Blind Mice

Retinitis pigmentosa is one of the most common causes of inherited vision deterioration in humans. Because the disease is caused by genetic mutation, and because there is no cure, scientists consider it to be a good candidate for gene therapy, specifically gene editing – where a mutation can be corrected in target cells (in this case retinal cells) with molecular tools that replace the errant DNA sequence. And now, proof-of-principle experiments for such a treatment have been carried out in mice, with the animals’ vision being successfully restored. Notice the thin, degraded retina of an untreated mouse with retinitis pigmentosa (left), and the thicker, healthier retina with abundant photoreceptor cells (red) after gene editing (right). While many steps exist between these results and the clinic, the work provides hope that such a molecular fix may one day restore vision in patients too.

Written by Ruth Williams

Ruth Williams

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Ruth is a freelance science journalist based in the US. She's a regular correspondent for The Scientist, and her work has appeared in The Lancet, Nature, Scientific American Mind, BBC Focus and elsewhere. Twitter @rooph

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• Image from work by Huan Qin, Wenliang Zhang, Shiyao Zhang & Yuan Feng and colleagues
• Institute of Visual Neuroscience and Stem Cell Engineering, Wuhan University of Science and Technology, Wuhan, China
• Image originally published with a Creative Commons Attribution 4.0 International (CC BY NC SA 4.0)
• Research published in the Journal of Experimental Medicine, March 2023