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PLENARY 3 | Loss of ARHGAP19 function as a novel driver of CMT | Natalia Dominik
Автор: ECMTF European CMT Federation
Загружено: 2025-11-24
Просмотров: 19
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This study identified that loss-of-function variants in the ARHGAP19 gene are a novel cause of inherited neuropathy. Using cellular and animal models, researchers screened a library of FDA-approved drugs to find potential treatments. The screen identified existing compounds that could rescue motor defects, highlighting a promising strategy for drug repurposing in CMT.
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