Epigenetic small molecule screening identifies a new HDACi compound for ameliorating Duchenne muscular dystrophy

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 resul..., , # Epigenetic small molecule screening identifies a new HDACi compound for ameliorating Duchenne muscular dystrophy.

Study Description: 
The data presented here is an original dataset of the raw data underlying the figures in the publication \"Epigenetic small molecule screening identifies a new HDACi compound for ameliorating Duchenne muscular dystrophy.\" In this publication, we used an established animal model for DMD, the zebrafish strain sapje, to screen a library of >800 epigenetic small molecules. We found that a single early treatment of the HDACi SR-4370 can ameliorate the muscle phenotype, increase lifespan in dmd zebrafish, and increase histone acetylation. These results support the effectiveness of small molecule screening in DMD zebrafish and add to the growing evidence of HDACi as promising candidates for treating DMD. The data was generated using the methods described in the resulting publication.

Research Domain: Biological sciences

Keywords: Duchenne muscular dystrophy,...,