Pompe disease is a severe neuromuscular disorder caused by mutations in the lysosomal enzyme acid a-glucosidase (GAA), which result in the pathological accumulation of glycogen in all tissues. Enzyme replacement therapy (ERT) is available for Pompe disease, however it has limited efficacy, high immunogenicity, and fails to correct nervous tissue and muscle groups refractory to cross-correction. Using bioinformatics analysis and protein engineering, we developed secretable GAA transgenes for enhanced cross-correction of Pompe disease via adeno-associated virus (AAV) vector liver gene transfer.

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