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Some Improvement for Two

Two patients with Leber congenital amaurosis have regained some vision — including being able to make out color — following a CRISPR-based treatment, NPR reports. It notes, though, that the treatment didn't work for everyone the study.

Leber congenital amaurosis is an inherited form of blindness, and in the study, sponsored by Editas Medicine, researchers targeted the CEP290 gene with a CRISPR-based treatment. As the Associated Press previously reported, the approach used here delivers the gene-editing machinery to patients' cells, rather than performing the edits outside the body as other CRISPR-based treatments have done.

According to Editas, preliminary results provide a proof of concept for their treatment. They report that two of the three patients in the mid-dose cohort exhibited signs of improvement on three tests gauging visual acuity, light sensitivity, and navigation.

"We're thrilled to see early signs of efficacy because that means gene editing is working. This is the first time we're having evidence that gene editing is functioning inside somebody and it's improving — in this case — their visual function," Massachusetts Eye & Ear's Eric Pierce, who is working on the study, tells NPR.

However, Bloomberg notes that Editas' stock fell upon the results, with FierceBiotech adding that the market doesn't seem to think the released data are "enough to form a full opinion on whether the gene editing therapy will work or not."