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Advanced filters: Author: Umut Cagin Clear advanced filters
  • Pompe disease is currently treated with enzyme replacement therapy (ERT) with recombinant human acid alpha-glucosidase (GAA). Here, the authors show hepatic-directed gene therapy with AAV vectors enhances GAA bioavailability compared with ERT, resulting in improved rescue of the disease phenotype in mice and broad enzyme distribution in mice and non-human primates.

    • Helena Costa-Verdera
    • Fanny Collaud
    • Federico Mingozzi
    ResearchOpen Access
    Nature Communications
    Volume: 12, P: 1-16