Monday, 26 October 2020

What is Gene therapy for sight ?

Human trials for genetic jab that lets blind mice see again.

A gene therapy that could restore the fading sight of the elderly is being tested on humans for the first time after positive results in blind mice.

It could be used to treat age-related macular degeneration, a common condition that usually first affects people in their 50s and 60s, scientists said.

It involves a one-time injection of a modified virus into the eye. This viral vector is altered to contain a synthetic gene that produces a protein that plays a critical role in the perception of light.

The gene at the heart of the treatment provides the code for cells in the eye to make a light-sensing protein called MCO1. This is known as an opsin, a molecule involved in the cascade of signals between cells that gives rise to sight. In a healthy eye, opsins are produced by cells known as rod and cone photoreceptors in the retina.

When activated by light, these photoreceptors send a signal through other retinal neurons, the optic nerve, and on to neurons in the brain.

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