Longevity Briefs: Gene Editing Can Restore Vision In Mice With Retinal Disease

Posted on 5 November 2020

Longevity briefs provides a short summary of a novel research, medicine, or technology that caught the attention of our researchers in Oxford, due to its potential to improve our health, wellbeing, and longevity.

Why is this research important: In the retina of the eye, a molecule called retinal plays a key role in our vision. Retinal changes shape when exposed to light, leading to a series of chemical changes that eventually result in electrical signals in the brain, allowing us to perceive images. To continue to function, however, retinal must be able to return to its original shape. Mutations in a gene called Rpe65 can prevent this from happening, resulting in blindness.

What did the researchers do: In this study, researchers used a new generation of CRISPR technology called ‘base editing’ in order to correct mutations in the Rpe65 gene in adult mice with inherited retinal disease. This technology allows for single nucleic acids within the DNA to be edited without breaking the DNA molecule, which reduces the risk of off-target modifications.

Key takeaway(s) from this research: The treatment was able to restore the mice’s vision to near normal levels. According to the authors, this constitutes ”the most successful rescue of blindness to date using genome editing”. There are over 250 genes that can mutate to cause inherited retinal disease and blindness. This study provides an exciting proof of concept that these diseases could one day be cured with gene editing.

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