Journal article
CaMKII-based gene therapy protects retinal ganglion cells in a broad range of disease: ischemic retinopathy and congenital glaucoma
Molecular therapy, Vol.34(5), pp.2770-2780
05/2026
DOI: 10.1016/j.ymthe.2026.01.036
PMCID: PMC13154303
PMID: 41635083
Abstract
Degeneration of retinal ganglion cells (RGCs) and their axons, as observed in ischemic retinopathy and glaucoma, results in irreversible vision loss. Elevated intraocular pressure (IOP) is a key factor driving these conditions, with existing treatments focused on IOP reduction often proving inadequate to halt disease progression. Here, we report that CaMKII-based gene therapy robustly protects RGCs and preserves visual function in mouse models of ischemic injury and congenital glaucoma, positioning CaMKII as a broad therapeutic target for retinal diseases driven by acute and chronic IOP elevation that damage RGCs.
Details
- Title: Subtitle
- CaMKII-based gene therapy protects retinal ganglion cells in a broad range of disease: ischemic retinopathy and congenital glaucoma
- Creators
- Jing Zhou - Sichuan UniversityYue Wan - Chinese Academy of Medical Sciences & Peking Union Medical CollegeMichael G Anderson - University of IowaElia J Duh - Johns Hopkins MedicineJonathan B Demb - Yale UniversityXinzheng Guo - Chinese Academy of Medical Sciences & Peking Union Medical CollegeBo Chen - Mount Sinai Hospital
- Resource Type
- Journal article
- Publication Details
- Molecular therapy, Vol.34(5), pp.2770-2780
- DOI
- 10.1016/j.ymthe.2026.01.036
- PMID
- 41635083
- PMCID
- PMC13154303
- NLM abbreviation
- Mol Ther
- ISSN
- 1525-0016
- eISSN
- 1525-0024
- Publisher
- Elsevier; CAMBRIDGE
- Grant note
- National Natural Science Foundation of China: 82371057 Nonprofit Central Research Institute Fund of Chinese Academy of Medical Sciences: 2022-RC180-01 CAMS Innovation Fund for Medical Sciences: 2022-I2M-1-024, 2022-I2M-2-004 NCTIB Fund for R&D Platform for Cell and Gene Therapy
This work was supported by the National Natural Science Foundation of China (82371057, to X.G.) , the Nonprofit Central Research Institute Fund of Chinese Academy of Medical Sciences (2022-RC180-01, to X.G.) , the CAMS Innovation Fund for Medical Sciences (2022-I2M-1-024 and 2022-I2M-2-004, to X.G.) , and the NCTIB Fund for R&D Platform for Cell and Gene Therapy (to X.G.) .
- Language
- English
- Electronic publication date
- 02/02/2026
- Date published
- 05/2026
- Academic Unit
- Molecular Physiology and Biophysics; Ophthalmology and Visual Sciences
- Record Identifier
- 9985139318402771
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