For the first time, US researchers have used gene therapy to deliver full-length dystrophin and correct dystrophic muscle in immunocompetent adult mice in an animal model of Duchenne muscular dystrophy, according to their report, published in this week’s online early edition of the Proceedings of the National Academy of Sciences.
“The results provide the best evidence to date that gene therapy has great potential to treat muscular dystrophy,” senior researcher Dr. Jeffrey S. Chamberlain, of the University of Washington in Seattle, told Reuters Health.
In previous work, dystrophin was generally delivered to neonatal mice to show that the disease could be prevented, he explained. “Our work now shows for the first time that it is also possible to reverse some major problems associated with dystrophy,” Dr. Chamberlain said.
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