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.

2014NPC
Written By Million Dollar Bike Ride

Awardee: Brittney Gurda

Institution: University of Pennsylvania, School of Veterinary Medicine

Award Amount: $35,000

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

Million Dollar Bike Ride
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Delineating the cause of gastrointestinal abnormalities in RASopathy disorders using human inducible pluripotent stem cells (iPSCs)
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Adeno-associated virus serotype 9-mediated gene therapy for Adrenomyeloneuropathy