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Gene Editing for Vision Loss

CRISPR used to treat eye disease in seven patients

Using harmless viruses carrying CRISPR gene editors, doctors treated seven patients who suffer vision loss due to Leber congenital amaurosis (LCA). Some of the patients reported significant improvement in their sight in as little as a month - they were able to see colors and shapes that were almost entirely occluded before the treatment.

This was the first time researchers have injected CRISPR editors directly into the patient. Typically, cells are removed from the patient, modified with CRISPR, and then reinserted. In this case, the CRIPR editor itself was directly administered since retinal cells cannot be taken out and reinserted into patients.

Each patient was given a varied dose of the virus in only one eye, in case they encountered any issues. While at least two of the patients have not experienced improvements yet, none of the patients had any significant side effects, and a few of the patients have seen dramatic results.

Researchers are planning an expanded trial with more patients. For more, head over to NPR.