title: Cellular Therapies for Muscular Dystrophies: Frustrations and Clinical Successes.
abstract: Cell-based therapy for muscular dystrophies was initiated in humans after promising results obtained in murine models. Early trials failed to show substantial clinical benefit, sending researchers back to the bench, which led to the discovery of many hurdles as well as many new venues to optimize this therapeutic strategy. In this review we summarize progress in preclinical cell therapy approaches, with a special emphasis on human cells potentially attractive for human clinical trials. Future perspectives for cell therapy in skeletal muscle are discussed, including the perspective of combined therapeutic approaches.
authors.affiliation: Sorbonne Universités, UPMC Univ Paris 06, INSERM UMRS974, CNRS FRE3617, Center for Research in Myology, Paris, France .
authors.forename: Elisa
authors.initials: E
authors.lastname: Negroni
authors.affiliation: Sorbonne Universités, UPMC Univ Paris 06, INSERM UMRS974, CNRS FRE3617, Center for Research in Myology, Paris, France .
authors.forename: Anne
authors.initials: A
authors.lastname: Bigot
authors.affiliation: Sorbonne Universités, UPMC Univ Paris 06, INSERM UMRS974, CNRS FRE3617, Center for Research in Myology, Paris, France .
authors.forename: Gillian S
authors.initials: GS
authors.lastname: Butler-Browne
authors.affiliation: Sorbonne Universités, UPMC Univ Paris 06, INSERM UMRS974, CNRS FRE3617, Center for Research in Myology, Paris, France .
authors.forename: Capucine
authors.initials: C
authors.lastname: Trollet
authors.affiliation: Sorbonne Universités, UPMC Univ Paris 06, INSERM UMRS974, CNRS FRE3617, Center for Research in Myology, Paris, France .
authors.forename: Vincent
authors.initials: V
authors.lastname: Mouly
mesh_terms: D000818:Animals; D064987:Cell- and Tissue-Based Therapy / Q000379:methods*; D002986:Clinical Trials as Topic; D006801:Humans; D051379:Mice; D024510:Muscle Development; D018482:Muscle, Skeletal / Q000473:pathology; D009136:Muscular Dystrophies / Q000473:pathology / Q000628:therapy*; D032446:Myoblasts / Q000166:cytology / Q000502:physiology / Q000637:transplantation*; D020286:Pericytes / Q000166:cytology / Q000502:physiology / Q000637:transplantation*; D039904:Pluripotent Stem Cells / Q000166:cytology / Q000502:physiology / Q000637:transplantation*; D032496:Satellite Cells, Skeletal Muscle / Q000166:cytology / Q000502:physiology / Q000637:transplantation*; D014182:Transplantation, Autologous; D014184:Transplantation, Homologous; D017211:Treatment Failure