Gene Therapy Potentially Better Than ERT for Pompe, Study Shows

Gene Therapy Potentially Better Than ERT for Pompe, Study Shows

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Gene Therapy Potentially Better Than ERT for Pompe, Study Shows

Gene therapy given with chaperones — specific molecules known to stabilize the structure of proteins — was found to enhance the availability of acid alpha-glucosidase, known as GAA, the missing or defective enzyme in Pompe disease (PD), in a mouse model. The treatment subsequently eased disease symptoms, with the mice showing significantly improved muscle strength compared with both untreated animals (controls) and those given standard enzyme replacing therapy or ERT. These findings suggest that gene…

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