Delineating the cause of gastrointestinal abnormalities in RASopathy disorders using human inducible pluripotent stem cells (iPSCs)

2014RASopthies
Written by Million Dollar Bike Ride

Awardee: Maria Kontaridis

Institution: Beth Israel Deaconess Medical Center/Harvard Medical School

Award Amount: $65,000

Funding Period: January 1, 2015 - December 31, 2015

Million Dollar Bike Ride
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STAT3 in Pulmonary Lymphangioleiomyomatosis (LAM)
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CNS gene therapy to restore NPC1 protein expression in cerebellar Purkinje cells and delay or prevent disease onset in feline Niemann-Pick type C1 (NPC1) disease.