Adeno-associated virus serotype 9-mediated gene therapy for Adrenomyeloneuropathy
Awardee: Florian Eichler
Institution: Massachusetts General Hospital / Harvard Medical School
Award Amount: $50,000
Funding Period: January 1, 2015 - December 31, 2015
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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.
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