Immunomodulatory, liver depot gene therapy for Pompe disease.


Pompe disease is caused by mutations in acid alpha glucosidase (GAA) that causes accumulation of lysosomal glycogen affecting the heart and skeletal muscles, and can be fatal. Enzyme replacement therapy (ERT) with recombinant human GAA (rhGAA) improves muscle function by reducing glycogen accumulation. Limitations of ERT include a short half-life and the… (More)
DOI: 10.1016/j.cellimm.2017.12.011


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