Discovery of new therapies for hypertrophic cardiomyopathy in RASopathies using an innovative hiPSC-based high-throughput drug screening platform

MDBRRASopthies
Written By Million Dollar Bike Ride

Awardee: Fabrice Jaffré

Institution: Weill Cornell Medical College

Grant Amount: $75,431

Funding Period: February 1, 2022 - January 31, 2023

Summary:

Children with RASopathies often present with severe cardiomyopathies and have a 22% mortality rate by the end of the first year of life. Currently, no specific treatment exists for RASopathy children with hypertrophic cardiomyopathy, therefore there is an urgent need to identify novel therapeutic strategies. The overall goal of this proposal is to uncover innovative therapeutic approaches using human induced pluripotent stem cell-derived cardiomyocytes as a RASopathy disease model and as innovative human 2D and 3D high-throughput drug screening platforms. Completion of this proposal will identify therapeutic molecules for RASopathy children with hypertrophic cardiomyopathy at an unparalleled speed.

Million Dollar Bike Ride
Previous

Discovery of glycogen synthase inhibitors for validation as a novel therapeutic target for adult polyglucosan body disease (APBD)
Next

EXPLOITING NEURONS- AND BRAIN-DERIVED EXOSOMES AS BIOMARKER FOR CDKL5 DEFICIENCY DISORDER