Genetic Mechanisms in Neuroendocrine cell Hyperplasia of Infancy (NEHI)

MDBRNEHI
Written by Million Dollar Bike Ride

Awardee: Lisa Young

Institution: The Children's Hospital of Philadelphia

Award Amount: $60,989

Million Dollar Bike Ride
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Pre-clinical animal model and cell evaluation of candidate therapies for human NUBPL disease.
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Tissue-specific and temporal reinstatement of Tcf4 to treat Pitt-Hopkins Syndrome