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

2014RASopthies

Jan 1

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

MDBR2014RASopathies

Million Dollar Bike Ride

STAT3 in Pulmonary Lymphangioleiomyomatosis (LAM)

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.