Cell therapies for inherited myopathies.
OBJECTIVE
Cell therapies for inherited myopathies are based on the implantation of normal or genetically corrected myogenic cells into the body. This review summarizes the recent progress in this field, systematized according to the factors important for success.


RESULTS
In the choice of donor cells, myoblasts derived from satellite cells remain the best choice. Some studies on the population of muscle-derived stem cells in mice suggested that these cells may have some advantages over myoblasts; however, no results supporting this advantage have been presented in a primate model. Recent studies on bone marrow transplantation as a systemic source of myogenic precursors for the treatment of myopathies were disappointing. Concerning donor cell delivery, intramuscular myoblast injection remains the only way that can significantly introduce exogenous myogenic cells into the muscles. A recent study in primates showed some parameters of myoblast injection that could be useful in the human. Progress was made in mice to understand the factors that could favor the migration of the donor myoblasts in the host muscles. Concerning donor cell survival, analysis of immune cell infiltration dynamics allowed a better understanding of the factors implicated in early donor cell death. Progress was made on the control of acute rejection for myoblast transplantation in primates. So far, few mouse experiments have advanced the field of tolerance induction toward myogenic cells.


CONCLUSIONS
Myoblast transplantation (intramuscular injection of satellite cell-derived myoblasts) currently remains the only cell-based therapy that has produced promising results in the context of a preclinical model such as the nonhuman primate.
Skuk
Daniel
D
Unité de recherche en Génétique humaine, Centre de Recherche du Centre Hospitalier de l'Université Laval, Québec, Canada.
Tremblay
Jacques P
JP
D000818:Animals; D002470:Cell Survival / Q000502:physiology; D017690:Cell Transplantation / Q000379:methods; D064987:Cell- and Tissue-Based Therapy / Q000379:methods*; D004195:Disease Models, Animal; D005260:Female; D005500:Follow-Up Studies; D030342:Genetic Diseases, Inborn / Q000628:therapy; D000882:Haplorhini; D008297:Male; D051379:Mice; D032342:Muscle Cells / Q000637:transplantation; D009135:Muscular Diseases / Q000235:genetics* / Q000628:therapy*; D032446:Myoblasts / Q000637:transplantation*; D018570:Risk Assessment; D012720:Severity of Illness Index; D014019:Tissue Donors; D016896:Treatment Outcome