title: Functional and morphological recovery of dystrophic muscles in mice treated with deacetylase inhibitors.
abstract: Pharmacological interventions that increase myofiber size counter the functional decline of dystrophic muscles. We show that deacetylase inhibitors increase the size of myofibers in dystrophin-deficient (MDX) and alpha-sarcoglycan (alpha-SG)-deficient mice by inducing the expression of the myostatin antagonist follistatin in satellite cells. Deacetylase inhibitor treatment conferred on dystrophic muscles resistance to contraction-coupled degeneration and alleviated both morphological and functional consequences of the primary genetic defect. These results provide a rationale for using deacetylase inhibitors in the pharmacological therapy of muscular dystrophies.
authors.affiliation: Dulbecco Telethon Institute at Fondazione A. Cesalpino, Institute of Cell Biology and Tissue Engineering, San Raffaele Biomedical Science Park of Rome, Via Castel Romano 100, 00128, Rome, Italy.
authors.forename: G C
authors.initials: GC
authors.lastname: Minetti
authors.forename: C
authors.initials: C
authors.lastname: Colussi
authors.forename: R
authors.initials: R
authors.lastname: Adami
authors.forename: C
authors.initials: C
authors.lastname: Serra
authors.forename: C
authors.initials: C
authors.lastname: Mozzetta
authors.forename: V
authors.initials: V
authors.lastname: Parente
authors.forename: S
authors.initials: S
authors.lastname: Fortuni
authors.forename: S
authors.initials: S
authors.lastname: Straino
authors.forename: M
authors.initials: M
authors.lastname: Sampaolesi
authors.forename: M
authors.initials: M
authors.lastname: Di Padova
authors.forename: B
authors.initials: B
authors.lastname: Illi
authors.forename: P
authors.initials: P
authors.lastname: Gallinari
authors.forename: C
authors.initials: C
authors.lastname: Steinkühler
authors.forename: M C
authors.initials: MC
authors.lastname: Capogrossi
authors.forename: V
authors.initials: V
authors.lastname: Sartorelli
authors.forename: R
authors.initials: R
authors.lastname: Bottinelli
authors.forename: C
authors.initials: C
authors.lastname: Gaetano
authors.forename: P L
authors.initials: PL
authors.lastname: Puri
mesh_terms: D000818:Animals; D016189:Dystrophin / Q000235:genetics; D004791:Enzyme Inhibitors / Q000494:pharmacology*; D005355:Fibrosis / Q000473:pathology; D038681:Follistatin / Q000378:metabolism; D006877:Hydroxamic Acids / Q000494:pharmacology; D051379:Mice; D008810:Mice, Inbred C57BL; D018101:Mice, Inbred mdx; D009132:Muscles / Q000187:drug effects / Q000201:enzymology* / Q000473:pathology*; D009137:Muscular Dystrophy, Animal / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology; D010654:Phenylbutyrates / Q000494:pharmacology; D049031:Sarcoglycans / Q000378:metabolism; D032496:Satellite Cells, Skeletal Muscle / Q000166:cytology / Q000201:enzymology; D014635:Valproic Acid / Q000494:pharmacology
chemical_list: D016189:Dystrophin; D004791:Enzyme Inhibitors; D038681:Follistatin; D006877:Hydroxamic Acids; D010654:Phenylbutyrates; D049031:Sarcoglycans; C012589:trichostatin A; D014635:Valproic Acid