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Treating glaucoma with gene therapy

Glaucoma is a serious eye condition that, if not treated in time and properly, can cause deterioration of the optic nerve, the "connecting cable" between the eye and the brain. If the functionality of this precious link is lost, the visual message generated by the retina cannot reach the brain and consequently there can be no visual perception.

The optic nerve consists of the extension of the axons of ganglion cells, nerve cells found in the innermost part of the retina. These are part of the central nervous system and when they are damaged they are unable to regenerate and "heal". For this reason, when the functionality of the optic nerve is lost, irreversible blindness results.

Today there are several lines of research aimed at developing therapies capable of regenerating the optic nerve to restore lost vision. Researchers at Cambridge University's Department of Clinical Neuroscience have identified a protein, protrudin, which appears to play a key role in the growth of axons in the developing central nervous system and in the regrowth of damaged peripheral nervous system axons (which , unlike the neurons of the central nervous system, they have good regenerative and functional recovery capacities). Consequently, the researchers decided to induce the production of protrudin even in ganglion cells unable to regenerate and analyze the consequences of this procedure.

The experiment was carried out in mouse ganglion cells, in which the synthesis of protrudin was induced by gene therapy, ie by inserting a viral vector containing the necessary genetic information. The study showed that the production of protrudin in ganglion cells induces a regenerative effect on the optic nerve damaged by glaucoma. These results, recently published in the prestigious scientific journal Nature Communications, offer concrete hope that the restoration of vision lost due to optic neuropathies such as glaucoma will soon become a concrete reality. More studies will be needed, says Dr. Veselina Petrova, head of the research, but the road to treating blindness is now open.

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