CaMKII-based gene therapy protects retinal ganglion cells in a broad range of disease: ischemic retinopathy and congenital glaucoma

Molecular Therapy 2026 Open access

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Authors: Jing Zhou, Yue Wan, Michael G. Anderson, Elia J. Duh, Jonathan B. Demb, Xinzheng Victor Guo, Bo Chen
DOI: 10.1016/j.ymthe.2026.01.036
Cited by: 1
References: 32

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.

Fields of Study & Subjects

Retinal Development and Disorders Glaucoma and retinal disorders Retinal Diseases and Treatments

Publication details

Journal / source
Molecular Therapy
Publisher
Elsevier BV
Volume
34
Issue
5
Pages
2770–2780
Publication date
3 February 2026
Language
EN