ON1-Specific Precision Medicine Study

Project: Research project

Project Details


Perform whole genome sequencing, analyze data. Identify type of GBA1 mutation, search for other risk genes; Fabricate micropattern substrates; Reprogram fibroblasts from ON1, characterize iPS, initiate neuronal differentiation. Several other lines will be used as additional controls, including Gaucher’s and asyn triplication. Initiate SAR for putative GCase activators.
Effective start/end date6/26/156/26/18


  • WildKat 1, LLC (Agmt 6/26/15)


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