Muscle Directed Gene Therapy Application for Maple Syrup Urine Disease (MSUD)

Awardee: Jenny Greig

Institution: Gene Therapy Program, University of Pennsylvania

Award Amount: $112,893

Funding Period: February 1, 2019 - January 31, 2020

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Repurposing FDA-approved drugs for Niemann Pick C Disease

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TFEB and Lysosomal exocytosis to prevent lung destruction in LAM