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New gene-delivering workhorse could make gene therapy safer, more effective for muscle diseases

Posted on: 13 September 2021, source: Broad Institute of MIT and Harvard
Genetic muscle diseases lead to progressive muscle wasting and often early death, with few treatment options and no cure. Some gene therapies that use a harmless virus to deliver a functioning copy of a disease-causing gene to cells have shown promise in clinical trials for a subset of muscular dystrophies, but have faced challenges. High doses of the gene-carrying virus are needed to reach the muscles throughout the body and the viruses used in these trials often end up in the liver more than in the muscle. This has led to high levels of the virus in the liver, severe adverse side effects, and even death in some trial participants. Researchers at the Broad Institute of MIT and Harvard and Harvard University have developed a new family of adeno-associated viruses (AAVs) - the gene-delivering workhorse of gene therapy - that improve targeting of the muscle tissue, which could be safer and more effective for patients with muscle disease.