Awardee: Brittney Gurda
Institution: University of Pennsylvania, School of Veterinary Medicine
Award Amount: $35,000
Funding Period: January 1, 2015 - December 31, 2015
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.
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Delineating the cause of gastrointestinal abnormalities in RASopathy disorders using human inducible pluripotent stem cells (iPSCs)
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