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New gene-therapy technique, MyoAAV, may improve treatment of certain muscle diseases

MyoAAV, a new gene-delivery vehicle, could make gene therapy for muscle diseases safer and better. Gene therapy-based treatments for genetic muscle disease that use harmless viruses to deliver healthy copies of damaged genes have shown promise in clinical trials, but require high doses of virus. In some cases, this can lead to adverse side effects, liver toxicity, or even death. Researchers at the Broad Institute of MIT and Harvard and Harvard University have engineered a gene-delivery virus that is more effective and can target muscle tissue at lower doses than viruses currently used in clinical trials, potentially reducing the risk of liver damage and other side effects.