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Deacetylase Inhibitors

Histone deacetylase inhibitors (HDAC inhibitors, HDI) are a class of compounds that interfere with the function of histone deacetylase.

Work undertaken on HDAC inhibitors on the mdx mouse (Minetit et al. 2006), demonstrated that HDAC2, among the various HDACs, is specifically deregulated in dystrophic muscles of mdx mice

Studies have since shown that dystrophic muscles show significant alterations in the epigenetic profile and they can be reversed by Deacetylase Inhibitors (DIs) or NO donors. The deregulated expression of HDAC2 and the altered epigenetic profile of dystrophic muscles have also been demonstrated in samples from DMD patients

A key aspect of DI action resides in the induction of follistatin (the myostatin antagonist) in muscle stem cells that appears to promote therapeutic regeneration of dystrophic muscles. The follistatin/myostatin axis is a relevant target for pharmacological intervention in DMD.

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  • "Regenerative pharmacology in the treatment of genetic diseases: The paradigm of muscular dystrophy". Mozzetta C, Minetti G, Puri PL.

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