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"Engineering" Myoblast Transplantation

Jacques P. Tremblay

Myoblast transplantation (MT) designates the intramuscular implantation of myogenic cells as a treatment for muscle diseases. As a therapeutic tool, MT can act in 2 complementary manners: it can be a vehicle for a normal genome and it can increase the myogenic capacity of the host muscle. Although many experiments in rodents demonstrate these properties, the experiments in nonhuman primates allow for a better definition of the parameters that allow for making MT an applicable strategy in humans. In the present review, special attention is given to the clinical possibilities of MT. Two challenging factors are especially analyzed: the strategy of cell delivery and the control of rejection. The 3 issues that the authors identify as requiring further study to introduce improvements in MT design are intramuscular donor-cell migration, early donor-cell survival, and methods to avoid allograft rejection (development of specific tolerance or autotransplantation of genetically corrected myoblasts).

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