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Drug Screening for Dysferlinopathy Using iPSCs

Given the lack of effective pharmaceutical approaches to the treatment of the progressive muscle disorder dysferlinopathy, researchers led by Yuko Kokubu and Hidetoshi Sakurai (Kyoto University, Japan) sought to use screen myocytes from induced pluripotent stem cells derived from a dysferlinopathy patient (Miyoshi myopathy). Reporting in STEM CELLS Translational Medicine, the authors now report nocodazole as a drug candidate that affects microtubule organization and autophagy function. The team hopes that their new findings will contribute to the enhance clinical treatment of dysferlinopathy.