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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
- Source :
- Journal of Inherited Metabolic Disease.
- Publication Year :
- 2023
- Publisher :
- Wiley, 2023.
- Subjects :
- Genetics
Genetics (clinical)
Subjects
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