October 25, 2022
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VIDEO: Gene therapy potential treatment target for Duchenne muscular dystrophy
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CHICAGO – Jeffrey Chamberlain, PhD, professor of neurology at the University of Washington, discussed types of gene therapy as a possible treatment for muscular dystrophy at ANA 2022.
“One of the biggest issues is, how do you deliver a gene back to muscles?” Chamberlain said. “Currently, [adeno-associated virus] vectors are the only known method to efficiently deliver genes body-wide to muscle.”
Chamberlain said that there are several clinical trials underway for the study of gene delivery. However, he said the results “are not perfect.”