguez T, Ding Y, Leszyk J, Dekker J, Shaffer SA, Zhu LJ,  Wolfe SA, & Sontheimer EJ. Nature Methods (2018) doi:10.1038/s41592-018-0006-2

Anastasia Khvorova Andrei Korostelev

May 4, 2018  Congratulations to Dr. Anastasia Khvorova and Dr. Andrei Korostelev for receiving tenure! This is a very fitting acknowledgment for both due to their hard work, fantastic mentorship and scholarly contributions.

70s Ribosome conformationMAY 3, 2018 New publication from the Korostelev labConformational Control of Translation Termination on the 70S RibosomeSvidritskiy E, Korostelev AA. Structure. 2018 Apr 23. pii: S0969-2126(18)30126-6. doi: 10.1016/j.str.2018.04.001.

Reka HarasztiAPRIL 23, 2018 News from the Khvorova lab.  Congratulations, Dr. Reka Haraszti for successfully defending her thesis, the first to graduate from Dr. Anastasia Khvorova's lab!

Timothy Chang PH.DAPRIL 20, 2018 News from the Zamore lab.  Congratulations, Dr. Timothy Chang, MD/PhD student for successfully defending his thesis!

In vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT DeficiencyMARCH 29, 2018
 New publication from the Xue lab.  
In vivo genome editing partially restores alpha1-antitrypsin in a murine model of AAT deficiency. Human Gene Therapy 29 March 2018; Dr. Chun-Qing Song, Dr. Dan Wang, Dr. Tingting Jiang, Mr. Kevin O'Connor, Dr. Qiushi Tang, Ms. Lingling Cai, Ms. Xiangrui Li, Dr. Zhiping Weng, Dr. Hao Yin, Dr. Guangping Gao, Dr. Christian Mueller, Dr. Terence R Flotte, Dr. Wen Xue.  https://doi.org/10.1089/hum.2017.225

Daniel Xin GaoMARCH 28, 2018  Congratulations Xin Daniel Gao, Sontheimer lab for receiving the 2017 - 2018 Fall - Winter Riccio-GSBS Travel Award!


Khvorova Lab

MARCH 6, 2018 New editorial from the Khvorova lab.  Nucleic Acids Research and Nucleic Acid Therapeutics Nucleic Acids Res. 2018 Feb 28;46(4):1563-1564.  Stoddard BL, Khvorova A, Corey DR, Dynan WS, Fox KR. doi: 10.1093/nar/gky059.

neo-CRISPR genesis

FEBRUARY 20, 2018 New publication from the Sontheimer labCRISPRs from scratch. Nature Microbiology 3, 261–262. Edraki, A, Sontheimer, E. doi:10.1038/s41564-018-0115-9


Wen XueFEBRUARY 14, 2018 New publication from the Xue lab.  CRISPR–Cas-related technologies in basic and translational liver research. Nature Reviews Gastroenterology & Hepatology. 2018. Song C-Q, Xue, W.  doi:10.1038/nrgastro.2018.11

Model. piRNAs Scan mRNAs within Perinuclear Nuage prior to Translation InitiationFEBRUARY 14, 2018
 New publication from the Mello lab.  
The Coding Regions of Germline mRNAs Confer Sensitivity to Argonaute Regulation in C. elegans. Seth M, Shirayama M, Tang W, Shen EZ, Tu S, Lee HC, Weng Z, Mello CC. Cell Rep. 2018 Feb 14. pii: S2211-1247(18)30177-3. doi: 10.1016/j.celrep.2018.02.009.

21ux-1 Acts with sex-1 to Regulate Dosage Compensation and Sex Determination

FEBRUARY 13, 2018 New publication from the Mello lab.  A Sex Chromosome piRNA Promotes Robust Dosage Compensation and Sex Determination in C. elegans. Tang W, Seth M, Tu S, Shen EZ, Li Q, Shirayama M, Weng Z, Mello CC. Dev Cell. 2018 Feb 13. pii: S1534-5807(18)30060-1. doi: 10.1016/j.devcel.2018.01.025.

piRNA binding sites FEBRUARY 10, 2018 New publication from the Mello labIdentification of piRNA Binding Sites Reveals the Argonaute Regulatory Landscape of the C. elegans Germline. Shen EZ, Chen H, Ozturk AR, Tu S, Shirayama M, Tang W, Ding YH, Dai SY, Weng Z, Mello CC. Cell. 2018 Feb 10. pii: S0092-8674(18)30117-X. doi: 10.1016/j.cell.2018.02.002

siRNA comparisonFEBRUARY 8, 2018 New publication from the Khvorova labComparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo.  Hassler MR, Turanov AA,  Alterman JF,  Haraszti RA, Coles AH,  Osborn MF,  Echeverria D,  Nikan M, Salomon WE,  Roux L,  Godinho BMDC,  Davis SM, Morrissey DV,  Zamore PD,  Karumanchi SA,  Moore MJ, Aronin N,  Khvorova A. Nucleic Acids Research, gky037, https://doi.org/10.1093/nar/gky037

exosome loading siRNAFEBRUARY 2, 2018 New publication from the Aronin & Khvorova lab. Loading of Extracellular Vesicles with Hydrophobically Modified siRNAs. Didiot MC, Haraszti RA, Aronin N, Khvorova A.Methods Mol Biol. 2018;1740:199-214. doi: 10.1007/978-1-4939-7652-2_16

piRNA Hi5 novel modelJANUARY  29, 2018 New publication from the Zamore lab.  The genome of the Hi5 germ cell line from Trichoplusia ni, an agricultural pest and novel model for small RNA biology.  Fu Y, Yang Y, Zhang H, Farley G, Wang J, Quarles KA, Weng Z, Zamore PD. Elife. 2018 Jan 29;7. pii: e31628. doi: 10.7554/eLife.31628. [Epub ahead of print] PMID: 29376823 [PubMed - as supplied by publisher]

Blasticidin S Translation Inhibiton JANUARY  26, 2018 New publication from the Korostelev lab.  Mechanism of Inhibition of Translation Termination by Blasticidin S.  Svidritskiy E, Korostelev AA. J Mol Biol. 2018 Jan 19. pii: S0022-2836(18)30010-X. doi: 10.1016/j.jmb.2018.01.007. 

Frataxin Activation; Locked Nucleic AcidJANUARY  18, 2018 New publication from the Watts lab.  Activation of Frataxin Protein Expression by Antisense Oligonucleotides Targeting the Mutant Expanded Repeat. Li L, Shen X, Liu Z, Norrbom M, Prakash TP, O'Reilly D, Sharma VK, Damha MJ, Watts JK, Rigo F, Corey DR. Nucleic Acid Ther. 2018 Jan 17. doi: 10.1089/nat.2017.0703.

Frataxin Protien Activation JANUARY 8, 2018 New publication from the Watts lab. Oligonucleotides Targeting TCF4 Triplet Repeat Expansion Inhibit RNA Foci and Mis-splicing in Fuchs' Dystrophy. Hu J, Rong Z, Gong X, Zhou Z, Sharma VK, Xing C, Watts JK, Corey DR, Mootha VV.  Hum Mol Genet. 2018 Jan 8. doi: 10.1093/hmg/ddy018.

kinetics of premRNA splicing DrosophilaDECEMBER 27, 2017 New publication from the Pai lab. The kinetics of pre-mRNA splicing in the Drosophila genome and the influence of gene architecture. Pai AA, Henriques T, McCue K, Burkholder A, Adelman K, Burge CB. Elife. 2017 Dec 27;6. pii: e32537. doi: 10.7554/eLife.32537.

Structural dynamics protein S1 70s Ribosome ensemble cryo-emDECEMBER 13, 2017 New publication from the Korostelev lab.  Structural dynamics of protein S1 on the 70S ribosome visualized by ensemble cryo-EM.  Loveland AB, Korostelev AA. Methods. 2017 Dec 13. pii: S1046-2023(17)30290-6. doi: 10.1016/j.ymeth.2017.12.004. 

arthropod piRNA analysis transposable elementsDECEMBER 11, 2017 New publication from the Zamore lab. Pan-arthropod analysis reveals somatic piRNAs as an ancestral defence against transposable elements. Lewis SH, Quarles KA, Yang Y, Tanguy M, Frézal L, Smith SA, Sharma PP, Cordaux R, Gilbert C, Giraud I, Collins DH, Zamore PD, Miska EA, Sarkies P, Jiggins FM. Nat Ecol Evol. 2017 Dec 4. doi: 10.1038/s41559-017-0403-4. 

miRNA rAAV reduces mutant Huntingtin mRNA in transgenic sheep modelDECEMBER 5, 2017 New publication from the Aronin lab. Artificial miRNAs reduce human mutant Huntingtin throughout the striatum in a transgenic sheep model of Huntington's disease. Pfister E, Dinardo N, Mondo E, Borel F, Conroy F, Fraser C, Gernoux G, Han X, Hu D, Johnson E, Kennington L, Liu P, Reid S, Sapp E, Vodicka P, Kuchel T, Morton AJ, Howland D, Moser R, Sena-Esteves M, Gao G, Mueller C, DiFiglia M, Aronin N. Hum Gene Ther. 2017 Dec 5. doi: 10.1089/hum.2017.199. 

type II CRISPR DECEMBER 4, 2017 New publication from the Sontheimer lab. Type II-C CRISPR-Cas9 Biology, Mechanism and Application. Mir A, Edraki A, Lee J, Sontheimer EJ. ACS Chem Biol. 2017 Dec 4. doi: 10.1021/acschembio.7b00855. 

Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brainNOVEMBER 21, 2017 New publication from the Aronin & Khvorova labs. 
Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain. 
Sanchez-Ramos J, Song S, Kong X, Foroutan P, Martinez G, Dominguez-Viqueria W, Mohapatra S, Mohapatra S, Haraszti RA, Khvorova A, Aronin N, Sava V. J Drug Deliv Sci Technol. 2018 Feb;43:453-460. doi: 10.1016/j.jddst.2017.11.013. Epub 2017 Nov 21.

modifications to guide RNA in vivo genome editingNOVEMBER 13, 2017 New publication from the Xue lab. Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing. Yin H, Song CQ, Suresh S,  Wu Q, Walsh S, Rhym LH, Mintzer E, Bolukbasi MF, Zhu LJ, Kauffman K, Mou H, Oberholzer A, Ding J, Kwan SY, Bogorad RL, Zatsepin T, Koteliansky V, Wolfe SA, Xue W, Langer R & Anderson DG. For more on this paper, click here.

Melissa Moore Symposium Group PictureOCTOBER 25-26, 2017:  Symposium to be held in honor of Melissa Moore's academic career. See Moore Lab homepage for announcement.

Erik Sontheimer Andrei KorostelevOCTOBER 20, 2017: Congrats to both Andrei Korostelev & Erik Sontheimer for receiving
awards! Andrei will be recognized for the Early-Career Award and Erik will be recognized for the Mid-Career Award! Story headlining UMassMedNow here, and announcement on The RNA Society's website here.

RNA Polymerase bound to 30s Ribosomal Subunit OCTOBER 13, 2017 New publication from the Korostelev lab. Structure of RNA polymerase bound to ribosomal 30S subunit. Demo G, Rasouly A, Vasilyev N, Svetlov V, Loveland AB, Diaz-Avalos R, Grigorieff N, Nudler E, Korostelev A. eLife 2017;6:e28560 DOI: 10.7554/eLife.28560

Pharmacokinetic Profiling of Conjugated Therapeutic OligonucleotidesOCTOBER 11, 2017 New publication from the Khvorova lab. Pharmacokinetic Profiling of Conjugated Therapeutic Oligonucleotides: A High-Throughput Method Based Upon Serial Blood Microsampling Coupled to Peptide Nucleic Acid Hybridization Assay. Godinho BMDC, Gilbert JW, Haraszti RA, Coles AH, Biscans A, Roux L, Nikan M, Echeverria D, Hassler M, Khvorova A. Nucleic Acid Ther. 2017 Oct 12. doi: 10.1089/nat.2017.0690. 

Li Weibo Institute for Rare Disease ResearchOCTOBER 11, 2017 UMass Medical School announces The Li Weibo (李伟波) Institute for Rare Diseases Research: $10 million endowment gift from Li Weibo Charitable Foundation to support world-leading research at UMMS. Learn more here.

18s rRNA ribosomal decay non-functionalSEPTEMBER 27, 2017 New publication from the Moore lab. 
ASC1 and RPS3: New actors in 18S non-functional rRNA decay. Limoncelli KA, Merrikh CN, Moore MJ. RNA. 2017 Sep 27. pii: rna.061671.117. doi: 10.1261/rna.061671.117.

Alterations in mRNA 3′ UTR Isoform Abundance Accompany Gene Expression Changes in Human Huntington’s Disease BrainsSEPTEMBER 26, 2017 New publication from the Aronin lab. Alterations in mRNA 3' UTR Isoform Abundance Accompany Gene Expression Changes in Human Huntington's Disease Brains.  Romo L, Ashar-Patel A, Pfister E, Aronin N. Cell Rep. 2017 Sep 26;20(13):3057-3070. doi: 10.1016/j.celrep.2017.09.009

Diatrack particle tracking softwareSEPTEMBER 25, 2017 New publication from the Grunwald lab. Diatrack particle tracking software: review of applications and performance evaluation. Vallotton P, van Oijen AM, Whitchurch CB, Gelfand V, Yeo L, Tsiavaliaris G, Heinrich S, Dultz E, Weis K, and Grunwald D. Traffic. Accepted manuscript online: 25 SEP 2017 DOI: 10.1111/tra.12530

Transcriptome wide Poly Adenylation sites preeclampsiaSEPTEMBER 25, 2017 New publication from the Moore lab. FLT1 and transcriptome-wide polyadenylation site (PAS) analysis in preeclampsia. Ashar-Patel A, Kaymaz Y, Rajakumar A, Bailey JA, Karumanchi SA, Moore MJ. Sci Rep. 2017 Sep 22;7(1):12139. doi: 10.1038/s41598-017-11639-6.

Oligonucleotide Therapeutics Society Craig Mello Jonathan WattsSEPTEMBER 24, 2017 Craig Mello presented a keynote address, 'A Worms Tale: Oligonucleotide-Guided Gene Regulation and Secrets of Inheritance and Immortality', at the 13th Annual Oligotherapeutics Society meeting. Jonathan Watts presented his work on 'Modification and Multimerization of Therapeutic Oligonucleotides'. Annabelle Biscans, Khvorova lab, was selected by the committee to give an oral presentation; Socheata Ly, Khvorova lab & Alexandre Debacker, Watts lab, both were awarded travel grants.

Li-Chun Tu David Grunwald SEPTEMBER 15, 2017 Congratulations, Li-Chun Tu, recipient of an NIH K99 grant award 'Deciphering real-time dynamics of the human genome organization in response to DNA damage and gene expression'! This proposal describes an approach that allows chromosome dynamics to be directly visualized, tracked, and characterized at high resolution in live cells. Understanding the dynamics and mechanisms of chromosome rearrangements in response to DNA damage and transcriptional activation will significantly improve our understanding of human genome stability and nuclear function.

Locked Nucleic Acid Gapmers and Conjugates Potently Silence ADAM33, an Asthma-Associated Metalloprotease with Nuclear-Localized mRNASEPTEMBER 15, 2017 New publication from the Watts lab. Locked Nucleic Acid Gapmers and Conjugates Potently Silence ADAM33, an Asthma-Associated Metalloprotease with Nuclear-Localized mRNA. Pendergraff HM, Krishnamurthy PM, Debacker AJ, Moazami MP, Sharma VK, Niitsoo L, Yu Y, Tan YN, Haitchi HM, Watts JK. Mol Ther Nucleic Acids. 2017 Sep 15.

Andrei Korostelev Lab RibosomeSEPTEMBER 12, 2017 Congratulations to Andrei Korostelev, recipient of the Earl & Thressa Stadtman Scholar Award! Dr. Korostelev will be awarded this honor and present a lecture at the annual American Society for Biochemistry & Molecular Biology meeting in April in San Diego, CA, USA. Learn more here.

Rhino gives voice to silent chromatinSEPTEMBER 7, 2017 Nature News & Views article written by Dr. Phillip Zamore, "Molecular biology: Rhino gives voice to silent chromatin" Nature 549, 38–39. 07 September 2017.

 The genome of Trichoplusia ni, an agricultural pest and novel model for small RNA biologySEPTEMBER 4, 2017 New publication from the Zamore lab. The genome of Trichoplusia ni, an agricultural pest and novel model for small RNA biology. Fu Y, Yang Y, Zhang H, Farley G, Wang J, Quarles KA, Weng Z, Zamore PD. BioRxiv, posted Sept 4.

Loading of Extracellular Vesicles with Chemically Stabilized Hydrophobic siRNAs for the Treatment of Disease in the Central Nervous System.

SEPTEMBER 1, 2017 New publications from the Khvorova lab:
Loading of Extracellular Vesicles with Chemically Stabilized Hydrophobic siRNAs for the Treatment of Disease in the Central Nervous System. Haraszti RA, Coles A, Aronin N, Khvorova A, Didiot MC. Bio Protoc. 2017 Aug 20;7(16)

A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons in vitro with Oligonucleotide Therapeutics   A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons in vitro with Oligonucleotide Therapeutics  Alterman JF, Coles A, Hall LM, Aronin N, Khvorova A, Didiot MC. Bio Protoc. 2017 Jun 20;7(12). 

Jonathan WattsSEPTEMBER 1, 2017 Congratulations to Jonathan Watts, the first recipient of the Ono Pharma Breakthrough Science Initiative Award! Awards of $300,000/year for oligonucleotide medicine research, possibly renewable, totaling a maximum of $900,000 for the normal term of a research project in oligonucleotide research.

Oligonucleotide Therapeutics Society Craig Mello Jonathan WattsAUGUST 29, 2017 Congratulations to Socheata Ly, Khvorova Lab and Alexandre Debacker, Watts Lab for receiving travel awards to the Oligonucleotide Therapeutics Society meeting in Bordeaux, France, September 24-27. Congratulations to Annabelle Biscans for being selected to present a short talk at this meeting. RTI Faculty Jonathan Watts and Craig Mello were invited to present talks, Craig Mello will be keynoting this conference.


AUGUST 23, 2017 New publication from the Sontheimer lab. A Broad-Spectrum Inhibitor of CRISPR-Cas9. Harrington LB, Doxzen KW, Ma E, Liu JJ, Knott GJ, Edraki A, Garcia B, Amrani N, Chen JS, Cofsky JC, Kranzusch PJ, Sontheimer EJ, Davidson AR, Maxwell KL, Doudna JA.Cell. 2017 Aug 23. pii: S0092-8674(17)30873-5. doi: 10.1016/j.cell.2017.07.037. [Epub ahead of print]

Mouse cerebellumAUGUST 23, 2017 RTI Gallery of images LIVE as a Cell Picture Show, hosted by the publisher of the journal CellTo look at all of the images in this gallery, click here. For more information, see this link to our UMassMedNow article here.

Anastasia Khvorova

AUGUST 16, 2017 U.S. Rep. Jim McGovern meets with Anastasia Khvorova, Chancellor Michael    Collins, Dean Terry Flotte, Beth   McCormick and others to discuss critical NIH funding for UMass   Medical School. Video of the discussion can be found here. Link to the article in the Worcester Telegram & Gazette here.


JUNE 29, 2017 New publication from the Sontheimer lab. Inhibition of CRISPR-Cas systems by mobile genetic elements. Sontheimer EJ, Davidson AR. Curr Opin Microbiol. 2017 Jun 29;37:120-127. doi: 10.1016/j.mib.2017.06.003. [Epub ahead of print] Review.

Exon SkippingJUNE 14, 2017 New publication from the Xue, Sontheimer, & Moore labs. CRISPR/Cas9-mediated genome editing induces exon skipping by alternative splicing or exon deletion. Mou H†, Smith JL†, Peng L, Yin H, Moore J, Zhang XO, Song CQ, Sheel A, Wu Q, Ozata DM, Yingxiang Li Y, Anderson DG, Emerson CP, Sontheimer EJ, Moore MJ, Weng Z, Xue W. Genome Biology 2017 18:108 DOI: 10.1186/s13059-017-1237-8  Editorial hihlighting this publication can be found here.

hsiRNA structureJUNE 12, 2017 New publication from the Khvorova lab. 5-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo. Haraszti RA, Roux L, Coles AH, Turanov AA, Alterman JF, Echeverria D, Godinho BMDC, Aronin N, Khvorova A. Nucleic Acids Res. 2017 Jun 7. doi: 10.1093/nar/gkx507. [Epub ahead of print] PMID: 28591791

Jonathan Watts, PhDJUNE 7, 2017 Worcester Foundation Research Grant recipients awarded to three faculty members in the RTI: 
David Grünwald, Ph.D.“Prototype development for a 20 color, 3D real time microscope”
Andrei Korostelev, Ph.D., “Elucidating the translation-transcription coupling”
Jonathan Watts, Ph.D.“T to C conversion for amplifiable metabolic labeling of DNA at single base resolution”

Andrei Korostelev, PhDMAY 24, 2017 New publication from the Korostelev lab. “Transfer RNAs & the ribosome decoding centers that read their genetic instructions are almost the same for all life on earth—that’s why we are very excited by this discovery, which explains the accuracy of genetic code reading,” -Dr. Korostelev   Ensemble cryo-EM elucidates the mechanism of translation fidelity. Loveland AB, Demo G, Grigorieff N, & Korostelev AA. Nature (2017) doi:10.1038/nature22397  For details, please click here

Melissa Moore, PhD

MAY 2, 2017 Melissa Moore, Ph.D., Eleanor Eustis Farrington Chair in Cancer Research and Professor in the RNA Therapeutics Institute has been newly elected to The National Academy of Sciences. Dr. Moore, who was appointed chief scientific officer of the messenger RNA platform at Moderna Therapeutics last fall, was among 84 new members and 21 foreign associates elected, in recognition of their distinguished and continuing achievements in original research. For details, please click here.

Mouse brain sectionMAY 2, 2017 New publication from the Khvorova & Aronin labs. Synthesis and Evaluation of Parenchymal Retention and Efficacy of a Metabolically Stable, O-phosphocholine-N-docosahexaenoyl-L-serine siRNA Conjugate in Mouse Brain. Nikan M, Osborn MF, Coles AH, Biscans A, Godinho B, Haraszti R, Sapp E, Echeverria D, DiFiglia M, Aronin N, and Khvorova A. Just Accepted Manuscript. Bioconjugate Chem, doi: 10.1021/acs.bioconjchem.7b00226

Ami Ashar-PatelAPRIL 21, 2017 Ami Ashar-Patel, a PhD candidate in the Moore lab, has been selected to participate in the Keck Graduate Institute (KGI) Certificate in Bioscience Management Program. She received a KGI Burroughs Wellcome Fund scholarship and a UMass Medical School Graduate School of Biomedical Sciences career development grant. For details, please click here.

Structure and dimer formation of RlmH.

APRIL 20, 2017 New publication from the Korostelev & Zamore labs. Small methyltransferase RlmH assembles a composite active site to methylate a ribosomal pseudouridine Koh CS, Madireddy R, Beane TJ, Zamore PD, & Korostelev AA. Scientific Reports 7, Article number: 969 (2017) doi:10.1038/s41598-017-01186-5 PMID: 28428565 

Neil AroninAPRIL 12, 2017 Neil Aronin, MD, Professor of Medicine & RNA Therapeutics Institute, co-director of the Neurotherapeutics Institute, chief of the division of endocrinology & metabolism will serve as the Higgins Family Professor of Neuroscience. A member of the UMMS faculty since 1981, Dr. Aronin established and has maintained a groundbreaking research focused on Huntington’s disease; and recent explorations of how gene therapy might be used to slow the progression, may lead to promising clinical trials.

APRIL 10, 2017 Andrei Korostelev, PhD, Associate Professor of RNA Therapeutics Institute, was in the Worcester Telegram & Gazette this past Saturday.  Andrei discussed how the new Cryo-EM at UMASS Medical School helps his lab study the movement and function of the ribosome, the most important cellular machine for making proteins. For details, please click here.

APRIL 4, 2017  New publication from the Korostelev lab. “This is an important paper that describes an alternate rescue system in bacteria for ribosomes that are stalled on an mRNA without a stop codon.” by a reviewer. Mechanism of ribosome rescue by ArfA and RF2. Demo G, Svidritskiy E, Madireddy R, Diaz-Avalos R, Grant T, Grigorieff N, Sousa D, Korostelev AA.  Elife. 2017 Mar 16;6. pii: e23687. doi: 10.7554/eLife.23687. PMID: 28300532.

MARCH 20, 2017  New publication from the Mello lab. The Antiviral RNA Interference Response Provides Resistance to Lethal Arbovirus Infection and Vertical Transmission in Caenorhabditis elegans.  Gammon DB, Ishidate T, Li L, Gu W, Silverman N, Mello CC.  Curr Biol. 2017 Mar 20;27(6):795-806. doi: 10.1016/j.cub.2017.02.004. Epub 2017 Mar 2. PMID: 28262484

FEBRUARY 15, 2017  Dr. Erik J. Sontheimer, professor in the RNA Therapeutics Institute, is quoted in Science in a story about the patent battle over CRISPR/Cas9 technology.  For details, please click here.

FEBRUARY 13, 2017  Wen Xue, PhD, Assistant Professor of RTI, is in The Boston Globe talking about promising Immunotherapy fighting lung cancer. For details, please click here.

FEBRUARY 1, 2017  Phillip D. ZamorePhD, co-director of the RNA Therapeutics Institute is in The Scientist to talk about RNA interference between plant and pathogen. For details, please click here.

Anastasia KhvorovaJANUARY 6, 2017  Anastasia Khvorova, PhD, professor of RNA Therapeutics Institute, explains the promise of oligonucleotide therapeutics, including the results of a Phase I clinical trial of a new injectable cholesterol-lowering drug Inclisiran in NEJM. Inclisiran is a chemically modified siRNA developed by Alnylam Pharmaceuticals, a company co-founded by Phillip Zamore, PhD, Howard Hughes Medical Institute Investigator, the Gretchen Stone Cook Chair of Biomedical Sciences & director of the RTI. For details, please click here.

DECEMBER 12, 2016  New publication from the Xue lab. This is a featured article on the journal homepage with an accompanying editorial. Genome-wide CRISPR Screen Identifies Regulators of MAPK as Suppressors of Liver Tumors in Mice. Song C-Q, Li Y, Mou H, Moore J, Park A, Pomyen Y, Hough S, Kennedy Z, Fischer A, Yin H, Anderson DG, Conte Jr D, Zender L, Wang XW, Thorgeirsson S, Weng Z, Xue W  Gastroenterology (2017), doi: 10.1053/j.gastro.2016.12.002

NOVEMBER 28, 2016  New publication from the Khvorova lab. Visualization of self-delivering hydrophobically modified siRNA cellular internalization.  Ly S, Navaroli DM, Didiot MC, Cardia J, Pandarinathan L, Alterman JF, Fogarty K, Standley C, Lifshitz LM, Bellve KD, Prot M, Echeverria D, Corvera S, Khvorova A. Nucleic Acids Res. 2016 Nov 28. pii: gkw1005. [Epub ahead of print] PMID: 27899655

NOVEMBER 20, 2016  New publication from the Khvorova lab.  High-resolution proteomic and lipidomic analysis of exosomes and microvesicles from different cell sources. Haraszti RA, Didiot MC, Sapp E, Leszyk J, Shaffer SA, Rockwell HE, Gao F, Narain NR, DiFiglia M, Kiebish MA, Aronin N, Khvorova A.J Extracell Vesicles. 2016 Nov 17;5:32570. doi: 10.3402/jev.v5.32570. eCollection 2016. PMID:27863537

OCTOBER 24,  2016  New publication from the Khvorova lab Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing. Didiot MC, Hall LM, Coles AH, Haraszti RA, Godinho BM, Chase K, Sapp E, Ly S, Alterman JF, Hassler MR, Echeverria D, Raj L, Morrissey DV, DiFiglia M, Aronin N, Khvorova A. Mol Ther. 2016 Oct;24(10):1836-1847. doi: 10.1038/mt.2016.126. PMID: 27506293

OCTOBER 17, 2016  Dr. Anastasia Khvorova, Professor of RNA Therapeutics Institute, was featured in Sunday's Telegram.  On October 16th, Dr. Khvorova and Julia Alterman, a graduate student in her lab, were both in the cover story “ Searching for cures at UMASS”. For details, please click here.

OCTOBER 13, 2016  New publication from the Zamore lab. Rapid Screening for CRISPR-Directed Editing of the Drosophila Genome Using white Coconversion. Ge DT, Tipping C, Brodsky MH, Zamore PD. G3 (Bethesda). 2016 Oct 13;6(10):3197-3206. doi: 10.1534/g3.116.032557. PMID: 27543296

OCTOBER 4, 2016  Wen Xue, PhD, Assistant professor of RNA Therapeutics Institute, won a DP2 award from NIH. The Xue lab research focuses on building precision mouse models of cancer and genetic diseases using RNA tools.  Wen's project titled "CRISPR-BASED MODULAR THERAPY FOR PRECISION MEDICINE " won the NIH Director’s New Innovator Award, which is part of a NIH High-Risk, High-Reward Research (HRHR) program supporting highly creative and exceptional scientists with 3bold approaches to major challenges in biomedical research. This award will be for five years, total about 2.5 millon dollars. For details, please click here.

SEPTEMBER 23, 2016  Dr. Wen Xue, Assistant professor of RNA Therapeutics Institute received grant from Hyundai Hope on Wheels. Wen accepted a $250,000 grant on Friday, Sept. 23 from the Hyundai Motor America Hope on Wheels campaign to continue his work toward improving care and treatment options for children and adults with liver cancer. For details, please click here.

SEPTEMBER 23, 2016  Dr. David Grunwald, Assistant Professor of RNA Therapeutics Institute, awarded R01. The Grunwald lab received an R01 grant from NIH/NIGMS for the project "Canonical and non-canonical nuclear HIV-RNA gene export in living cells". 

SEPTEMBER 19, 2016  Wen Xue, PhD, Assistant Professor of RTI,  selected to receive award from The Lung Cancer Research Foundation (LCRF).   Dr. Xue received the award as an extension for his LCRF grant of last year on his research targeting small RNA-mediated KRAS inhibition in lung cancer. His application received outstanding reviews.

AUGUST 26, 2016  New publication from the Watts lab.  Single-Stranded Silencing RNAs: Hit Rate and Chemical Modification. Pendergraff HM, Debacker AJ, Watts JK. Nucleic Acid Ther. 2016 Aug;26(4):216-22. doi: 10.1089/nat.2015.0557. PMID: 27123752

AUGUST 15, 2016 New publication from the Khvorova labDocosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain. Nikan M, Osborn MF, Coles AH, Godinho BM, Hall LM, Haraszti RA, Hassler MR, Echeverria D, Aronin N, Khvorova A. Mol Ther Nucleic Acids. 2016 Aug 9;5(8):e344. doi: 10.1038/mtna. 2016.50. PMID:27504598

AUGUST 10, 2016  Dr. Wen Xue, Assistant professor of RNA Therapeutics Institute, awarded a subproject of P01 grant with the UMass program project team. The Xue lab received a five-year P01 grant (PI: Dr. Terence R. Flotte) from NIH/NHLBI to study “Liver-directed somatic gene correction rAAV system of regulatable Cas9/sgRNA”. For details, please click here.

AUGUST 4, 2016  Paper of the year award from OTS goes to the Zamore and Moore Labs. Paper entitled “Single-Molecule Imaging Reveals that Argonaute Reshapes the Binding Properties of Its Nucleic Acid Guides” has been selected by the Oligonucleotide Therapeutics Society awards committee as the OTS paper of the year for the period June 2015-June 2016. Paper of the Year award is to honor the most impactful paper in the field of oligonucleotide therapeutics. Formal presentation of the award will be at the OTS Annual Meeting in Montreal, Canada this year.

JULY 26, 2016  Dr. Anastasia Khvorova, Professor of RNA Therapeutics Institute, awarded R01.  The Khvorova lab received a five-year R01 grant from NIH/NICHD to study “Development of RNAi based sFLT1-targeting therapeutics for treatment of Preeclampsia”.

JULY 19, 2016  New publication from the Korostelev lab.  Ribosome•RelA structures reveal the mechanism of stringent response activation. Loveland AB, Bah E, Madireddy R, Zhang Y, Brilot AF, Grigorieff N, Korostelev AA. Elife. 2016 Jul 19;5. pii: e17029. doi: 10.7554/eLife.17029. PMID: 27434674

JUNE 6, 2016  Bruno Godinho, PhD, awarded a Milton Safenowitz Post Doctoral Fellowship. Dr Bruno Godinho in the Khvorova lab was selected by The ALS Association for his research project "Silencing mutant SOD1 and C9orf72 in vivo using innovative hydrophobic siRNA scaffolds: a novel therapeutic path for the treatment of ALS". He will be supported for two years.

MAY 31, 2016  New publication from the Moore lab. Single molecule analysis reveals reversible and irreversible steps during spliceosome activation. Hoskins AA, Rodgers ML, Friedman LJ, Gelles J, Moore MJ. Elife. 2016 May 31;5. pii: e14166. doi: 10.7554/eLife.14166. PMID: 27244240

MAY 22, 2016  New publication from the Korostelev lab.  Structural Basis for Translation Termination on a Pseudouridylated Stop Codon. Svidritskiy E, Madireddy R, Korostelev AA. J Mol Biol. 2016 May 22;428(10 Pt B):2228-36. doi: 10.1016/j.jmb.2016.04.018. PMID: 27107638

MAY 12, 2016  Erik Sontheimer, PhD, Professor of the RNA Therapeutics Institute, received a four-year award from NIH/NIGMS to study the “Engineered Cas9 Nucleases with Single-Genomic-Site Precision for CYBB Correction”, with co-investigator Scot Wolfe. This award will be used to further the development of high-fidelity CRISPR-derived genome editing platforms that provide single-site precision within the human genome. These platforms will then be used to correct mutations in the cells of patients with chronic granulomatous disease, which often leads to premature death due to immune dysfunction and severe recurrent infections.

MAY 9, 2016  New publication from the Korostelev lab.  Ensemble cryo-EM uncovers inchworm-like translocation of a viral IRES through the ribosome. Abeyrathne PD, Koh CS, Grant T, Grigorieff N, Korostelev AA. Elife. 2016 May 9;5. pii: e14874. doi: 10.7554/eLife.14874. PMID: 27159452

APRIL 18, 2016  New publication from the Pedersen lab.  Multiplexed labeling of genomic loci with dCas9 and engineered sgRNAs using CRISPRainbow.  Ma H, Tu LC, Naseri A, Huisman M, Zhang S, Grunwald D, Pederson T.  Nat Biotechnol. 2016 May;34(5):528-30. doi: 10.1038/nbt.3526. Epub 2016 Apr 18. PMID: 27088723

APRIL 11, 2016  Wen Xue, PhD, Assistant professor of the RNA Therapeutics Institute, won UMASS Clinical and Translational Science Award (UMCCTS) Pilot Award. The award will support Wen's project “CRISPR-based precision medicine for tyrosinemia” for a year.

APRIL 11, 2016  New publication from the Khvorova lab.  A High-Throughput Method for Direct Detection of Therapeutic Oligonucleotide-Induced Gene Silencing In Vivo.  Coles AH, Osborn MF, Alterman JF, Turanov AA, Godinho BM, Kennington L, Chase K, Aronin N, Khvorova A. Nucleic Acid Ther. 2016 Apr;26(2):86-92. doi: 10.1089/nat.2015.0578. Epub 2015 Nov 23. PMID: 26595721


APRIL 7, 2016 Wen Xue, PhD, Assistant professor of the RNA Therapeutics Institute at UMass Medical School, won research grant from ASC.  Wen will use the $791,000 in funds to study KRAS inhibition in lung cancer, according to the American Cancer Society. For more, please click here.

MARCH 26, 2016  Maire Osborn, PhD, received NIH research fellowship award. Dr. Osborn, a postdoctoral fellow in the  Khvorova lab, received a NIH research fellowship award from National Institutes of Neurological Disorders and Stroke. The award will support her for three years on her project titled " Enhancing siRNA delivery and efficacy in the brain through conjugation of bioactive, polyunsaturated fatty acids".

MARCH 19, 2016  New publication from the Aronin lab.  Does the Mutant CAG Expansion in Huntingtin mRNA Interfere with Exonucleolytic Cleavage of its First Exon? Liu W, Pfister EL, Kennington LA, Chase KO, Mueller C, DiFiglia M, Aronin N. J Huntingtons Dis. 2016 Mar 19;5(1):33-8. doi: 10.3233/JHD-150183. PMID: 27003665

FEBRUARY 26, 2016  Erik Sontheimer, PhD, Professor of the RNA Therapeutics Institute, elected to Fellowship in the American Academy of Microbiology The Academy, the honorific leadership group within the American Society for Microbiology, recognizes excellence, originality, and leadership in the microbiological sciences, election to this group is a mark of distinction.

FEBRUARY 25, 2016  New publication from the Mello lab. The RNase PARN-1 Trims piRNA 3' Ends to Promote Transcriptome Surveillance in C. elegans. Tang W, Tu S, Lee HC, Weng Z, Mello CC.  Cell. 2016 Feb 25;164(5):974-84. doi: 10.1016/j.cell.2016.02.008.  PMID: 26919432

FEBRUARY 11, 2016  New publication  Redefining the Translational Status of 80S Monosomes. Heyer EE, Moore MJ.  Cell. 2016 Feb 11;164(4):757-69. doi: 10.1016/j.cell.2016.01.003. PMID: 26871635

FEBRUARY 11, 2016  Yan Zhang, PhD, a postdoctoral fellow in Dr. Erik Sontheimer’s lab has received a NIH Pathway to Independence K99/R00 Award from NIGMS. The project title is “The alternative roles of Neisseria CRISPR-Cas9 in endogenous gene regulation and bacterial physiology”. This award is for a period of five years consisting of two phases, with two years being funded for mentored research and three years of funding as an independent investigator.

FEBRUARY 4, 2016   RNA Therapeutics Institute established as an academic department; Phillip D. Zamore, PhD appointed as founding chair  UMass Medical School announced today that the RNA Therapeutics Institute (RTI) has been accorded academic department status under the leadership of Phillip D. Zamore, PhD, Howard Hughes Medical Institute Investigator, the Gretchen Stone Cook Chair in Biomedical Sciences and professor of biochemistry & molecular pharmacology, appointed as its founding chair. The RTI was created in June 2009 with four co-directors “charged with the responsibility of creating an innovative, nimble and scientifically rigorous home for RNA research dedicated to human impact”.  Chancellor Michael F. Collins stated:  “They have delivered on this promise, so much so that the maturation of the RTI invites this next step". For more, please click here.

FEBRUARY 1, 2016  David Grunwald, PhD, Assistant Professor of RTI, was featured in Biotechniques for his lab's study on what happens when a single mRNA moves from the cell nucleus to the cytoplasm. For details, please click here.

FEBRUARY 1, 2016  New publication  Wen Xue, PhD, Assistant Professor of RTI, is corresponding author of the first study to provide proof that the CRISPR/Cas9 gene editing system can be administered in a therapeutically applicable formulation to repair genes in live, adult animals.   “Therapeutic Genome Editing By Combined Viral and Non-viral Delivery of Crispr System Components in Vivo.”  Yin, H, Song, C-Q, Dorkin, JR, Zhu, LJ, Li, Y, Wu, Q, Park, A, Yang, J, Suresh, S, Bizhanova, A, Gupta, A, Bolukbasi, MF, Walsh, S, Bogorad, RL, Gao, G, Weng, Z, Dong, Y, Koteliansky, V, Wolfe, SA, Langer, R, Xue, W, Anderson, DG.  Nat Biotechnol. 2016 Mar;34(3):328-33. doi: 10.1038/nbt.3471. Epub 2016 Feb 1. PMID: 26829318  For more detail about the news, please click here.

JANUARY 22, 2016 New publication  Biogenesis and function of tRNA fragments during sperm maturation and fertilization in mammals.  Sharma U, Conine CC, Shea JM, Boskovic A, Derr AG, Bing XY, Belleannee C, Kucukural A, Serra RW, Sun F, Song L, Carone BR, Ricci EP, Li XZ, Fauquier L, Moore MJ, Sullivan R, Mello CC, Garber M, Rando OJ.  Science. 2016 Jan 22;351(6271):391-6. doi: 10.1126/science.aad6780. Epub 2015 Dec 31.PMID: 26721685

JANUARY 1, 2016  Marie Didiot, PhD, received fellowship award.  Dr. Didiot, a postdoctoral fellow in the  Khvorova lab, received a competitive fellowship award from the Huntington’s Disease Society of America (HDSA).  She will receive $150,000 over two years for her project titled “Study of the Impact of CAG Repeat Expansion on Htt mRNA Cellular Localization”.

DECEMBER 21, 2015 New publication  In vivo single-particle imaging of nuclear mRNA export in budding yeast demonstrates an essential role for Mex67p.  Smith C, Lari A, Derrer CP, Ouwehand A, Rossouw A, Huisman M, Dange T, Hopman M, Joseph A, Zenklusen D, Weis K, Grunwald D, Montpetit B. J Cell Biol. 2015 Dec 21;211(6):1121-30. doi: 10.1083/jcb.201503135. PMID: 26694837

DECEMBER 1, 2015 New publication  Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain.  Alterman JF, Hall LM, Coles AH, Hassler MR, Didiot MC, Chase K, Abraham J, Sottosanti E, Johnson E, Sapp E, Osborn MF, Difiglia M, Aronin N, Khvorova A. Mol Ther Nucleic Acids. 2015 Dec 1;4:e266. doi: 10.1038/mtna.2015.38.PMID: 26623938

NOVEMBER 5, 2015  Wen Xue, PhD, Assistant Professor of RTI, won The Lung Cancer Research Foundation (LCRF) Scientific Merit Award, in addition to 2015 LCRF grant.  The Scientific Merit Award acknowledges the investigator whose proposal was selected for outstanding overall merit by the Foundation's Scientific Advisory Board. Dr. Xue received the accolade for his research targeting small RNA-mediated KRAS inhibition in lung cancer. For more details, click here.

NOVEMBER 5, 2015 New publicatioProbability-based particle detection that enables threshold-free and robust in vivo single-molecule tracking.  Smith CS, Stallinga S, Lidke KA, Rieger B, Grunwald D. Mol Biol Cell. 2015 Nov 5;26(22):4057-62. doi: 10.1091/mbc.E15-06-0448. Epub 2015 Sep 30.  PMID: 26424801

NOVEMBER 3, 2015 New publication  Ribosome Structure Reveals Preservation of Active Sites in the Presence of a P-Site Wobble Mismatch. Svidritskiy E, Korostelev AA. Structure. 2015 Nov 3;23(11):2155-61. doi: 10.1016/j.str.2015.08.011. Epub 2015 Sep 24.PMID: 26412335

NOVEMBER 1, 2015  Phillip D. ZamorePhD, co-director of the RNA Therapeutics Institute, is on The World’s
Most Influential Scientific Minds 2014 by Reuters.
Phil is one of the researchers who have produced work that is most frequently acknowledged by peers in the field of Molecular Biology & Genetics.  These highly cited researchers were determined by analyzing at citation data over the last 11 years to identify those who published the highestimpact work (2002—2012 and 2012—2013). These individuals are influencing the future direction of their fields, and of the world.  For details, please click here.  For a copy of the report, click here.

OCTOBER 23, 2015  Socheata Ly is selected for Initiative to Maximize Student Development (IMSD) program.  Soki, a GSBS graduate student in the Khvorova lab is selected by Drs. Brian Lewis and Kate Lapane to paticipate in the IMSD research education grant awarded to UMMS. A primary goal of the IMSD program is to support the development and implementation of strategies that increase the number of underrepresented students that attain PhDs in the biomedical sciences.

OCTOBER 15, 2015  New publication from the Sontheimer lab. DNase H Activity of Neisseria meningitidis Cas9.   Zhang Y, Rajan R, Seifert HS, Mondragón A, Sontheimer EJ. Mol Cell. 2015 Oct 15;60(2):242-55. doi:10.1016/j.molcel.2015.09.020. PMID: 26474066

OCTOBER 15, 2015  New publication from the Khvorova lab.  Guanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAs. Osborn MF, Alterman JF, Nikan M, Cao H, Didiot MC, Hassler MR, Coles AH, Khvorova A. Nucleic Acids Res. 2015 Oct 15;43(18):8664-72. doi: 10.1093/nar/gkv942. Epub 2015 Sep 22. PMID: 26400165

OCTOBER 8, 2015  Wen Xue, PhD, Assistant Professor of RNA Therapeutics Institute, won 2015 Lung Cancer Research Foundation (LCRF)  philanthropic grant  for his project titled " Small RNA-mediated KRAS inhibition in lung cancer". The goal of this project is to investigate mechanisms of tumor regression and relapse upon KRAS inhibition in vivo use small RNA tools. For details, click here.

OCTOBER 7, 2015  New publication  Primary processing of CRISPR RNA by the endonuclease Cas6 in Staphylococcus epidermidis. Wakefield N, Rajan R, Sontheimer EJ. FEBS Lett. 2015 Oct 7;589(20 Pt B):3197-204. doi: 10.1016/j.febslet.2015.09.005. Epub 2015 Sep 10. PMID: 26364721

OCTOBER 1, 2015  New publication  Influenza A virus preferentially snatches noncoding RNA caps.  Gu W, Gallagher GR, Dai W, Liu P, Li R, Trombly MI, Gammon DB, Mello CC, Wang JP, Finberg RW. RNA. 2015 Oct 1. [Epub ahead of print]  PMID: 26428694

SEPTEMBER 30, 2015  David Grunwald, PhD, Assistant Professor of RNA Therapeutics Institute won U01 three year Award from the National Institute of Biomedical Imaging and Bioengineering. His project titled  “Visualizing local and global chromatin architecture, and gene expression in the individual cell by structural (SUSHI) and temporal (3D-SMRT) single molecule imaging” is part of a larger 4D Nucleome initiative with NIH. For more, please click here.

SEPTEMBER 30, 2015  New publication   Tailor: a computational framework for detecting non-templated tailing of small silencing RNAs. Chou MT, Han BW, Hsiao CP, Zamore PD, Weng Z, Hung JH. Nucleic Acids Res. 2015 Sep 30;43(17):e109. doi: 10.1093/nar/gkv537. Epub 2015 May 24. PMID: 26007652

SEPTEMBER 29, 2015  New publication   Safety of Striatal Infusion of siRNA in a Transgenic Huntington's Disease Mouse Model.  Johnson E, Chase K, McGowan S, Mondo E, Pfister E, Mick E, Friedline RH, Kim JK, Sapp E, DiFiglia M, Aronin N. J Huntingtons Dis. 2015;4(3):219-29. doi: 10.3233/JHD-150163.PMID: 26444021

SEPTEMBER 10, 2015  New publication  Primary processing of CRISPR RNA by the endonuclease Cas6 in Staphylococcus epidermidis. Wakefield N, Rajan R, Sontheimer EJ.  FEBS Lett. 2015 Sep 10. pii: S0014-5793(15)00822-4. doi: 10.1016/j.febslet.2015.09.005. [Epub ahead of print] PMID: 26364721

SEPTEMBER 8, 2015  New publication  Caenorhabditis elegans ALG-1 antimorphic mutations uncover functions for Argonaute in microRNA guide strand selection and passenger strand disposal. Zinovyeva AY, Veksler-Lublinsky I, Vashisht AA, Wohlschlegel JA, Ambros VR.  Proc Natl Acad Sci U S A. 2015 Sep 8. pii: 201506576. [Epub ahead of print] PMID: 26351692

SEPTEMBER 3, 2015  New publication  Slicing and Binding by Ago3 or Aub Trigger Piwi-Bound piRNA Production by Distinct Mechanism Wang W, Han BW, Tipping C, Ge DT, Zhang Z, Weng Z, Zamore PD.  Mol Cell. 2015 Sep 3;59(5):819-30. doi: 10.1016/j.molcel.2015.08.007. PMID: 26340424


AUGUST 13, 2015  Gina V. Caldas, PhD, named 2015 Damon Runyon Fellow.  Dr. Gina Caldas, a postdoctoral fellow in the Mello lab, is among the 16 new 2015 Damon Runyon Fellows. The recipients of this prestigious, four-year award are outstanding postdoctoral scientists conducting basic and translational cancer research in the laboratories of leading senior investigators across the country. Gina was also a 2015 Pew Latin American Fellow in Biomedical Sciences by The Pew Charitable Trusts.  Gina is working on identifying new aspects of cell division control that can be targeted for cancer therapy using C. elegans as a model system. For more details, click here.

Jon Watts

AUGUST 1, 2015  Jonathan K Watts PhD joins RTI.  Dr. Watts joins us from University of Southhampton of UK.  His lab will focus on developing molecules that mimic natural nucleic acids but with a number of distinct properties, like reversibly and repeatedly respond to light, or selective targeting of nucleic acid species such as nuclear noncoding RNA and chromosomal DNA. His lab will also work on the development of tools and methods to study cellular nucleic acids. 

JULY 15, 2015  New publication  Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses.  Wang D, Mou H, Li S, Li Y, Hough S, Tran K, Li J, Yin H, Anderson DG, Sontheimer EJ, Weng Z, Gao G, Xue W. Hum Gene Ther. 2015 Jul;26(7):432-42. doi: 10.1089/hum.2015.087. PMID: 26086867


JULY 13, 2015  Phillip D. ZamorePhD, co-director of the RNA Therapeutics Institute, received prestigious MERIT awards from NIH.  Phil and another researcher have received Method to Extend Research in Time (MERIT) awards from the National Institutes of Health for their ability to demonstrate “superior competence and productivity during their previous endeavors” and for being “likely to continue to perform in an outstanding manner in the future,” according to the NIH. This is the second MERIT award for Dr. Phil Zamore.

JULY 6, 2015  New publication POS-1 Promotes Endo-mesoderm Development by Inhibiting the Cytoplasmic Polyadenylation of neg-1 mRNA.  Elewa A, Shirayama M, Kaymak E, Harrison PF, Powell DR, Du Z, Chute CD, Woolf H, Yi D, Ishidate T, Srinivasan J, Bao Z, Beilharz TH, Ryder SP, Mello CC.  Dev Cell. 2015 Jul 6;34(1):108-18. doi: 10.1016/j.devcel.2015.05.024. Epub 2015 Jun 18.  PMID: 26096734

JULY 2, 2015  New publication  Single-Molecule Imaging Reveals that Argonaute Reshapes the Binding Properties of Its Nucleic Acid Guides. Salomon WE, Jolly SM, Moore MJ, Zamore PD, Serebrov V.  Cell. 2015 Jul 2;162(1):84-95. doi: 10.1016/j.cell.2015.06.029.  PMID: 26140592

JUNE 1, 2015  New publication  A versatile reporter system for CRISPR-mediated chromosomal rearrangements.  Li Y, Park A, Mou H, Colpan C, Bizhanova A, Akama-Garren E, Joshi N, Hendrickson EA, Feldser D, Yin H, Anderson DG, Jacks T, Weng Z, Xue W.  Genome Biol. 2015 May 28;16(1):111. [Epub ahead of print] PMID: 26018130


MAY 29, 2015  Bo Han won Chacellor's Award. Bo Han, a graduating PhD student in the Zamore lab was recognized for his academic achievement with the UMMS Chancellor's Award. He worked on "Using experimental and computational strategies to understand the biogenesis of microRNAs and piRNAs" and recently published on Science. For details, click here.


MAY 25, 2015  New publication  Nuclear accessibility of β-actin mRNA is measured by 3D single-molecule real-time tracking. Smith CS, Preibisch S, Joseph A, Abrahamsson S, Rieger B, Myers E, Singer RH, Grunwald D. J Cell Biol. 2015 May 25;209(4):609-19. doi: 10.1083/jcb.201411032. PMID: 26008747

MAY 15, 2015  New publication  Noncoding RNA. piRNA-guided transposon cleavage initiates Zucchini-dependent, phased piRNA production.  Han BW, Wang W, Li C, Weng Z, Zamore PD. Science. 2015 May 15;348(6236):817-21. doi: 10.1126/science.aaa1264. PMID: 25977554

MAY 5, 2015  New publication  Caenorhabditis elegans microRNAs of the let-7 family act in innate immune response circuits and confer robust developmental timing against pathogen stress. Ren Z, Ambros VR.  Proc Natl Acad Sci U S A. 2015 May 5;112(18):E2366-75. doi:10.1073/pnas.1422858112. Epub 2015 Apr 20. PMID: 25897023

APRIL 28, 2015  Craig C. Mello, PhD, Nobel Laureate and co-director of the RNA Therapeutics Institute, talks about CRISPER on WBUR’s On Point Radio. For details, click here. 

APRIL 23, 2015  Wen Tang, PhD, postdoctoral fellow in Dr. Craig Mello’s lab, is a 2015 recipient of Hope Funds for Cancer Research Postdocotral Fellowship.  This will be a three-year fellowship to support Dr. Wen Tang’s research.  The Hope Funds for Cancer Research is offering fellowships to postdoctoral scientists who propose to work on highly innovative research projects that challenge the traditional paradigms of understanding the causes, mechanisms, progression, disease markers or risk factors of the most difficult-to-treat cancers.

APRIL 14, 2015  Wen Xue, PhD, Assistant Professor of RNA Therapeutics Institute won 2015 Worcester Foundation Grants for his project titled "Liver cancer gene discovery and correction using CRISPR-mediated genome editing".  Worcester Foundation grants are to be awarded to investigators at the Medical School in support of promising and innovative research projects.

APRIL 14, 2015 New publication  Assessing long-distance RNA sequence connectivity via RNA-templated DNA-DNA ligation. Roy CK, Olson S, Graveley BR, Zamore PD, Moore MJ. Elife. 2015 Apr 13;4. doi: 10.7554/eLife.03700. [Epub ahead of print] PMID: 25866926

MARCH 25, 2015  Anastasia Khvorova, PhD, Professor of RNA Therapeutics Institute, received NIH Shared Instrumentation Grant (S10).  Anastasia’s project titled ”Mid-Scale RNA Synthesis, Purification and Quality Control System” will be funded for over half million dollars to help purchasing a series of equipment for the chemistry core.

JANUARY 29, 2015 New publication A ribonuclease coordinates siRNA amplification and mRNA cleavage during RNAi. Tsai HY, Chen CC, Conte D Jr, Moresco JJ, Chaves DA, Mitani S, Yates JR 3rd, Tsai MD, Mello CC. Cell. 2015 Jan 29;160(3):407-19. doi: 10.1016/j.cell.2015.01.010.
PMID: 25635455

JANUARY 20, 2015 New publication Control of stem cell self-renewal and differentiation by the heterochronic genes and the cellular asymmetry machinery in Caenorhabditis elegans. Harandi OF, Ambros VR. Proc Natl Acad Sci U S A. 2015 Jan 20;112(3):E287-96. doi: 10.1073/pnas.1422852112.  PMID: 25561544

JANUARY 12, 2015  Dr. Craig C. Mello, Nobel Laureate and co-director of the RNA Therapeutics Institute will be in the You're the Expert comedy show Thursday, January 15th. This show, presented by the WBUR iLab, is trying to bridge the worlds of comedy and academia, and let people understand the importance of the expert's field of study while having fun. For detail, please click here. 

JANUARY 7, 2015  Molecular Cell cover features new study from Zamore lab.  The study provides new insights into the biochemical mechanisms governing how piRNAs target select genetic elements for silencing.  The Initial Uridine of Primary piRNAs Does Not Create the Tenth Adenine that Is the Hallmark of Secondary piRNAs. Wang W, Yoshikawa M, Han BW, Izumi N, Tomari Y, Weng Z, Zamore PD.  Mol Cell. 2014 Dec 4;56(5):708-16. doi: 10.1016/j.molcel.2014.10.016. Epub 2014 Nov 20. PMID: 25453759 [PubMed - in process]

JANUARY 7, 2015  Jeremy Luban, MD, RTI affiliated faculty,  receives National Human Genome Research Institute grant to develop model for predicting gene expression in human dendritic cells.  Dr. Luban and Manuel Garber, PhD, another faculty at UMMS, will be principal investigators on a three-year, $6.1 million grant to develop a model for predicting whether a given gene will be turned on or off under specific conditions. For details, click here.

DECEMBER 16, 2014  Phillip D. Zamore, PhD,  co-director of the RNA Therapeutics Institute, was elected to NAI Fellow status.  Election to National Academy of Inventors Fellow status is a high profession distinction accorded to academic inventors who demonstrated a highly prolific spirit of innovation in creating or facilitating outstanding inventions that have made a tangible impact on quality of life, economic development, and the welfare of society. For details, please click here.

DECEMBER 12, 2014 New publication  An Optimized Kit-free Method for Making Strand-specific Deep Sequencing Libraries From RNA Fragments. Heyer EE, Ozadam H, Ricci EP, Cenik C, and Moore MJ. Nucleic Acids Res. published 12 December 2014, 10.1093/nar/gku1235


NOVEMBER 12, 2014  Victor Ambros, PhD,  co-director of the RNA Therapeutics Institute, won 2015 Breakthrough Prize in Life Sciences.  Victor Ambros and his long-time collaborator Gary Ruvkun, Massachusetts General Hospital and Harvard Medical School, have each been awarded a 2015 Breakthrough Prize in Life Sciences for their co-discovery of a class of tiny RNA molecules that inhibit translation or destabilize complementary mRNA targets. Each received a $3 million award. The Breakthrough Prize in Life Sciences honors transformative advances toward understanding living systems and extending human life.  For details, please click here.

OCTOBER 29, 2014  Craig Mello, PhD, co-director of the RNA Therapeutics Institute, talks about the need for the region's life sciences industry to work together in Worcester Telegram & Gazette. For details, click here.

OCTOBER 17, 2014  New publication piPipes: a set of pipelines for piRNA and transposon analysis via small RNA-seq, RNA-seq, degradome- and CAGE-seq, ChIP-seq and genomic DNA sequencing.   Han, BW, Wang, W, Zamore, PD, and Weng, Z.  Bioinformatics. published 17 October 2014, 10.1093/bioinformatics/btu647

OCTOBER 16, 2014, Juahdi Monbo won 2014 ASCB MAC Travel Award. Juahdi Monbo, a graduate student in David Grunwald's lab, has been selected to receive a MAC Travel Award to attend the ASCB/IFCB Meeting in Philadelphia, PA, December 6-10. For details, please click here.

SEPTEMBER 19, 2014  Phillip D. Zamore, PhD, co-director of the RNA Therapeutics Institute, was recently featured on Nature Biotechnology.  The journal interviewed him for his experience on starting up Alnylam and Voyager, two biotech companies using RNAi and gene therapy, and his insight on the commercialization of RNAi.  For details, click here.

SEPTEMBER 2, 2014  New publications  Small RNA Combination Therapy for Lung Cancer.  Xue W, Dahlman JE, Tammela T, Khan OF, Sood S, Dave A, Cai W, Chirino LM, Yang GR, Bronson R, Crowley DG, Sahay G, Schroeder A, Langer R, Anderson DG, Jacks T.    Proc Natl Acad Sci U S A. 2014 Aug 26;111(34):E3553-61. doi: 10.1073/pnas.1412686111. Epub 2014 Aug 11.   CRISPR-mediated direct mutation of cancer genes in the mouse liver. Xue W, Chen S, Yin H, Tammela T, Papagiannakopoulos T, Joshi NS, Cai W, Yang G, Bronson R, Crowley DG, Zhang F, Anderson DG, Sharp PA, Jacks T. Nature. 2014 Oct 16;514(7522):380-4. doi: 10.1038/nature13589. Epub 2014 Aug 6. PMID:25119044

SEPTEMBER 1, 2014  New publication  Circulating microRNA profiles in human patients with acetaminophen hepatotoxicity or ischemic hepatitisWard J, Kanchagar C, Veksler-Lublinsky I, Lee RC, McGill MR, Jaeschke H, Curry SC, Ambros VR.  Proc Natl Acad Sci U S A. 2014 Aug 19;111(33):12169-74. doi: 10.1073/pnas.1412608111. Epub 2014 Aug 4.

SEPTEMBER 1, 2014  Wen Xue PhD joins RTI  Dr. Wen Xue will be joining us here in the RTI as an Assistant Professor. Wen earned his Ph.D. with Scott Lowe at the Cold Spring Harbor Laboratories and did post-doctoral studies with Tyler Jacks at MIT.  Wen studies the role of microRNAs in cancer using mouser models.  He has just received an R00 award from the NIH! The R00 program supports young investigators as they transition from post-doc to independent faculty.

AUGUST 26, 2014  Dr. Heng-Chi Lee, a post-doc in the Mello lab, has received a K99 award from the NIH. The K99 grant is the post-doctoral component of the K99/R00 program and supports the post-doctoral studies of talented young scientists as they work toward independence.

AUGUST 25, 2014  Dr. Anastasia Khvorova received a four-year award from NIH/NIGMS to study the “Inter and intra-cellular trafficking pathways for natural and therapeutic RNAs”, with co-investigators Victor Ambros, Sylvia Corvera, and Kevin Fogarty.  The project will use a combination of RNA medicinal chemistry, TIRF microscopy and quantitative mass-spectrometry to map uptake pathways for different classes of therapeutic oligonucleotides, identify and characterize mechanism of small regulatory RNS cell and transfer, and deliver the conceptual framework necessary to generate novel RNS chemistries that exploit natural uptake mechanisms leading to transformational improvements in oligonucleotide therapeutics field.

AUGUST 15, 2014 New publication  SPO24 is a transcriptionally dynamic, small ORF-encoding locus required for efficient sporulation in Saccharomyces cerevisiae. Hurtado S, Kim Guisbert KS, Sontheimer EJ. PLoS One. 2014 Aug 15;9(8):e105058. doi: 10.1371/journal.pone.0105058. eCollection 2014. PMID: 25127041

JULY 31, 2014  Erik Sontheimer PhD joins RTI   Dr. Erik Sontheimer joins us from Northwestern University.  The Sontheimer laboratory focuses on the roles of RNA molecules in gene expression, with an emphasis on genetic interference pathways.  Using biochemical approaches to characterize the function of the RNAi machinery, Dr. Sontheimer's research has yielded fundamental insights into the biochemical mechanism of RNAi and the pathway by which the RNAi machinery assembles on a double-stranded RNA.  His research has recently expanded into a distinct interference pathway specified by CRISPR loci that limits the spread of antibiotic resistance in pathogenic bacteria.

JULY 23, 2014  New publication  An efficient and sensitive method for preparing cDNA libraries from scarce biological samples.  Catherine H. Sterling, Isana Veksler-Lublinksy, and Victor Ambros  Nucleic Acids Res. published 23 July 2014, 10.1093/nar/gku637  [Abstract]

JULY 2, 2014  Two RNA Therapeutics Insititute faculties won 2014 UMass Life Science Moment Fund (LSMF) in one day.  Anastasia Khvorova, Ph.D., with project “Ex vivo RNAi-based therapy for modulating T cell response as a novel treatment for Graft-vs-Host Disease”,  and Neil Aronin, Ph.D., in conjunction with Dr. Michael Brodsky (Program in Gene Function and Expression) and Dr. Gregory Tew (UMass Amherst), with project “CRISPR- Cas9-derived Nucleases and PTDMs for Huntingtin Disease Treatment”, were selected for funding by the 2014 UMass Life Science Moment Fund (LSMF).  The two projects will bring together investigators from both the Worcester and Amherst campuses.

JUNE 27, 2014  Craig C. Mello, PhD, Nobel Laureate and co-director of the RNA Therapeutics Institute, talks to National Public Radio about the exciting future of CRISPR. He believes medical applications using the genome-engineering tool known as CRISPR are possible in the near future, he said in an interview with National Public Radio science reporter Joe Palca. For details, click here.

JUNE 25, 2014 New publication   A single vertebrate DNA virus protein disarms invertebrate immunity to RNA virus infection.  Don B Gammon, Sophie Duraffour, Daniel K Rozelle, Heidi Hehnly, Rita Sharma, Michael E Sparks, Cara C West, Ying Chen, James J Moresco, Graciela Andrei, John C Connor, Darryl Conte, Dawn E Gundersen-Rindal, William L Marshall, John Yates, Neal Silverman, and Craig C Mello   eLife Sci. published 25 June 2014, 10.7554/eLife.02910 

JUNE 17, 2014  Victor R. Ambros, PhD, the Silverman Chair In Natural Sciences and co-director of the RNA Therapeutics Institute, has been awarded the 2014 Gruber Genetics Prize.  Dr. Ambros, along with long time collaborator Gary Ruvkun, PhD, professor of genetics at Massachusetts General Hospital and Harvard Medical School, and David Baulcombe, PhD, professor of botany at the University of Cambridge, received the prize for their pioneering discoveries of the existence and function of microRNAs and small interfering RNAs, molecules that are now known to play a critical role in gene expression. Please join us in congratulating him on this tremendous honor!  For details, click here.

JUNE 11, 2014  New publication   Structural mechanism is revealed of how the messenger RNA of Taura syndrome virus hijacks host ribosomes using structured RNA, called internal ribosome entry site (IRES).  Koh, C. S., Brilot, A. F., Grigorieff, N., & Korostelev, A. A. (2014). Taura syndrome virus IRES initiates translation by binding its tRNA-mRNA–like structural element in the ribosomal decoding center. Proceedings of the National Academy of Sciences. doi:10.1073/pnas.1406335111

JUNE 10, 2014 David Grunwald, PhD, Assistant Professor of RNA Therapeutics Institute won Faculty Scholar Award (FSA).  The Faculty Scholar Award was created by the UMMS Women’s Faculty Committee and the Women’s Leadership Work Group, and is funded jointly by UMMS and UMMHC to assist faculty during a finite period of increased family care responsibilities.  Dr. Grunwald was selected as a recipient of the award for June 2014 due to the innovation and technical challenges of his project across many disciplines.

JUNE 4, 2014 Anastasia Khvorova, PhD, Professor of RNA Therapeutics Institute, is featured in Exiqon Application stories.  Khvorova lab focuses on the development of novel oligonucleotide-based therapeutics, as well ways to improve delivery and tissue distribution for neurodegenerative diseases.  Her lab has been using LNA™ GapmeRs to successfully silence two different genes, which are valid therapeutic targets for neurodegenerative diseases, both in vitro and in vivo mouse and rat animal models.
 To read about her exciting research, click here.

MAY 30, 2014 New publication  A Co-CRISPR Strategy for Efficient Genome Editing in Caenorhabditis elegans.  Heesun Kim, Takao Ishidate, Krishna S. Ghanta, Meetu Seth, Darryl Conte, Jr., Masaki Shirayama, and Craig C. Mello  Genetics. published 30 May 2014, 10.1534/genetics.114.166389

APRIL 8, 2014 New publication   The Vasa Homolog RDE-12 Engages Target mRNA and Multiple Argonaute Proteins to Promote RNAi in C. elegans.  Masaki Shirayama, William Stanney III, Weifeng Gu, Meetu Seth, Craig C. Mello  Curr Biol. 2014 Mar 26. pii: S0960-9822(14)00267-X. doi: 10.1016/j.cub.2014.03.008. [Epub ahead of print].

APRIL 7, 2014  Gang Han, PhD, RTI affiliated faculty, was awarded 2014 Young Investigator's Grant from Human Frontier Science Program. The HFSP supports novel, innovative and interdisciplinary basic research focused on the complex mechanisms of living organisms. The grant will support Han lab’s project on “Wireless optogenetic interrogation of non-image forming photoreceptor function by Nano-antennae” for three years and about $900,000 in total. For details, click here.

MARCH 14,2014  Melissa Moore PhD and Anastasia Khvorova PhD are among the founders of MassTERi. The Massachusetts Therapeutic and Entrepreneurial Realization Initiative (MassTERi), is a faculty-led initiative that will build upon existing efforts at the UMass Center for Clinical and Translational Science (UMCCTS) and other university organizations to support scientists who are interested in advancing development of small molecule drugs and in commercializing their innovation. More...

MARCH 14, 2014  Colin Conine, PhD candidate in Dr. Craig Mello’s lab, has been awarded the 2014 Harold M. Weintraub Graduate Student Award.  This prestigious award is sponsored by the Basic Sciences Division of Fred Hutchinson Cancer Research Center.  Nominations were solicited internationally; the winners were selected on the basis of the quality, originality and significance of their work. More…

FEBRUARY 25, 2014  Jeremy Luban, MD, David L. Freelander Memorial Professor in HIV/AIDS Research and RNA Therapeutics Institute affiliated faculty, has been elected fellow of the American Academy of Microbiology for his scientific achievement and original contributions to the field of microbiology.  More...

FEBRUARY 18, 2014  Phillip Zamore, PhD, is co-principal investigator of a Broadening Experience in Scientific Training (BEST) grant from the NIH. UMass is the first group of 10 graduate schools nationwide to be awarded. With the grant, Zamore and Cynthia Fuhrmann, PhD, assistant dean of professional and career development for the GSBS, will implement “An Integrated Curriculum and Community-Based Approach to Career Development” which will guide students and postdocs to create and implement a strategic plan for building the skills and experience needed to reach their career objectives. More

FEBRUARY 12, 2014  Phillip D. Zamore, PhD, co-director of the RNA Therapeutics Institute, and Guangping Gao, PhD, director of the Gene Therapy Center and RTI affiliated faculty, are among the four world leaders in the fields of AAV gene therapy, RNA biology and neuroscience to launch Voyager Therapeutics today. Voyager Therapeutics is a gene therapy company to develop life-changing treatments for fatal and debilitating diseases of the central nervous system (CNS). More...

FEBRUARY  6, 2014  Kelly Limoncelli received Ruth L. Kirschstein National Research Service Awards (NRSA) for Individual Predoctoral Fellows from NIH.  Kelly Limoncell, a graduate student in Melissa Moore’s lab, will receive up to 4 years of support, which covers stipend, tuition, fees, supplies, and scientific meeting expenses.  The purpose of this training fellowship is to provide support for promising doctoral candidates who will be performing dissertation research and training in scientific health-related fields relevant to the missions of the participating NIH Institutes and Centers (ICs) during the tenure of the award.  Her project title is “Investigating the functional roles of RPS3 in RNA and DNA damage”.  For details, click here.

FEBRUARY 3, 2014 New publication  Inorganic phosphate blocks binding of pre-miRNA to Dicer-2 via its PAZ domain  Ryuya Fukunaga, Cansu Colpan, Bo W Han, and Phillip D Zamore  EMBO J. published 31 January 2014, 10.1002/embj.201387176  [Abstract].

JANUARY 21, 2014 Victor R. Ambros is awarded prestigious Wolf Prize in Medicine for the discovery of the micro- RNA molecules that play a key role in controlling gene expression in natural processes and disease development. For details, click here.

JANUARY 17, 2014 New publication Endogenous U2·U5·U6 snRNA complexes in S. pombe are intron lariat spliceosomes Weijun Chen, Hennady P. Shulha, Ami Ashar-Patel, Jing Yan, Karin M. Green, Charles C. Query, Nick Rhind, Zhiping Weng, and Melissa J. Moore  RNA. published 17 January 2014, 10.1261/rna.040980.113  [Abstract].

DECEMBER 10, 2013 Rene Maehr, PhD, became an RTI affiliated faculty. His group works on pluripotent stem cell-based models of immune deficiencies and autoimmunity and has an exciting publication recently.  Kearns NA, Genga RM, Enuameh MS, Garber M, Wolfe SA, Maehr R.Cas9 effector-mediated regulation of transcription and differentiation in human pluripotent stem cells. Development. 2014 Jan;141(1):219-223.

OCTOBER 28, 2013  Gang Han, PhD, awarded  EUREKA R01.  With a perfect impact score (10) Han lab received EUREKA (Exceptional, Unconventional Research Enabling Knowledge Acceleration) RO1 grant from the National Institute of Health to develop light activated nanoparticles to form the basis of a new wireless “optogenetic” tool that promises to help map and decode previously inaccessible neural circuitry deep in the brain. For details, click here.

OCTOBER 8, 2013 Joerg Braun won Karl Lohmann Award from Germany.  Dr. Joerg E. Braun, a postdoctoral fellow in Dr. Melissa Moore’s laboratory, is awarded the 2013 Karl Lohmann Award from The Society for Biochemistry and Molecular Biology (GBM) for his doctoral research in Frankfurt am Main, Germany.  The Karl Lohmann Award is conferred every second year to reward young researchers under 35 years of age. For details, click here.

OCTOBER 4, 2013 Phillip Zamore, codirector of the RNA Therapeutics Institute shares his experience on childcare and his strong belief on work family balance in The Boston Globe.  For full articleclick here.

SEPTEMBER 16, 2013 Victor R. Ambros is awarded Keio Medical Science Prize. The 18th Keio Medical Science Prize has been awarded to Victor R. Ambros, PhD, the Silverman Chair in Natural Science.  Keio University in Japan annually awards the Keio Medical Science Prize “to recognize researchers who have made an outstanding contribution to the field of medicine or life sciences.”  For more details, click here.

SEPTEMBER 3, 2013  Neil Aronin, MD,  awarded grant from collaborative NIH Extracellular RNA Communication program.  He will explore the Clinincal Utility of Extracellular RNA for Therapy Development focusing on Huntington's disease. For more details about his project and other UMass awardees, click here.  

AUGUST 19, 2013 Two RTI graduate students awarded prestigious HHMI awards.  Haleen Saini, who is a 3rd year Neuroscience Program student in Dr. Melissa Moore's laboratory and, Meetu Seth, a 4th year Interdisciplinary Graduate Program student in Dr. Craig Mello's laboratory were among only 42 awardees across the nation for HHMI 2013 International Student Research Fellowships.  Both students will receive up to three years of stipend, fees and research supports.

AUGUST 8, 2013  New publication  ASPeak: an abundance sensitive peak detection algorithm for RIP-Seq Alper Kucukural, Hakan Özadam, Guramrit Singh, Melissa J. Moore, and Can Cenik   Bioinformatics. published 8 August 2013, 10.1093/bioinformatics/btt428  [Abstract].

AUGUST 1, 2013  New publication  Long Noncoding RNA Mediates Both Activation and Repression of Immune Response Genes  Susan Carpenter, Maninjay Atianand, Daniel Aiello, Emiliano P. Ricci, Pallavi Gandhi, Lisa L. Hall, Meg Byron, Brian Monks, Meabh Henry-Bezy, Jeanne B. Lawrence, Luke A. J. O’Neill, Melissa J. Moore, Daniel R. Caffrey, and Katherine A. Fitzgerald   Science. published 1 August 2013, 10.1126/science.1240925 [Abstract].

AUGUST 1, 2013  Andrei Korostelev, PhD, awarded R01.  The Korostelev lab received a five-year R01 award from the NIH to study "Structural bases for cellular stress responses mediated by stalled translation".  The project aims at understanding the molecular mechanisms that result in high pathogenicity of bacteria; elucidation of these mechanisms may lead to development of novel antibiotics.

JULY 3,  2013  New publication from Andrei Korostelev's lab in Proceedings of the National Academy of Sciences.  The article describes a novel mode of action of an inhibitor of translation. Korostelev lab, in collaboration with the Ermolenko lab (University of Rochester), has found that blasticidin S strongly inhibits translation termination by trapping a distorted conformation of tRNA on the ribosome. This finding may serve as a platform for the development of therapeutics that specifically suppress translation termination in human diseases associated with premature termination codons.  Svidritskiy E., Ling C., Ermolenko D. N. and Korostelev A. A. "Blasticidin S inhibits translation by trapping deformed tRNA on the ribosome". Proc Natl Acad Sci U S A. July 2013.

JUNE 6, 2013 Joerg Braun awarded Otto Hahn Medal. Joerg E.Braun, a postdoctoral associate in Melissa Moore's lab, was awarded Otto Hahn Medal for his outstanding PhD work. The Otto Hahn Medals were awarded at the Annual Meeting of the Max Planck Society in Potsdam, Germany, on June 5 and 6.  Since 1978, a few young and especially highly gifted researchers are awarded the Otto Hahn Medal each year – for their outstanding PhD work and to motivate them for a future scientific career. More...

JUNE 1, 2013  Joerg Braun awarded Human Frontier Science Program Organization Fellowship.  Joerg Braun, a postdoctoral associate in Melissa Moore’s lab, was awarded a fellowship for his project “Single molecule analysis of the mammalian alternative pre-mRNA splicing”. This three year award will support Dr. Braun’s work dissecting the dynamics of human spliceosome assembly.

MAY 20, 2013  Phillip Zamore and Keith Boundy receive Umass Medical School Office of Globe Health Award.  Phillip Zamore, PhD and Keith Bounty, Resident Physician and PhD Student, receive UMass Medical School Office of Global Health award to study antiviral RNA interference in Aedes aegypti mosquitos collected at the joint US/Thai Armed Forces Medical Research Institute of Medical Sciences (AFRIMS) in Bangkok, Thailand.

MAY 15, 2013 – Novartis Institutes for BioMedical Research signs Research Agreement.
Anastasia Khvorova, PhD, Victor Ambros, PhD, and Silvia Corvera, MD have signed an agreement with David Morrissey, PhD and Novartis for a collaborative project titled “Inter and intra-cellular trafficking pathways for natural and therapeutic oligonucleotides”. This three year agreement will support research activities in the lab and share knowledge in the areas of exosome preparation and quality control, and oligonucleotide synthesis.

MAY 13, 2013  Victor Ambros is recently profiled in JCB (Journal of Cell Biology), "Victor Ambros: The broad scope of microRNAs" For full article, click here

APRIL 1, 2013  Andrei A Korostelev receives UMass Center for Clinical & Translational Science (UMCCTS) Award for his project, Novel Therapeutic Routes against Premature - Termination Disease.

MARCH 2013  New Molecular Cell publication from Phil Zamore's Lab:
Li, X. Z., Roy, C. K., Dong, X., Bolcun-Filas, E., Wang, J., Han, B. W., Xu, J., Moore, M. J., Schimenti, J. C., Weng, Z., and Zamore, P. D. (2013). An Ancient Transcription Factor Initiates the Burst of piRNA Production during Early Meiosis in Mouse Testes. Mol Cell 50, 67-81.

JANUARY 30, 2013  Craig Mello, at opening of Albert Sherman Center, "The world will remember what we do here.."  For full article, click here

JANUARY 1, 2013  EMBO Fellowship for Joerg Braun
Postdoctoral associate Joerg Braun, in the Moore Lab, was awarded an EMBO Long-Term fellowship for his proposal "Single molecule analysis of mammalian spliceosome assembly."  This fellowship will support Joerg's work on the molecular mechanism of alternative splcing of pre-mRNAs.  Alternative splicing of pre-mRNAs allows the synthesis of multiple proteins from a single gene and is considered as a critical source of complexity in higher eukaryotes, especially in mammals.  Joerg is studying the mechanism of splicing in mammals with single molecule techniques in collaboration with the Gelles laboratory at Brandeis University.  His work aims to improve our understanding of the regulation of human gene expression and pave the way to address diseases caused by aberrant spicing.

DECEMBER 2012  Paper from Han Lab featured in Nanotech News of National Cancer Institute
"Nanoparticles Glow through Thick Layer of Tissue"  An international research team has created unique photoluminescent nanoparticles that shine clearly through more than three centimeters, or more than an inch, of biological tissue, a depth that makes them a promising tool for deep-tissue optical bioimaging.  For full article, click here

DECEMBER 17, 2012  News from the Luban Lab
Though retroviruses like HIV are best known as the cause of diseases such as AIDS, a huge portion of the human genome consists of fossil remnants of retrovirus infection from millions of years ago. The Luban Lab has discovered that one of these so-called endogenous retroviruses generates RNA that is likely to be essential for human pluripotent stem cells. Ongoing work at UMass to understand the role of this endogenous retrovirus in stem cells will aid attempts to harness stem cells for therapies of a wide-range of human diseases.

DECEMBER 10, 2012 David Grunwald PhD receives CFAR development grant

DECEMBER 3, 2012  Anastasia Khvorova PhD joins RTI  
Dr. Khvorova brings outstanding accomplishment and innovation as a team leader and chief science officer with major oligonucleotide reagent and pharmaceutical companies.  Dr. Khvorova's lab will focus on the development of novel oligonucleotide-based therapeutics and drug development; mechanism of small RNA trafficking; RNA medicinal chemistry and delivery.

NOVEMBER 1, 2012  RNA silencing to treat Huntington's disease
Two UMass Medical School researchers report on promising new developments in the quest for a treatment for Huntington's disease in the Thursday, Nov. 1, issue of the New England Journal of Medicine. 

OCTOBER 17, 2012  UMMS, Carnegie Institute receive U.S. patent for RNAi
Discovery by Mello, Fire holds the promise of shutting down disease-causing genes in humans.  More...

JUNE 19, 2012 Victor R. Ambros PhD receives Janssen Award for Biomedical Research
With collaborator Gary B. Ruvkun PhD, of Mass General Hospital and Harvard Medical School, the two were awarded the 2012 Dr. Paul Janssen Award for Biomedical Research today by Johnson & Johnson for their co-discovery of microRNA (also known as miRNA), tiny molecules that are now understood to play a powerful role in gene expression and regulation. For the full article click here


MAY 15, 2012 David Grunwald PhD joins RTI   Dr. David Grunwald has decided to join the RTI faculty. He started his own group two years ago at the Technical University of Delft, The Netherlands. Dr. Gruenwald's lab is working on the interface between microscopy development and (nuclear) cell biology, with a strong focus on the travels of mRNA and nucleocytoplasmic transport. Observing the functions of individual molecules within high resolution spatial topographies with millisecond dynamics adds a unique perspective on the function of the living cell. Dr. Grunwald will move his lab to the new Albert Sherman building in early 2013.


MAY 1, 2012 Jane Freedman PhD one of two sites chosen for Translational Research Centers in Thrombotic and Hemostatic Disorders clinical studies using miRNA/mRNA.
This NIH U54 award will utilize multiple technologies and screening of an early-phase translation mRNA nanochip in humans with metabolic syndromes and determine the impact on thrombosis. Jane Freedman, MD, Director Translational Research Professor in Medicine, was awarded $3,355,400 to complete 3 clinical and translational studies over five years examining the role of mRNA and miRNA in obesity and weight loss and cardiovascular disease.

MARCH 4, 2012 Potential therapy for dyslipidemia & other diseases caused by miRNA deregulation.  Long-term, efficient inhibition of microRNA function in mice using rAAV vectors. Xie J, Ameres SL,Friedline R, Hung JH, Zhang Y, Xie Q, Zhong L, Su Q, He R, Li M, Li H, Mu X, Zhang H, Broderick JA, Kim JK, Weng Z, Flotte TR, Zamore PD, Gao G.  Nat Methods. 2012 Mar 4;9(4):403-9

JULY 18, 2011 Investiture  Melissa Moore, PhDHoward Hughes Medical Institute Investigator, professor of biochemistry & molecular pharmacology, was invested as the Eleanor Eustis Farrington Chair in Cancer Research

APRIL 27, 2011 Melissa Moore PhD win Gates Foundation grant
Grand Challenge Exploration grant was awarded to Melissa J. Moore, PhDHoward Hughes Medical Institute Investigator and Eleanor Eustis Farrington Chair of Cancer Research, professor of biochemistry & molecular pharmacology to pursue an innovative global health and development research project, titled “siRNA-based Therapeutics for Preeclampsia.”

APRIL 19, 2011 RNAi-Based Therapeutic Review is published
RNAi-based therapeutic strategies for metabolic diseases
Michael P. Czech, Myriam Aouadi and Gregory J. Tesz Nat. Rev. Endocrinol. 7, 473–484 (2011)

MARCH 21, 2011 Encapsulation technology for potential RNAi therapeutics
Glucan particles for selective delivery of siRNA to phagocytic cells in mice
Gregory J. Tesz1, Myriam Aouadi1, Matthieu Prot, Sarah M. Nicoloro, Emilie Boutet, Shinya U. Amano, Anca Goller, Mengxi Wang, Chang‑An Guo, William E. Salomon, Joseph V. Virbasius, Rebecca A. Baum, Mark J. O'Connor, Ernesto Soto, Gary R. Ostroff and Michael P. Czech2 Biochem. J. (2011) 436, 351–362

MARCH 19, 2011 Gene Therapy Center a step closer to treating Central Nervous System disorders.  MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression.  Xie J, Xie Q, Zhang H, Ameres SL, Hung JH, Su Q, He R, Mu X, Seher Ahmed S, Park S, Kato H, Li C, Mueller C, Mello CC, Weng Z, Flotte TR, Zamore PD, Gao G.Mol Ther. 2011 Mar;19(3):526-35.

JANUARY 2011 UMass Medical School and Lundbeck Inc. announce collaboration  Neil Aronin, MD, professor in medicine and cell biology, announced a research collaboration aimed at further development of a targeted therapy to slow or halt the progression of Huntington's disease using RNAi.

NOVEMBER 2010 Move Date is Announced
The RNA Therapeutic Institute is happy to announce it will be moving into the newly built Albert Sherman Center in March of 2013. To learn more about this exciting move and to see the RTI floor plan, click here.

OCTOBER 2010 UMMS Faculty Become Affiliates Of The RTI
The Directors of the RTI reached out around the UMMS campus to a vast variety of MD's and PhD's to begin building a team of outstanding researchers with specific interests and skills to have daily operational contact, in hopes to investigate ways to create new therapies that can be moved from the bench top to the bedside.

JULY 2010 RTI hires first Recruit 
Andrei Korostelev PhD,  has joined us from the University of California Santa Cruz, where he studied bacterial translation in the lab of Harry F. Noller. Dr. Korostelev will study translation and translation regulation in bacteria and eukaryotes using a combination of X-ray crystallography and biochemistry. Dr. Korostelev lab will be temporally housed in the biochemistry & molecular pharmacology until the RTI moves in 2013.

JUNE 2009 RNA Leadership Announced
Chancellor Michael Collins and Dean Terrence Flotte announced the leadership plan for the RNA Therapeutics Institute (RTI) – a key component of our Advanced Therapeutics Cluster. An executive group has been formed to govern the new institute. This group will work as a team to recruit new faculty and design the new home for RNA Therapeutics within the Albert Sherman Center. 
The executive group will be led by Nobel Laureate Craig C. Mello, PhD, Howard Hughes Medical Institute Investigator, Blais University Chair in Molecular Medicine and professor of molecular medicine and cell biology. The three other directors will be Melissa J. Moore, PhD, Howard Hughes Medical Institute Investigator and professor of biochemistry & molecular pharmacology; Phillip D. Zamore, PhD, Howard Hughes Medical Institute Investigator,Gretchen Stone Cook Chair in Biomedical Sciences and professor of biochemistry & molecular pharmacology; and Victor Ambros, PhD, Silverman Chair in Natural Sciences and professor of molecular medicine.