Muscle bound.
Muscles mouse treated with modified human stem cells exhibit muscle fibers of producing human dystrophin (yellow), integrated from mouse muscle fibers (red).
Mirella Meregalli / University of Milan
recent breakthrough skin cells reprogrammed to act like embryonic stem cells stole the show ( science NOW, December 6) cells, but adult stem prove they have advantages of their own. In December 13 number Cell Stem Cell , researchers report using stem cells from patients with a form of muscular dystrophy to correct the disorder in mice. The results suggest that this strategy might someday treat muscular dystrophy in humans and other genetic disorders.
Duchenne muscular dystrophy, which affects mainly males, is caused by a mutation in the gene for a protein called dystrophin is vital for the proper functioning of muscles. The condition leads to muscle degeneration, and patients typically die in their 30s. One particular type of stem cells found in muscle may lead to new muscle tissue, so a team led by geneticist Luis Garcia Genethon, a biotechnology company nonprofit in Evry, France, investigated whether these cells could be used to reverse problems of dystrophin.
The researchers first obtained stem cells from patients with a muscle biopsy. Then they used a virus to insert a gene into the cells which corrects the mutation in the dystrophin gene. The researchers then injected the modified stem cells in the mouse leg arteries suffering from muscular dystrophy. In just three weeks, the muscles in the foot, shin and thigh began expressing human dystrophin protein, indicating that stem cells gave rise to muscle cells that had taken up residence in mouse muscle.
The real proof came in treadmill tests. The treated mice were able to run longer, maxing out at 15 minutes, the animals untreated patients, who managed only 10 minutes before being exhausted, the researchers report.
Garcia said his team now plans to test the strategy people with muscular dystrophy. He added that the technique could be used to treat a variety of genetic diseases, including other muscle disorders and skin.
Stem cell scientist Robert Lanza of Advanced Cell Technology in Worcester, Massachusetts, called promising strategy. He added that the stem cells used in the study have benefits on skin cells reprogrammed, including the removal of delicate matter to induce the cells to become muscle cells, but noted that both cells could pose risks because the viruses used to change could cause cancer.
geneticist Kay Davies of the University of Oxford, UK, said that for the approach to be successful in humans, stem cells must be delivered to the muscles. This could prove a huge challenge, she noted, because of repeated injections and high costs.
Related Sites
- More Duchenne muscular dystrophy
- More on stem cells
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