Myoblast transplantation: clinical trials and perspectives. Mini-review

Abstract

Myoblast transplantation (MT) is proposed to challenge the repair of striated muscles in various pathological conditions. Indeed, myoblasts are of easy procurement and expansion from small muscle biopsies. They are naturally able to participate to skeletal muscle regeneration through cell-cell fusion, leading to the formation of new or hybrid muscle fibers. In an autologous context, MT relies on the localized increased muscle generative capacity brought by large number of myoblasts : cells may be transferred from a clinically unaffected area to the disabled territory. In an heterologous context, MT further allows to transfer a non-pathologic, donor genome into the disabled recipient muscle fibers. Autologous approaches are then developed as localized repairs are mandated, while heterologous approaches are used in the context of generalized, advanced degenerative diseases. Whatever the immunological situation, however, MT is hampered by several issues, mainly the poor survival and the limited migratory ability of the myoblasts. Some solutions are emerging, which recently allowed the set-up of new clinical trials in dedicated indications.