Tissue-specific and temporal reinstatement of Tcf4 to treat Pitt-Hopkins Syndrome

MDBRPitt Hopkins
Written By Million Dollar Bike Ride

Awardee: Andrew Kennedy

Institution: Bates College

Award Amount: $68,709

Million Dollar Bike Ride
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Genetic Mechanisms in Neuroendocrine cell Hyperplasia of Infancy (NEHI)
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SETBP1 deficiency syndrome as an AKT modifer in human neurons