CRISPR Gene Therapy Shows Promise for Inherited Vision Loss in Children

A therapy using CRISPR gene editing has shown promise in treating Leber’s congenital amaurosis (LCA), the most common form of inherited vision loss in children. The therapy, called EDIT-101, involves injecting a single dose directly into the eye, and results from an early trial suggest that it can improve vision in people with LCA. The trial, published in the journal Nature Medicine, included 14 participants, and 11 of them experienced measurable improvements on at least one vision test after receiving the treatment. None of the participants experienced adverse side effects, although it’s important to note that the therapy cannot reverse the loss of cells that have already died. The next step is to test the therapy in a larger number of patients, including younger patients who may have even better outcomes.

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