Advances in Gene Therapy Treatment for Blindness

Researchers using a gene therapy technique to treat one form of congenital blindness have reported some success. Two teams of scientists have used modified viruses injected into the eye to partially restore vision to people suffering from Leber congenital amaurosis, an illness that results in the degradation of the retina.

People with LCA slowly lose all vision, and typically are completely blind by their 20s or 30s. The modified virus used in the therapy carries a replacement gene to the retina, allowing it to produce a missing protein responsible for the disease.

Researcher Katherine A. High discusses the findings, published in The New England Journal of Medicine.



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