The gene therapy was able to perform in all muscles in the mouse, and would not necessarily have to carry the dystrophy gene
Researchers have found a delivery method for gene therapy that reaches all the voluntary muscles of a mouse – including heart, diaphragm and limbs – and reverses the process of muscle-wasting found in muscular dystrophy.
"We have a clear proof of principle that it is possible to deliver new genes body-wide to all the striated muscles of an adult animal. Finding a delivery method for the whole body has been a major obstacle limiting the development of gene therapy for the muscular dystrophies. Our new work identifies for the first time a method where a new dystrophin gene can be delivered, using a safe and simple method, to all of the affected muscles of a mouse with muscular dystrophy," said Dr. Jeffrey S. Chamberlain, professor of neurology and director of the Muscular Dystrophy Cooperative Research Center at the University of Washington School of Medicine in Seattle. He also has joint appointments in the departments of medicine and biochemistry.
Walter Neary | EurekAlert!
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