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Muscle‐specific, liver‐detargeted <scp>adeno‐associated virus</scp> gene therapy rescues Pompe phenotype in adult and neonate <scp>Gaa−/−</scp> mice

Authors :
P. Sellier
P. Vidal
B. Bertin
E. Gicquel
E. Bertil‐Froidevaux
C. Georger
L. van Wittenberghe
A. Miranda
N. Daniele
I. Richard
D. A. Gross
F. Mingozzi
F. Collaud
G. Ronzitti
Source :
Journal of Inherited Metabolic Disease.
Publication Year :
2023
Publisher :
Wiley, 2023.

Subjects

Subjects :
Genetics
Genetics (clinical)

Details

ISSN :
15732665 and 01418955
Database :
OpenAIRE
Journal :
Journal of Inherited Metabolic Disease
Accession number :
edsair.doi...........53b8b15b79520c612117082232b0431b
Full Text :
https://doi.org/10.1002/jimd.12625