Research Focus - Medicinal Chemistry of Therapeutic RNAs
Development of novel oligonucleotide-based therapeutics
Drug development
Mechanism of small RNA trafficking
RNA medicinal chemistry and delivery
Representative Publications
An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis. Yenilmez B, Wetoska N, Kelly M, Echeverria D, Min K, Lifshitz L, Alterman JF, Hassler MR, Hildebrand S, DiMarzio C, McHugh N, Vangjeli L, Sousa J, Pan M, Han X, Brehm MA, Khvorova A, Czech MP.Mol Ther. 2021 Nov 10:S1525-0016(21)00578-5. doi: 10.1016/j.ymthe.2021.11.007. Online ahead of print.PMID: 34774753Read Publication
Atalanta Therapeutics founded by UMass Medical School and three faculty members
Atalanta Therapeutics, a biotech founded by UMass Medical School and three faculty research scientists to pioneer treatment options for neurodegenerative diseases, has launched with financing by venture capital fund F-Prime Capital and strategic collaborations with Biogen and Genentech.
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, Graduate School of Biomedical Sciences, School of Medicine, Medicine, Clinician, Faculty, Craig C. Mello, Anastasia Khvorova, RNA Therapeutics Institute, Aronin Lab, Program in Molecular Medicine, Neil Aronin,
Chancellor, Community News
Oct 14, 2020
UMass Medical School community mourns loss of Jake Kennedy
On behalf of the UMass Medical School community, we extend our sincere condolences to Jake Kennedy’s family and to all who knew and loved him. Jake Kennedy died Tuesday, Oct. 13, at age 65, in his Salem, N.H., home of amyotrophic lateral sclerosis.
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, Graduate School of Biomedical Sciences, School of Medicine, Michael F. Collins, Amyotrophic Lateral Sclerosis, Anastasia Khvorova, Neurology, RNA Therapeutics Institute, Program in Molecular Medicine,
Research News
Dec 18, 2020
Inside the new mRNA vaccines for COVID-19
The new vaccines by Pfizer/BioNTech and Moderna use messenger RNA to stimulate the immune system to protect against COVID-19. These vaccines are the first of their kind and researchers at UMass Medical School are among the leading RNA biologists in the world.
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, Graduate School of Biomedical Sciences, School of Medicine, Office of Advancement, Faculty, Craig C. Mello, Anastasia Khvorova, Jonathan K. Watts, Novel Coronavirus, Biochemistry and Molecular Pharmacology, RNA Therapeutics Institute, Ambros Lab, Phillip Zamore, Program in Molecular Medicine,
Community News
Apr 5, 2022
Boston Marathon runners raise thousands for UMass Chan ALS research
ALS research at UMass Chan Medical School is the big beneficiary as select Boston Marathon runners raise tens of thousands of dollars each year. This year, five competitors in the April 18 event are running as part of the UMass Chan ALS Cellucci Fund Boston Marathon team and two sons of the late Jake Kennedy will run in support of the Jake Kennedy ALS Fund.
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, Graduate School of Biomedical Sciences, School of Medicine, Clinician, Faculty, Amyotrophic Lateral Sclerosis, Anastasia Khvorova, Neurology, RNA Therapeutics Institute, Latest News,
Research News
Nov 17, 2021
UMass Cancer Center funds five UMass Chan research projects
The UMass Cancer Center Pilot Project Program has awarded seed grants to five UMass Chan Medical School research projects to enable investigators to gather pilot data for external cancer-focused funding.
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, Graduate School of Biomedical Sciences, School of Medicine, Dermatology, Medicine, Cancer Center, Thomas Fazzio, Michael R. Green, Anastasia Khvorova, John Harris, Molecular, Cell and Cancer Biology, Population and Quantitative Health Sciences, RNA Therapeutics Institute, Fazzio Lab, Rajani Sadasivam, Marcus Ruscetti, Latest News, Prabhani Atukorale, Chan Zhou, Elise Stevens,
Research News
Apr 7, 2021
Erik Sontheimer co-leading efforts to develop gene editing toolkit by NIH Somatic Cell Genome Editing Consortium
Six UMass Medical School scientists are among the members of the National Institutes of Health’s Somatic Cell Genome Editing Consortium to publish a paper in Nature outlining the program’s goals.
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, Graduate School of Biomedical Sciences, School of Medicine, Cancer Center, Watts Lab, Faculty, Rare Disease, Guangping Gao, Anastasia Khvorova, Jonathan K. Watts, Scot A. Wolfe, Wen Xue, Wolfe Lab, Sontheimer Lab, Molecular, Cell and Cancer Biology, RNA Therapeutics Institute, Li Weibo Institute for Rare Diseases Research, Gao Lab, Microbiology and Physiological Systems,
Research News
May 11, 2020
Anastasia Khvorova elected director of the American Society of Gene and Cell Therapy
UMass Medical School RNA therapeutics expert Anastasia Khvorova, PhD, has been elected by the membership of the American Society of Gene and Cell Therapy to a three-year term as director-at-large/scientific & research council chair, which will begin at the 2020 ASGCT virtual annual meeting.
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, Graduate School of Biomedical Sciences, Cancer Center, Faculty, Anastasia Khvorova, RNA Therapeutics Institute,