title: Histone deacetylase inhibitors: a potential epigenetic treatment for Duchenne muscular dystrophy.
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.
authors.affiliation: IRCCS Santa Lucia Foundation, Via Del Fosso di Fiorano 64, 00143 Rome, Italy.
authors.forename: Silvia
authors.initials: S
authors.lastname: Consalvi
authors.forename: Valentina
authors.initials: V
authors.lastname: Saccone
authors.forename: Chiara
authors.initials: C
authors.lastname: Mozzetta
mesh_terms: D000107:Acetylation; D000818:Animals; D002219:Carbamates / Q000494:pharmacology / Q000627:therapeutic use; D002454:Cell Differentiation / Q000187:drug effects / Q000235:genetics; D017321:Clinical Trials, Phase I as Topic; D017322:Clinical Trials, Phase II as Topic; D044127:Epigenesis, Genetic / Q000187:drug effects; D005786:Gene Expression Regulation; D056572:Histone Deacetylase Inhibitors / Q000494:pharmacology / Q000627:therapeutic use*; D006657:Histones / Q000378:metabolism; D006801:Humans; D018482:Muscle, Skeletal / Q000378:metabolism; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000235:genetics* / Q000378:metabolism; D009154:Mutation; D012038:Regeneration / Q000187:drug effects / Q000235:genetics; D032496:Satellite Cells, Skeletal Muscle / Q000166:cytology / Q000187:drug effects / Q000378:metabolism
chemical_list: D002219:Carbamates; D056572:Histone Deacetylase Inhibitors; D006657:Histones; C575255:givinostat
keywords: HDAC inhibitors; chromatin; epigenetics; muscle stem cells; muscular dystrophy; regeneration