RESEARCHERS COMBINE STEM CELLS,
GENE THERAPY TO TREAT DUCHENNE DYSTROPHY

MDA-supported researchers Louis Kunkel of Harvard Medical School and Jeffrey Chamberlain and Sheng Li of the University of Washington announced in the March 9 issue of Proceedings of the National Academy of Sciences that they’ve combined stem cell and gene therapy to successfully treat mice with Duchenne muscular dystrophy (DMD). Estanislao Bachrach at Harvard was a major contributor to this work.

The mice were treated with a special type of muscle-derived stem cell known as SP (side population) cells, which were given corrected genes for human dystrophin, the protein needed but deficient in DMD. The cells came from mouse muscles, and similar cells are believed present in human muscles.

The investigators injected the cells into mouse tail veins. They observed the cells migrate through the circulation and home specifically to damaged muscle areas in the DMD-affected animals, where they then inserted themselves and produced low but measurable amounts of human dystrophin.

“This work represents one of the first steps toward human trials of adult-derived [nonembryonic] stem cells,” Kunkel said, adding that the way is now paved for using genetically corrected stem cells from patients’ own muscles to treat muscle diseases.

Researchers believe this technique will minimize the risk of the body rejecting the therapeutic material.