Gene-edited stem cells for modeling rare mutations in cystic fibrosis (CF)
Awardee: Amy Firth
Institution: University of Southern California
Award Amount: $63,000
Funding Period: January 1, 2017 - December 31, 2017
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Known and novel members of the endolysosomal transportome/channelome as candidates to rescue loss of TRPML1 function in MLIV patients
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