Pei-Sin Chen
Current Organizations / Institutes
The organizations/institutes from the scholar's latest article
Research Subjects
Research Interests
Pei-Sin Chen has a total of 5 co-authors. Here are the top co-authors by number of shared publications:
- Dau-Ming Niu
Institute of Clinical Medicine, National Yang Ming Chiao Tung University
1 shared publication - Fu-Pang Chang
Institute of Clinical Medicine, College of Medicine, National Yang Ming Chiao Tung University, Taipei 112304, Taiwan
1 shared publication - Pao-Hsung Liu
Institute of Clinical Medicine, College of Medicine, National Yang Ming Chiao Tung University, Taipei 112304, Taiwan
1 shared publication - Ya-Ting Lee
Institute of Clinical Medicine, College of Medicine, National Yang Ming Chiao Tung University, Taipei 112304, Taiwan
1 shared publication - Yun-Ru Chen
Department of Pediatrics, Taipei Veterans General Hospital, Taipei 11217, Taiwan
1 shared publication
Latest Publications
- Open Access
Comparative Evaluation of AAV8 and AAV9 Gene Therapy in Fabry Knockout (Gla−/y) and Symptomatic (G3STg/+Gla−/y) Murine Models
Background: Fabry disease (FD) is an X-linked lysosomal storage disorder caused by mutations in the GLA gene, resulting in α-galactosidase A (α-Gal A) deficiency and progressive accumulation of globotriaosylceramide (Gb3). Current therapies, such as enzyme replacement and chaperone therapy, have limitations, including incomplete biodistribution and mutation-specific efficacy. Gene therapy using adeno-associated virus (AAV) vectors presents a promising alternative. Methods: In this study, we assessed the dose-dependent effects of AAV8 and AAV9 vectors encoding human GLA in Gla knockout (Gla−/y) mice by measuring α-Gal A activity and monitoring safety. To evaluate therapeutic efficacy, symptomatic Fabry mice (G3STg/+Gla−/y) were used. Results: AAV9-GLA produced significantly higher and more sustained enzyme activity than AAV8-GLA across plasma, liver, heart, and kidney. In symptomatic mice, AAV9-GLA achieved superior reductions in serum Gb3 and lyso-Gb3 levels, greater Gb3 clearance in heart and kidney tissues, and improved proteinuria. Anti-GLA IgG titers remained below threshold for the first four weeks and increased modestly by week eight, indicating a limited humoral immune response. No significant clinical signs or weight loss were observed in Gla−/y mice over the 3.5-month study period, supporting the favorable safety profile of AAV-mediated gene therapy. Conclusions: These findings demonstrate that AAV9 provides enhanced biodistribution and therapeutic efficacy compared to AAV8, supporting its potential for the treatment of Fabry disease.
Open AccessComparative Evaluation of AAV8 and AAV9 Gene Therapy in Fabry Knockout (Gla−/y) and Symptomatic (G3STg/+Gla−/y) Murine Models
Background: Fabry disease (FD) is an X-linked lysosomal storage disorder caused by mutations in the GLA gene, resulting in α-galactosidase A (α-Gal A) deficiency and progressive accumulation of globotriaosylceramide (Gb3). Current therapies, such as enzyme replacement and chaperone therapy, have limitations, including incomplete biodistribution and mutation-specific efficacy. Gene therapy using adeno-associated virus (AAV) vectors presents a promising alternative. Methods: In this study, we assessed the dose-dependent effects of AAV8 and AAV9 vectors encoding human GLA in Gla knockout (Gla−/y) mice by measuring α-Gal A activity and monitoring safety. To evaluate therapeutic efficacy, symptomatic Fabry mice (G3STg/+Gla−/y) were used. Results: AAV9-GLA produced significantly higher and more sustained enzyme activity than AAV8-GLA across plasma, liver, heart, and kidney. In symptomatic mice, AAV9-GLA achieved superior reductions in serum Gb3 and lyso-Gb3 levels, greater Gb3 clearance in heart and kidney tissues, and improved proteinuria. Anti-GLA IgG titers remained below threshold for the first four weeks and increased modestly by week eight, indicating a limited humoral immune response. No significant clinical signs or weight loss were observed in Gla−/y mice over the 3.5-month study period, supporting the favorable safety profile of AAV-mediated gene therapy. Conclusions: These findings demonstrate that AAV9 provides enhanced biodistribution and therapeutic efficacy compared to AAV8, supporting its potential for the treatment of Fabry disease.