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Jonathan K. Watts, PhD

Research Focus - Platform Technology for Oligonucleotide Therapeutics

  • Chemical optimization of oligonucleotide therapeutics (for gene silencing and gene activation), with a focus on the brain and lung
  • Design and delivery of RNA guides for genome editing
  • Design of DNA donors for high efficiency precision gene editing
  • Identification of disease targets in glioblastoma and neurodegenerative diseases

Representative Publication

Khvorova A, Watts JK. The chemical evolution of oligonucleotide therapies of clinical utility. Nat Biotechnol. 2017;35(3):238-248.

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In the News

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  • UMass Chan ALS paper selected for STAT Madness
    Research News

    UMass Chan ALS paper selected for STAT Madness

    Robert H. Brown Jr., DPhil, MD, Jonathan Watts, PhD, and colleagues showed the ability to suppress mutant forms of the ALS gene known as C9ORF72 in a single-patient pilot study. The paper reporting the results is part of the 2022 STAT News STAT Madness competition.

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  • UMass Chan clinical trial shows antisense oligonucleotide safely suppresses mutant ALS gene in pilot human study
    Research News

    UMass Chan clinical trial shows antisense oligonucleotide safely suppresses mutant ALS gene in pilot human study

    Robert H. Brown Jr., DPhil, MD, Jonathan Watts, PhD, and colleagues have shown the ability to suppress mutant forms of an ALS gene in a single-patient pilot study.

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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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  • 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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