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Scot A. Wolfe, PhD

Research Focus - Therapeutic Genome Editing Systems

  • Application of novel Cas9/Cas12a-based genome editing systems
  • Use of therapeutic genome editing for β-hemoglobinopathies and other hematopoietic disorders  
  • Use of therapeutic genome editing for muscular dystrophies 
  • Development of delivery approaches for somatic cell genome editing
  • Development of artificial zinc finger-based transcriptional regulators 

Representative Publication

Iyer S, Suresh S, Guo D, et al. Precise therapeutic gene correction by a simple nuclease-induced double-strand break. Nature. 2019;568(7753):561-565.

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In the News

Getting Results…
  • 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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