Gene augmentation therapy restores vision and preserves photoreceptors in a mouse model of CNGA1-retinitis pigmentosa
To the best of our knowledge, this work demonstrates for the first time that a gene augmentation approach can restore vision and preserves photoreceptors in an animal model of CNGA1-RP. These findings pave the way for future development of gene augmentation therapy for patients with CNGA1-RP.
Authors
- Huixun Jia (ORCID: https://orcid.org/0000-0001-9341-5587)
- Xiaodong Sun (ORCID: https://orcid.org/0000-0001-5015-0945)
- Chenyue Hang (ORCID: https://orcid.org/0000-0003-2311-7255)
- Tianyuan Zhao
- Yazhi Wang (ORCID: https://orcid.org/0009-0009-0784-084X)
- Xiaoling Wan (ORCID: https://orcid.org/0000-0003-4583-8686)
- Tong Li (ORCID: https://orcid.org/0000-0002-2544-1254)
- Ting Zhang (ORCID: https://orcid.org/0000-0001-5482-6076)
- Fenghua Wang (ORCID: https://orcid.org/0000-0002-4575-1858)
- Wang Yf (ORCID: https://orcid.org/0000-0001-7737-1406)
- Yang Liu (ORCID: https://orcid.org/0000-0002-0581-0665)
- Lei Zhang (ORCID: https://orcid.org/0000-0002-5453-8215)
- Junran Sun (ORCID: https://orcid.org/0000-0001-6545-5223)
- Jieqiong Chen (ORCID: https://orcid.org/0000-0003-1673-6517)
- Zhixuan Chen (ORCID: https://orcid.org/0000-0001-8767-7177)
- Yidong Wu (ORCID: https://orcid.org/0000-0003-0232-5100)
Institutions
- Shanghai Medical College of Fudan University (CN)
- Shanghai Jiao Tong University (CN)
- Shanghai Eye Disease Prevention & Treatment Center (CN)
- Theranostics (New Zealand) (NZ)
- Shanghai First People's Hospital (CN)
- Center for High Pressure Science & Technology Advanced Research (CN)
- Wuyi University (CN)
Publication Details
- Journal
- Communications Medicine
- Published
- 2025-09-02
- DOI
- https://doi.org/10.1038/s43856-025-01108-x
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Natural Science Foundation of Shanghai
- Shanghai Science and Technology Development Foundation
- National Natural Science Foundation of China
- Science and Technology Commission of Shanghai Municipality