Cell found that gene therapy can provide neuroprotection and prevent glaucoma vision loss

A study supported by the National Eye Institute of the National Institutes of Health (NIH) showed that in a mouse model of glaucoma, a gene therapy can protect the optic nerve cells and preserve vision

Glaucoma is caused by irreversible neurodegeneration of the optic nerve, which is the axon bundle of retinal ganglion cells that transmits signals from the eye to the brain to produce vision

"Our research shows for the first time that activating the CaMKII pathway helps protect retinal ganglion cells from various injuries and in multiple glaucoma models," said the lead researcher of the study, Dr.

The CaMKII (calcium/calmodulin-dependent protein kinase II) pathway regulates key cellular processes and functions throughout the body, including the retinal ganglion cells of the eye

Using antibody markers of CaMKII activity, Chen's team found that when retinal ganglion cells are exposed to toxins or trauma caused by optic nerve crush, the CaMKII pathway signal is impaired, indicating that there is a gap between CaMKII activity and retinal ganglion cell survival.

In the process of searching for intervention, they found that activating the CaMKII pathway through gene therapy can protect retinal ganglion cells

In mice receiving gene therapy, 77% of retinal ganglion cells survived 12 months after toxic injury, while the survival rate of mice in the control group was 8%

Similarly, gene therapy to increase CaMKII activity demonstrated the protective effect of retinal ganglion cells in a glaucoma model based on increased intraocular pressure or genetic defects

According to the cell activity and visual cortex activity pattern measured by electroretinogram, the increased survival rate of retinal ganglion cells means that visual function is more likely to be preserved

Three visual-based behavioral tests also confirmed that the treated mice have sustained visual function

"If we make retinal ganglion cells more resistant and tolerant to the damage that causes glaucoma cell death, they may be able to survive longer and maintain their function," Chen concluded

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Guo X, Zhou J, Starr C, Mohns EJ, Li Y, Chen E, Yoon Y, Kellner CP, Tanaka K, Wang H, Liu W, LR, Demb JB, Crair MC, and Chen B.