Comparative Evaluation of AAV8 and AAV9 Gene Therapy in Fabry Knockout (Gla−/y) and Symptomatic (G3STg/+Gla−/y) Murine Models
These findings demonstrate that AAV9 provides enhanced biodistribution and therapeutic efficacy compared to AAV8, supporting its potential for the treatment of Fabry disease.
Authors
- Dau-Ming Niu (ORCID: https://orcid.org/0000-0003-3962-1739)
- Ya-Ting Lee
- P Paul Liu
- Pei-Sin Chen
- Yunru Chen (ORCID: https://orcid.org/0000-0002-7410-0624)
- Fu-Pang Chang
Institutions
- National Yang Ming Chiao Tung University (TW)
- Taipei Veterans General Hospital (TW)
Publication Details
- Journal
- Genes
- Published
- 2025-06-29
- DOI
- https://doi.org/10.3390/genes16070766
- Citations
- 1
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.16
Funders
- Academia Sinica
- Taipei Veterans General Hospital
- National Science and Technology Council