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Edits Eyed

An Australian team of researchers has used the CRISPR/Cas9 approach to edit a gene in mouse eye, New Scientist reports. This, it adds, suggests that CRISPR-based approaches might be harnessed to treat inherited eye diseases and save patients' sight.

"We are certainly very excited by the potential," the University of Tasmania's Alex Hewitt tells New Scientist.

He and his colleagues used two adeno-associated virus 2 constructs, one bearing Cas9 and the other bearing guide RNA, to deliver the CRISPR/Cas9 system to the retinal cells of a Thy1-YFP transgenic mouse, as they report in Investigative Opthalmology and Visual Science. The guide RNA was designed against either YFP or against LacZ, as a control. Using this approach, Hewitt and his colleagues found an 84 percent reduction in YFP-positive cells after treatment.

This is a "really encouraging result," New Scientist says, noting that modifying just 10 percent of retinal cells might be enough to save patients' vision. It adds that companies like Editas Medicine are also interested in the approach — Editas plans to soon being testing a treatment for Leber congenital amaurosis that involves turning off a certain gene.

However, New Scientist also says the CRISPR approach needs to be refined to be more efficient if it's to be applied to eye diseases.