The first participant has already begun the treatment, which aims to restore the vision of patients with severe blindness caused by a rare genetic disease. Physicians at Portland, Ore.-based Casey Eye Institute reportedly injected into the patient’s eye several microscopic droplets containing a harmless virus that will carry out the CRISPR editing mechanisms.
The researchers reportedly expect to know in the next few weeks whether the new technique is working. If it is, they should be able to predict how effective the in vivo application will be within two or three months.
“We’re really optimistic that this has a good chance of being effective,” Eric Pierce, MD, PhD, a professor of ophthalmology at Boston-based Harvard Medical School and director of the Inherited Retinal Disorders Service at Massachusetts Eye and Ear, who is leading the study, told NPR. “We’re helping open, potentially, an era of gene editing for therapeutic use that could have impact in many aspects of medicine.”
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