title: RNAi-based gene therapy for dominant Limb Girdle Muscular Dystrophies.
abstract: Limb Girdle Muscular Dystrophy (LGMD) refers to a group of 25 genetic diseases linked by common clinical features, including wasting of muscles supporting the pelvic and shoulder girdles. Cardiac involvement may also occur. Like other muscular dystrophies, LGMDs are currently incurable, but prospective gene replacement therapies targeting recessive forms have shown promise in pre-clinical and clinical studies. In contrast, little attention has been paid to developing gene therapy approaches for dominant forms of LGMD, which would likely benefit from disease gene silencing. Despite the lack of focus to date on developing gene therapies for dominant LGMDs, the field is not starting at square one, since translational studies on recessive LGMDs provided a framework that can be applied to treating dominant forms of the disease. In this manuscript, we discuss the prospects of treating dominantly inherited forms of LGMD with gene silencing approaches.
authors.affiliation: Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
authors.forename: Jian
authors.initials: J
authors.lastname: Liu
authors.forename: Scott Q
authors.initials: SQ
authors.lastname: Harper
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D015316:Genetic Therapy; D006801:Humans; D051379:Mice; D035683:MicroRNAs / Q000235:genetics / Q000627:therapeutic use; D049288:Muscular Dystrophies, Limb-Girdle / Q000145:classification / Q000235:genetics / Q000628:therapy; D009154:Mutation; D034741:RNA, Small Interfering / Q000235:genetics / Q000627:therapeutic use
chemical_list: D035683:MicroRNAs; D034741:RNA, Small Interfering