Gene Injection Helps Mice Fight Muscular Dystrophy

Researchers appear to have partially corrected muscular dystrophy in mice by injecting corrective genes directly into the mice’s muscles, according to a report last week in the British journal Nature. The study points the way toward the eventual use of gene therapy to arrest or reverse muscular dystrophy in patients, but years of research will be required before then, according to researchers from the University of Wisconsin and the John Radcliffe Hospital in Oxford, England.

The insertion of the genes led to the production of low levels--about 1% of the normal amount--of the key protein that is missing in people with muscular dystrophy. One particularly exciting aspect of the research was that the critical protein, called dystrophin, was produced in the heart as well as the skeletal muscles, said Leon I. Charash of the Muscular Dystrophy Assn.

Researchers do not know whether the mice’s muscles actually became stronger. Experts said it was unlikely that having 1% of the normal amount of dystrophin would alleviate symptoms in a person with muscular dystrophy.