Impact on Niemann-Pick C disease of the cross-talk between the c-Abl and the TFEB signaling pathways

2016NPC
Written By Million Dollar Bike Ride

Awardee: Silvana Zanlugo

Institution: Pontificia Universidad Católica de Chile

Award Amount: $51,000

Funding Period: January 1, 2017 - December 31, 2017

Million Dollar Bike Ride
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A multi-center, prospective, minimal intensity BMT trial for dyskeratosis congenita
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Establishing 3-Dimensional (3D) Retinal Optic Cups using patient-specific CRB1 Induced Pluripotent Stem Cells.