Gene therapy to prevent immune response to enzyme replacement therapy

Posted by & filed under Part 16: LYSOSOMAL DISORDERS, Treatment.

Enhanced Response to Enzyme Replacement Therapy in Pompe Disease after the Induction of Immune Tolerance
Baodong Sun, Andrew Bird, Sarah P. Young, Priya S. Kishnani, Y.-T. Chen, and Dwight D. Koeberl
Am. J. Hum. Genet., 81:1042-1049, 2007

In this puplication, investigators from Duke University administered an AAV encoding alpha-glucosidase(GAA) to GAA-knockout mice. This prevented the antibody response to subsequently administered enzyme replacement therapy.


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Philippe Campeau, MD
Resident in Medical Genetics at McGill University
OMMBID Blog Administrator

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