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Anastasia Khvorova, PhD

Professor, RNA Therapeutics Institute and Remondi Family Chair in Biomedical Research

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: 34774753 Read Publication

In the News

  • Atalanta Therapeutics founded by UMass Medical School and three faculty members
    Research News

    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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  • UMass Medical School community mourns loss of Jake Kennedy
    Chancellor, Community News

    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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  • Inside the new mRNA vaccines for COVID-19
    Research News

    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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  • Boston Marathon runners raise thousands for UMass Chan ALS research
    Community News

    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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  • UMass Cancer Center funds five UMass Chan research projects
    Research News

    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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  • Erik Sontheimer co-leading efforts to develop gene editing toolkit by NIH Somatic Cell Genome Editing Consortium
    Research News

    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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  • Anastasia Khvorova elected director of the American Society of Gene and Cell Therapy
    Research News

    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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