Histone deacetylase inhibitors: a potential epigenetic treatment for Duchenne muscular dystrophy

Epigenomics Review of the rationale behind the use of an epigenetic therapy as a potential pharmacological treatment of Duchenne muscular dystrophy, by Silvia Consalvi, Valentina Saccone and Chiara Mozzetta, from IRCCS Santa Lucia Foundation and CNR Institute of Cellular Biology & Neurobiology (both Rome, Italy).

Duchenne muscular dystrophy is a life-threatening genetic disease that currently has no available cure, and is caused by recessive mutations in the X-linked dystrophin gene. It is the most common and severe form of muscular dystrophy, and as a result various interventions are currently in research and development.

This Epigenomics Review focuses on the potential of histone deacetylase inhibitors to promote muscle regeneration and prevent the fibroadipogenic degeneration. By Silvia Consalvi, Valentina Saccone and Chiara Mozzetta, from IRCCS Santa Lucia Foundation and CNR Institute of Cellular Biology & Neurobiology (both Rome, Italy), this article highlights cellular effectors of skeletal muscle regeneration, new players and targets in epigenetic regulation of muscle differentiation, HDAC inhibitors and finally the Phase I/II clinical trial with givinostat, which was the first clinical trial for this disease that investigated an epigenetic drug.

Consalvi S, Saccone V, Mozzetta C. Histone deacetylase inhibitors: a potential epigenetic treatment for Duchenne muscular dystrophy. Epigenomics 6(5), 547—560 (2016).

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