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

MDBRPitt Hopkins
Written By Million Dollar Bike Ride

Awardee: Andrew Kennedy

Institution: Bates College

Award Amount: $51,242

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

Million Dollar Bike Ride
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In vitro differentiation of parathyroid cells from stem cells
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Pilot study to develop a small molecule activator of TCF4 as a treatment for Pitt-Hopkins syndrome