Fibrosis development in early-onset muscular dystrophies: Mechanisms and translational implications.
Duchenne muscular dystrophy (DMD) is one of the most devastating neuromuscular genetic diseases caused by the absence of dystrophin. The continuous episodes of muscle degeneration and regeneration in dystrophic muscle are accompanied by chronic inflammation and fibrosis deposition, which exacerbate disease progression. Thus, in addition of investigating strategies to cure the primary defect by gene/cell therapeutic strategies, increasing efforts are being placed on identifying the causes of the substitution of muscle by non-functional fibrotic tissue in DMD, aiming to attenuate its severity. Congenital muscular dystrophies (CMDs) are early-onset diseases in which muscle fibrosis is also present. Here we review the emerging findings on the mechanisms that underlie fibrogenesis in muscular dystrophies, and potential anti-fibrotic treatments.
Cell Biology Group, Department of Experimental and Health Sciences, Pompeu Fabra University (UPF), CIBER on Neurodegenerative diseases (CIBERNED), Barcelona, Spain. Electronic address: antonio.serrano@upf.edu.
Antonio L
AL
Serrano
Cell Biology Group, Department of Experimental and Health Sciences, Pompeu Fabra University (UPF), CIBER on Neurodegenerative diseases (CIBERNED), Barcelona, Spain; Institució Catalana de Recerca i Estudis Avançats (ICREA), Barcelona, Spain; Program of Vascular Physiology, Fundación Centro Nacional de Investigaciones Cardiovasculares (CNIC), Madrid, Spain. Electronic address: pura.munoz@upf.edu.
Pura
P
Muñoz-Cánoves
D017668:Age of Onset; D000818:Animals; D005355:Fibrosis; D006801:Humans; D008264:Macrophages / Q000473:pathology; D008954:Models, Biological; D009136:Muscular Dystrophies / Q000473:pathology* / Q000628:therapy; D057170:Translational Research, Biomedical
CMD; DMD; Fibrosis; Inflammation; Muscular dystrophy; Regeneration; Skeletal muscle