A lot of work is being done in the fields of CRISPR gene-editing, and recently the revolutionary therapy was used to partially restore the vision in patients with a rare genetic disorder.
55-year-old Carlene Knight is now able to move around her call center office without the aid of her walking stick, easily locate objects, and can see colors much more vividly now.
43-year-old Michael Kalberer also learned he could see colors again while on the dancefloor of his cousin’s wedding, and he has gradually regained some of the peripheral field of vision.
They were two of seven patients who received CRISPR injected directly into their eye, a procedural method never before done with CRISPIR, which normally involves removing cells, editing them in vitro, and injecting them back where they were found.
Both patients are far from cured of their Leber congenital amaurosis, or LCA, a rare mutation in the retina, but they’re no longer legally blind, and while the treatment failed to work for some patients during the three- and nine-month followups, no side effects were reported.
“I’ve always loved colors,” said Knight, who lives near Portland. “Since I was a kid it’s one of those things I could enjoy with just a small amount of vision. But now I realize how much brighter they were as a kid because I can see them a lot more brilliantly now; it’s just amazing.”
Mark Pennesi, a professor of ophthalmology holding an MD and a Ph.D. from the Baylor College of Medicine, presented the results at a recent symposium, calling it “a really amazing technology and very powerful,” according to NPR.
-Goodnewsnetwork
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