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

Silvia Consalvi, Valentina Saccone, Chiara Mozzetta

Research output: Contribution to journalArticlepeer-review

Abstract

Duchenne muscular dystrophy (DMD) is a life-threatening genetic disease that currently has no available cure. A number of pharmacological strategies that aim to target events downstream of the genetic defect are currently under clinical investigation, and some of these are outlined in this report. In particular, we focus on the ability of histone deacetylase inhibitors to promote muscle regeneration and prevent the fibro-adipogenic degeneration of dystrophic mice. We describe the rationale behind the translation of histone deacetylase inhibitors into a clinical approach, which inspired the first clinical trial with an epigenetic drug as a potential therapeutic option for DMD patients.

Original languageEnglish
Pages (from-to)547-560
Number of pages14
JournalEpigenomics
Volume6
Issue number5
DOIs
Publication statusPublished - Oct 1 2014

Keywords

  • chromatin
  • epigenetics
  • HDAC inhibitors
  • muscle stem cells
  • muscular dystrophy
  • regeneration

ASJC Scopus subject areas

  • Genetics
  • Cancer Research
  • Medicine(all)

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