Monkeys that had a gene injected into their legs developed bigger, stronger thighs in an experiment that may pave the way for human trials testing the therapy in people with muscle-wasting diseases.
Several diseases including muscular dystrophy and amyotrophic lateral sclerosis, or Lou Gehrig’s disease, cause muscle weakness and have no effective treatments, said Jerry Mendell, director of the gene therapy center at Nationwide Children’s Hospital in Columbus, Ohio, and an author of the study published today in Science Translational Medicine.
The therapy works by blocking a protein, myostatin, that degrades muscle. Reversing muscle loss in the thigh may help patients who struggle to stand or walk, said R. Rodney Howell, chairman of the board of the Muscular Dystrophy Association, a nonprofit group based in Tucson, Arizona. The results may also interest a group of people who aren’t the intended beneficiaries -- athletes who want to improve performance, Howell said.
“We’re always looking for new treatments and at this time we don’t have any specific cures for the muscular dystrophies so an observation like this that might well benefit a lot of people is very exciting,” Howell said today in a telephone interview.
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