Data from: Epigenetic small molecule screening identifies a new HDACi compound for ameliorating Duchenne muscular dystrophy
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https://datadryad.org/dataset/doi:10.5061/dryad.q573n5twd
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These data are the original data used to produce the figures, tables, and
supplemental information for the manuscript "Epigenetic small
molecule screening identifies a new HDACi compound for ameliorating
Duchenne muscular dystrophy". In this study, our goal was to identify
histone deacetylase inhibitors (HDACi), or other classes of epigenetic
small molecules that are beneficial for DMD. Using an established animal
model for DMD, the zebrafish dmd mutant strain sapje, we screened
a library of over 800 epigenetic small molecules. Our screening identified
a new HDACi, SR-4370, that ameliorated dmd mutant zebrafish skeletal
muscle degeneration, as well as additional HDACi that have previously been
shown to improve dmd zebrafish. We find that a single early treatment of
HDACi can ameliorate the muscle phenotype and increase lifespan in dmd
zebrafish. Furthermore, we find that HDACi treatments that improve dmd
muscle also cause increased histone acetylation in zebrafish larvae. Our
results add to the growing evidence that HDACi are promising candidates
for treating DMD. Our study also provides further support for the
effectiveness of small-molecule screening in dmd zebrafish.
提供机构:
Dryad
创建时间:
2025-09-09



