References
- Papers of special note have been highlighted as either of interest (•) or of considerable interest (••) to readers.
- Dalkilic I, Kunkel LM. Muscular dystrophies: genes to pathogenesis. Curr Opin Genet Dev. 2003;13(3):231–238.
- Petrof BJ, Shrager JB, Stedman HH, et al. Dystrophin protects the sarcolemma from stresses developed during muscle contraction. Proc Natl Acad Sci USA. 1993;90(8):3710–3714.
- Straub V, Campbell KP. Muscular dystrophies and the dystrophin-glycoprotein complex. Curr Opin Neurol. 1997;10(2):168–175.
- Mercuri E, Muntoni F. Muscular dystrophy: new challenges and review of the current clinical trials. Curr Opin Pediatr. 2013;25(6):701–707. DOI:10.1097/MOP.0b013e328365ace5.
- Serrano AL, Mann CJ, Vidal B, et al. Cellular and molecular mechanisms regulating fibrosis in skeletal muscle repair and disease. Curr Top Dev Biol. 2011;96:167–201. DOI:10.1016/B978-0-12-385940-2.00007-3.
- Farup J, Madaro L, Puri PL, et al. Interactions between muscle stem cells, mesenchymal-derived cells and immune cells in muscle homeostasis, regeneration and disease. Cell Death Dis. 2015;6:e1830. DOI:10.1038/cddis.2015.198.
- Judson RN, Zhang RH, Rossi FM. Tissue-resident mesenchymal stem/progenitor cells in skeletal muscle: collaborators or saboteurs? Febs J. 2013;280(17):4100–4108. DOI:10.1111/febs.12370.
- Lemos DR, Babaeijandaghi F, Low M, et al. Nilotinib reduces muscle fibrosis in chronic muscle injury by promoting TNF-mediated apoptosis of fibro/adipogenic progenitors. Nat Med. 2015;21(7):786–794. DOI:10.1038/nm.3869.
- Mozzetta C, Consalvi S, Saccone V, et al. Fibroadipogenic progenitors mediate the ability of HDAC inhibitors to promote regeneration in dystrophic muscles of young, but not old Mdx mice. EMBO Mol Med. 2013;5(4):626–639. DOI:10.1002/emmm.201202096.
•Exciting paper studying the fibroadipogenic progenitors (FAPs) as cellular determinants of the beneficial effects of HDAC inhibitors. FAPs mediate HDACi effects promoting muscles regeneration in a stage-specific manner.
- Minetti GC, Colussi C, Adami R, et al. Functional and morphological recovery of dystrophic muscles in mice treated with deacetylase inhibitors. Nat Med. 2006;12(10):1147–1150.
•Interesting paper describing the functional and morphological effects of HDAC inhibitors treatments. HDAC inhibitors increase myofiber size and counter the functional decline of dystrophic muscles
- Consalvi S, Mozzetta C, Bettica P, et al. Preclinical studies in the mdx mouse model of Duchenne muscular dystrophy with the histone deacetylase inhibitor Givinostat. Mol Med. 2013;19:79–87. DOI:10.2119/molmed.2013.00011.
••Excellent paper demonstrating the effectiveness of a long-term treatment with Givinostat. The findings of this paper provide preclinical basis for a translation on DMD patients.
- Colussi C, Mozzetta C, Gurtner A, et al. HDAC2 blockade by nitric oxide and histone deacetylase inhibitors reveals a common target in Duchenne muscular dystrophy treatment. Proc Natl Acad Sci USA. 2008;105(49):19183–19187. DOI:10.1073/pnas.0805514105.
- Cacchiarelli D, Martone J, Girardi E, et al. MicroRNAs involved in molecular circuitries relevant for the Duchenne muscular dystrophy pathogenesis are controlled by the dystrophin/nNOS pathway. Cell Metab. 2010;12(4):341–351. DOI:10.1016/j.cmet.2010.07.008.
- Saccone V, Consalvi S, Giordani L, et al. HDAC-regulated myomiRs control BAF60 variant exchange and direct the functional phenotype of fibro-adipogenic progenitors in dystrophic muscles. Genes Dev. 2014;28(8):841–857. DOI:10.1101/gad.234468.113.