Targeting molecular chaperone HSP90 to treat NPC1 disease

2017NPC
Written By Million Dollar Bike Ride

Awardee: Nina Pipalia

Institution: Weill Cornell Medical College

Award Amount: $49,645

Funding Period: January 1, 2018 - December 31, 2018

Publications:

HSP90 inhibitors reduce cholesterol storage in Niemann-Pick type C1 mutant fibroblasts

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AAVrh.10-mediated Npc2 Gene transfer for the treatment of Npc2 Deficient Niemann-Pick disease
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Exploring the therapeutic potential of RANKL inhibition in Fibrous Dysplasia of bone: studies on murine transgenic models of the disease.