Cell therapy flexes its muscles

Muscular dystrophy is a group of diseases that degenerates skeletal muscle. Duchenne muscular dystrophy, caused by a mutation in the gene that codes the dystrophin protein, affects one in every 3,500 male infants and leaves many sufferers unable to walk beyond the age of 11 years. There is no cure, but a promising route to treatment is to replace patients' defective cells with adult stem cells that proliferate in response to injury and cause skeletal muscle to regenerate.

Ingo Riederer and colleagues from the Pierre and Marie Curie University, Paris, in collaboration with researchers at the Oswaldo Cruz Institute in Rio de Janeiro, Brazil, and the Faculty of Sciences in Agadir, Morocco, have taken an important step towards bringing this potential therapy into the clinic by studying the kinetics of adult myoblasts, cells destined to become skeletal muscle, injected into mice that had been exposed to a muscle injury.

"There are many problems with transplanting cells because most of them die before they have a chance to appropriately spread in the injured muscle," says team member Wilson Savino of the Oswaldo Cruz Institute.

After injection, the researchers found that most of the human myoblasts had already differentiated into mature muscle cells by day five, and that the differentiation coincided with a reduction in proliferation and limited migration of the donor cells. The underlying cause is unknown, but the study shows that rapid differentiation is a limiting factor during transplantation. The team now plans to investigate specific molecules that can simultaneously delay cell differentiation and enhance proliferation.

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