Scientists have succeeded in using gene therapy to restore some muscle function in patients with a certain type of muscular dystrophy.
“This study provides additional information regarding the feasibility of gene therapy for the treatment of muscular dystrophy,” said Dr. Valerie Cwik, executive vice president and research and medical director of the Muscular Dystrophy Association, which helped fund the research. “Specifically, it provides proof of principle, in people, for sustained gene expression [for at least six months] following treatment.”
This is the first time such a feat has been performed in humans, state the authors, who are presenting their findings at the annual meeting of the American Society of Gene & Cell Therapy in Washington, D.C.
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