Seeing is believing: gene therapy shows promise for ocular disorders.

摘要

IN THE LAST FEW YEARS, A HANDFUL OF small but provocative clinical studies have demonstrated that gene therapy can improve vision in patients with a rare type of inherited blindness called type 2 Leber congenital amauro-sis (LCA2). Now that the clinical benefits from just one injection of the therapeutic gene have been shown to persist for several years, researchers are hoping to expand this approach to treat more common retinal diseases.