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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

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.

Getting Results…

UMass Chan scientists receive $2.3 million from Rett Syndrome Research Trust for new research

Rett syndrome is a rare genetic neurological disorder that occurs primarily in girls, eventually robbing them of the ability to speak, walk or use their hands, among other devastating effects.

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Science for Living: Scot Wolfe explains revolutionary CRISPR gene-editing treatment

A UMass Chan professor of molecular, cell & cancer biology explains the science behind CRISPR/Cas9 gene-editing technology, following the FDA’s first approval of a gene-editing treatment for sickle cell disease.

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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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