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Redirecting Gene Therapy Restores Sight

By targeting rhodopsin genes to neurons, scientists help blind mice see.

Written byAmanda B. Keener
| 1 min read

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WIKIMEDIA, SANTIAGO RAMON Y CAJAL

A team of researchers at the University of Manchester enabled blind mice with advanced retinal degeneration to see by delivering the gene for a light-sensing protein into nervous cells within their retinas, according to a study published last month (July 30) in Current Biology.

“This is the most effective example yet of the use of genetic therapy to treat advanced retinal degeneration,” Robin Ali, who studies gene therapy at University College London and was not involved in the work told New Scientist.

Researchers have previously used virus-based gene therapies to replace lost or defective genes in rods and cones, the photoreceptor cells of the retina. But for the present study, the team set its sights on ganglion and bipolar cells, which transduce signals from rods ...

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