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2016 News

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, Ph.D., 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 an 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, a total of about 2.5 million dollars. For details, please click here.

SEPTEMBER 23, 2016  Dr. Wen Xue, Assistant professor of RNA Therapeutics Institute received a 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, Ph.D., Assistant Professor of RTI,  selected to receive an 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. The 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 the 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, Ph.D., 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, Ph.D., 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 the 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, Ph.D., 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

WenGrant

APRIL 7, 2016 Wen Xue, Ph.D., Assistant professor of the RNA Therapeutics Institute at UMass Medical School, won a 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, Ph.D., received NIH research fellowship award. Dr. Osborn, a postdoctoral fellow in the  Khvorova lab, received an 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, Ph.D., 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, Ph.D., a postdoctoral fellow in Dr. Erik Sontheimer’s lab has received an 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, Ph.D. 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, Ph.D., 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. 

FEBRUARY 1, 2016, New publication  Wen Xue, Ph.D., Assistant Professor of RTI, is the 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, Ph.D., received a 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”.

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