CRISPR therapy is an experimental gene therapy that injects CRISPR therapy directly into the eyes of visually impaired patients. This has greatly improved their vision and allowed them to see more color.
NPR spoke with multiple study participants who all had severe Leber congenital blindness. Although their vision is not perfect, they are able to navigate safely and find small objects with less assistance. Michael Kalberer, one of the patients, stated that he was able to see color for first time in many years after receiving the treatment.
Kalberer, in addition to being able now to discern food on a plate and navigate it, shared his story about getting dinner with a friend. He was shocked at the sight of pink in the sky.
She says, "Yes, you can see the sunset." He told NPR that it was the sunset. We both smiled at one another. It was a wonderful moment.
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Carlene Knight, another patient, stated that she is able to see colors clearly and feels more secure walking around at work.
Patient Carlene Knight shared her story about how she worked before the treatment. Its nice. It's nice that I don't scare people.
Knight dyed her hair green in celebration of her ability to see her favorite color.
Gene therapies that are being tested for gene therapy are usually less direct. Researchers can remove cells from volunteers and treat them before reintroducing them to their bodies. NPR reports that the treatment for the retina was not possible so patients were given eye injections. Researchers announced Wednesday at the International Symposium on Retinal Degeneration that the treatment worked. They found that many of their patients with severe genetic vision impairment, Leber congenital aurosis, could see much better.
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Eric Pierce, an ophthalmologist at Harvard Medical School, said that they were thrilled to hear about the results. He is also helping to run the experiment. Early signs of efficacy are a sign that gene editing is working, and we were thrilled. This is the first time we have seen evidence that gene editing is working inside someone, and it is improving their vision function.
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