Optimizing Delivery and Endpoints of an AAV9-mediated Gene Therapy Trial in AMN

Awardee: Florian Eichler

Institution: Massachusetts General Hospital, Harvard Medical School

Award Amount: $50,000

Funding Period: January 1, 2016 - December 31, 2016

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Development of next-gen animal models for fibrous dysplasia (FD)/McCune-Albright syndrome (MAS)

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Towards Precision Medicine in the Treatment of Congenital Hyperinsulinism in Infancy