is in the delivery. mice treated with dystrophic muscle gene delivered via the leaky blood vessels (bottom) looks more like normal tissue (top) than untreated dystrophic muscle (middle).
Any seller of door-to-door knows that the key to success is getting the customer to open simply. Now, therapists genes in the hope of curing muscular dystrophy found the same thing: Get the blood vessels to open the secret is to deliver a therapeutic gene to the muscle tissue. The result shows for the first time as genes injected into the bloodstream can be incorporated in a specific type of tissue all over the body.
The most common form of muscular dystrophy, Duchenne muscular dystrophy, is caused by a mutant protein called dystrophin. The protein affects all muscles of the body - including skeletal muscle, heart, and diaphragm which controls breathing - and the disease usually kills its victims in their 20s. The researchers attempted to replace the dystrophin gene for over a decade. Although they were designed a common cold virus to carry a corrected version of the dystrophin gene, researchers have had difficulty delivering the solution to all muscles.
To see if the capillaries that leak might help, molecular geneticist Jeffrey Chamberlain and colleagues at the University of Washington, Seattle, first injected healthy mice with a virus containing a test protein turns blue fabric with a compound called VEGF, which causes blood vessels to expand and makes them leaky in the process. More VEGF they used the bluest muscles'S mice became, indicating higher levels of the virus. non-muscle organs have taken the virus, but did not make the color transforming protein. Encouraged, the team injected VEGF and virus containing a functional copy of the dystrophin gene in mice lacking the gene. Eight weeks later, most of the muscles contain dystrophin appropriate, and the supplemented muscles resist injury better. Just one injection could restore normal dystrophin levels and prevent muscle break, reports the online team in the July 25 Nature Medicine .The result is "fantastic" and a "big step forward "for gene therapy, therapist says Savio Woo of Mount Sinai School of Medicine in New York gene. And research has made a convert of neurobiologist Beverly Davidson of the University of Iowa, Iowa City, who said that it had become skeptical that delivery every muscle could be accomplished. "He gave me wrong," she said, but warned that "the biggest hurdle will be how to translate that to a human patient."
Related Sites
Muscular Dystrophy Association
Information Duchenne NIH
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