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Peer-reviewed veterinary case report

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

Journal:
Molecular therapy : the journal of the American Society of Gene Therapy
Year:
2026
Authors:
Zhou, Jing et al.
Affiliation:
Eye Research Institute · United States

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.

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Original publication: https://pubmed.ncbi.nlm.nih.gov/41635083/