Transcriptomics

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X-linked myotubular myopathy is associated with epigenetic alterations and is ameliorated by HDAC inhibition


ABSTRACT: X-linked myotubular myopathy (XLMTM) is a fatal neuromuscular disorder caused by loss of function mutations in MTM1 . At present, there are no directed therapies for XLMTM, and incomplete understanding of disease pathomechanisms. To address these knowledge gaps, we performed a drug screen in mtm1 mutant zebrafish andidentified four positive hits, including valproic acid, which functions as a potent suppressor of the mtm1 zebrafish phenotype via HDAC inhibition. We translated these findings to a mouse XLMTM model, and showed that valproic acid ameliorates the murine phenotype.

ORGANISM(S): Mus musculus Danio rerio

PROVIDER: GSE210642 | GEO | 2022/08/08

REPOSITORIES: GEO

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