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Gene Therapy Shows Promise for Muscular Dystrophy
Date:5/21/2010

ts from an inherited deficiency of alpha-sarcoglycan, a muscle protein.

"There is no effective therapy to prevent the progressive weakness and loss of muscle that occurs in LGMD type 2D," Moxley explained. "The disease typically begins between 2 and 15 years of age, and many patients become wheelchair-bound by their teens. They have marked weakness of their shoulder and thigh muscles, and have difficulty performing many of the activities of daily life."

"Treatments are needed urgently," stated Cwik.

A previous study of the same gene transfer procedure had been successful in three patients with levels of the protein staying elevated for at least three months after treatment.

Here, a prominent group of muscular dystrophy researchers from the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital in Columbus, Ohio, injected three LGMD patients with a healthy gene, which succeeded in increasing both gene expression and muscle fiber levels. The effect persisted for six months, the longest yet.

Next, the researchers hope to inject the gene directly into a leg artery to see if those muscles will take up and use the protein.

But several obstacles remain.

"For gene therapy to be clinically beneficial -- meaning an improvement in strength -- multiple muscle groups will need to be treated simultaneously," Cwik said. "To do this will require regional [to an entire limb] or systemic delivery [to the entire body, such as intravenously]."

"This technique delivers the gene directly into the muscle through a needle. It is not practical to do this on large muscles, let alone several muscles, as it would require hundreds of injections," Tawil added. "To make this treatment viable, a system has to be devised where the virus-plus-normal gene can be injected into the circulation and have it deposited into all the muscles. The other obstacle is making sure that injecting the virus
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