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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

Authors :
Helena Costa-Verdera
Fanny Collaud
Christopher R. Riling
Pauline Sellier
Jayme M. L. Nordin
G. Michael Preston
Umut Cagin
Julien Fabregue
Simon Barral
Maryse Moya-Nilges
Jacomina Krijnse-Locker
Laetitia van Wittenberghe
Natalie Daniele
Bernard Gjata
Jeremie Cosette
Catalina Abad
Marcelo Simon-Sola
Severine Charles
Mathew Li
Marco Crosariol
Tom Antrilli
William J. Quinn
David A. Gross
Olivier Boyer
Xavier M. Anguela
Sean M. Armour
Pasqualina Colella
Giuseppe Ronzitti
Federico Mingozzi
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

Subjects :
Science

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