New discoveries are expected to bring gene therapy to glaucoma

Glaucoma is the main cause of impaired vision and blindness, affecting 76 million patients worldwide

Glaucoma is caused by irreversible degeneration 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

CaMK II (calcium/calmodulin-dependent protein kinase II) pathway can regulate key cell processes and functions throughout the body, including retinal ganglion cells in the eye

In the process of searching for intervention methods, the researchers confirmed that activating the CaMKⅡ pathway with gene therapy has a protective effect on retinal ganglion cells

In the experiment, before the mice were poisoned (causing rapid damage to the cells) and after the optic nerve was squeezed (causing slower cell damage), the researchers passed the modified version of CaMKⅡ with mutated amino acids through the adeno-associated virus vector (American Food And the gene delivery system approved by the Drug Administration) was transferred to mouse primitive retinal ganglion cells and found that this gene therapy method can increase the activity of CaMKⅡ and effectively protect the retinal ganglion cells

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

In addition, the researchers also proved that increasing the activity of CaMKⅡ through gene therapy has a protective effect on retinal ganglion cells in glaucoma models based on high intraocular pressure or genetic defects

In the next step, researchers will test in larger animal models, paving the way for this therapy to enter clinical trials