Scot Wolfe receives funding for collaborative research to develop gene editing therapy for muscular dystrophy

Scot Wolfe, PhD, and collaborator Charles Emerson, Jr. (professor of neurology and Director of the Wellstone Muscular Dystrophy Program at UMass Chan) have received a three-year grant from the Muscular Dystrophy Association to develop a precise genome editing therapy to correct a common mutation in the TCAP gene, which leads to the R7 subtype of limb girdle muscular dystrophy. The project, which combines the use of stem cell, nanoparticle, mouse model and genome editing technologies, will provide a versatile platform to develop correction therapies for other gene mutations that cause other types of muscular dystrophy.