Myoblast transplantation for inherited myopathies: a clinical approach

Myoblast transplantation (MT) is an experimental strategy for the potential treatment of myopathies. MT has two properties that make it potentially beneficial: genetic complementation and myogenic potential. Preclinical experiments on monkeys have shown that promising results can be obtained with MT in large muscles of primates depending on two conditions: appropriate immunosuppression and cell delivery by a method of high-density injections. Preclinical work on MT is being, or may be, addressed to: develop efficient methods of donor cell delivery applicable to clinics; control or avoid acute rejection by methods with the fewest secondary effects; understand the factors that condition the early survival of donor cells following transplantation; increase the success of each individual injection; re-engineer a functional structure in muscles that degenerates to fibrosis and fat substitution; and search for precursor cells with potential advantages over myoblasts.