Research Objective Intramuscular delivery of two repurposed FDA approved drugs will activate resident muscle stem cells. This therapeutic strategy will augment regeneration and restore strength to atrophied muscles. Impact Currently…
Research Objective Gene correction of muscle stem cells Impact These studies will develop a gene editing based therapy for one of the most prevalent lethal childhood disorders called Duchenne Muscular…
Research Objective To target therapeutics to muscle stem cells, the building blocks of skeletal muscle. Impact Drugs, genes and gene editing strategies can be delivered directly to muscle stem cells…
Research Objective The goal of this proposal is to define protocols to generate expandable, self-renewing human muscle stem cells (MuSC) from hiPS cells for Duchenne Muscular Dystrophy disease modeling and…
Research Objective Delivery of muscle stem cells presents a major roadblock for therapy. We explore novel approaches to increase the efficiency of delivering and monitoring muscle stem cells derived from…
Research Objective The goal of the proposed research is to use human embryonic stem cells to generate bladder epithelial progenitor cells that can be used to replace a cancerous bladder…
Research Objective To explore if iPSC-based therapy can prevent bladder damage due to radiation therapy, thereby limiting the unintended consequences of treatments for prostate, gynecologic and colorectal cancers. Impact This…
Research Objective To examine the efficacy of blocking blood borne GDF11 activity to rejuvenate aged human muscle stem cell regenerative capacity. Impact This project will provide a proof-of-principle that GDF11…
Translational Candidate A gene editing therapy for Duchenne muscular dystrophy that permanently removes a hotspot region of patient mutations to restore dystrophin. Area of Impact Duchenne muscular dystrophy (DMD), a…
Translational Candidate We engineered a human embryonic stem cell-secreted signaling protein into a biologic for treatment of skeletal muscle disorders. Area of Impact Skeletal muscle disorders (including DM1 and sarcopenia)…
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