October 25, 2022
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Source/Disclosures Source:
Chamberlain J, et al. Neurogenetics and gene therapy. Presented at: American Neurological Association Annual Meeting; October 23-25; Chicago.
Disclosures: Chamberlain reports that some of the gene therapy vehicles discussed are currently the subject of patent applications.
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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 return a gene in the muscles?” said Chamberlain. “Currently, [adeno-associated virus] Vectors are the only known method to efficiently deliver genes throughout the body to muscle. “Chamberlain said there are several clinical trials underway to study gene delivery. However, he said the results are ‘not perfect.’
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