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

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

Jing Zhou, Yue Wan, Michael G Anderson, Elia J Duh, Jonathan B Demb, Xinzheng Guo and Bo Chen
Molecular therapy
02/02/2026
DOI: 10.1016/j.ymthe.2026.01.036
PMID: 41635083

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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.
congenital glaucoma ischemic retinopathy retinal ganglion cells vision preservation neuroprotection CaMKII-based gene therapy

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