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

MDBRPitt Hopkins

Feb 1

Written By Million Dollar Bike Ride

Awardee: Andrew Kennedy

Institution: Bates College

Award Amount: $68,709

MDBRPitt Hopkins2019

Million Dollar Bike Ride

Genetic Mechanisms in Neuroendocrine cell Hyperplasia of Infancy (NEHI)

SETBP1 deficiency syndrome as an AKT modifer in human neurons