A Novel AAV Capsid-Mediated <i>RS1</i> Gene Therapy Restored Retinal Function to Wild-Type Levels in <i>Rs1</i><sup>R213W</sup> Mouse Model
The novel AAV capsid-mediated RS1 gene therapy effectively improved retinal structure and function while downregulating inflammation in Rs1R213W mice. These results provide a robust foundation for future clinical trials on XLRS gene therapy, offering the potential to improve the vision and quality of life of patients.
Authors
- Huixin Liu (ORCID: https://orcid.org/0000-0001-7073-4366)
- Duanyang Wang
- Xuan Zou (ORCID: https://orcid.org/0000-0002-1145-5508)
- Xing Wei (ORCID: https://orcid.org/0000-0002-5025-3941)
- Ruifang Sui (ORCID: https://orcid.org/0000-0002-6659-0926)
- Yunyu Zhou
- Yingying Fu (ORCID: https://orcid.org/0000-0002-3503-8610)
- Sisi Ma (ORCID: https://orcid.org/0000-0003-2956-6844)
- Haoliang Cui (ORCID: https://orcid.org/0009-0007-8362-0967)
- Huihui Han
- Zixi Sun (ORCID: https://orcid.org/0000-0002-8238-2115)
- Wei Li (ORCID: https://orcid.org/0000-0002-2897-649X)
- Shan Zhang (ORCID: https://orcid.org/0000-0002-1530-7561)
- Cheng Wang (ORCID: https://orcid.org/0000-0002-1834-2119)
- Yamei Li
- Xiaoxu Han (ORCID: https://orcid.org/0000-0003-1427-8428)
- Hui Li (ORCID: https://orcid.org/0000-0002-3565-1773)
- Wuyi Li (ORCID: https://orcid.org/0000-0002-2511-0316)
Institutions
- Chinese Academy of Medical Sciences & Peking Union Medical College (CN)
- Peking Union Medical College Hospital (CN)
- Beijing Biocytogen (China) (CN)
Publication Details
- Journal
- Investigative Ophthalmology & Visual Science
- Published
- 2025-04-15
- DOI
- https://doi.org/10.1167/iovs.66.4.37
- Citations
- 1
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.81
Funders
- Chinese Academy of Medical Sciences
- Peking Union Medical College Hospital
- Peking Union Medical College
- Beijing Nova Program