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AAV2/8‐LSPhGAA gene therapy with transduction of hepatocytes. With hepatic expression of GAA, protein then will act in other tissues (heart, muscle, and, at high doses, the brain) following uptake into tissue and protein processing. Ultimately, processed GAA will function in the lysosome. A benefit of liver specific GAA expression is suppression of anti‐GAA antibodies that can limit efficacy of gene therapy.
