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Gene Therapy Gives Hope For Patients With Muscular Dystrophy

Researchers say their recent discovery could be the first step in the path for a treatment for humans with muscular dystrophy. They say for the first time they have found a successful way to deliver gene therapy // to all of the muscles in a mouse.

Gene therapy for muscular dystrophy has been a hopeful treatment idea. However, finding a delivery method for the whole body has been a major obstacle for scientists. Initially researchers looked at different gene therapy delivery systems in mice , now they say they have a clear “proof of principle” that their discovery is a possible way to deliver genes to the whole body.

In their study, researchers say a type of viral vector, a specific type of virus, is able to hone in on muscle cells and does not trigger the immune system. Then the use of a growth factor appears to increase penetration into muscles of the gene. They say a single injection is able to carry the gene to all of the muscles in the mouse including the heart, diaphragm and limbs.

Researchers say they are not sure if this delivery system will work in larger animals or humans. However, they are seeking approval for a limited trial in humans to determine the safety of the viral vector in humans. If it is safe, then researchers hope to test the method in larger animals and humans.
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