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Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates
- Source :
- Nature Communications, Vol 12, Iss 1, Pp 1-16 (2021)
- Publication Year :
- 2021
- Publisher :
- Nature Portfolio, 2021.
-
Abstract
- 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.
- Subjects :
- Science
Subjects
Details
- Language :
- English
- ISSN :
- 20411723
- Volume :
- 12
- Issue :
- 1
- Database :
- Directory of Open Access Journals
- Journal :
- Nature Communications
- Publication Type :
- Academic Journal
- Accession number :
- edsdoj.7a66d5fe4b0e825ff6e616f835cf
- Document Type :
- article
- Full Text :
- https://doi.org/10.1038/s41467-021-26744-4