Parent Project Muscular Dystrophy Awards $348,891 Grant to University of Florida Wellstone Center
Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy, awarded the University of Florida Wellstone Muscular Dystrophy Cooperative Center a grant for $348,891. The project titled “Understanding and Improving AAV Gene Therapy for DMD” is led by H. Lee Sweeney, Ph.D., director of the UF Myology Institute and the Thomas H. Maren, M.D., eminent scholar chair in pharmacology and therapeutics in the UF College of Medicine. Dr. Sweeney has been the director of a Wellstone Center (one of the six) since 2005, which he relocated to the University of Florida in 2015.
In this project, Dr. Sweeney and his team at the University of Florida will evaluate ways to improve the delivery of gene therapy in disease with considerable fibrosis. Dr. Sweeney will also investigate if there are differences in how the loss of dystrophin impacts skeletal muscle compared to cardiac muscle and what this means for micro-dystrophin gene therapy. This information will potentially help inform the design of gene therapy and/or drug therapies to treat the hearts of people living with Duchenne, in a way that is independent of, or complementary to, gene therapy.
Learn more about Parent Project Muscular Dystrophy Awards $348,891 Grant to University of Florida Wellstone Center.Related news PPMD Awards University of Florida $1 Million for Novel Gene Therapy Approach Targeting the Heart.