Known and novel members of the endolysosomal transportome/channelome as candidates to rescue loss of TRPML1 function in MLIV patients

Awardee: Christian Michael Grimm

Institution: University of Munich, LMU

Award Amount: $40,000

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

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The Role of Regulatory T cells in the Pathogenesis and Therapy of LAM

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Gene-edited stem cells for modeling rare mutations in cystic fibrosis (CF)