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Gene therapy could lead to cure for genetic blindness:

This article was originally published in Clinica

Executive Summary

Gene therapy can improve the sight of patients with Leber's congenital amaurosis (LCA), a rare genetic eye disease that causes blindness, a study has shown. Researchers from the UCL Institute of Ophthalmology and Moorfields Eye Hospital, both in London, UK, delivered healthy copies of a gene that malfunctions in the disorder - RPE65 - into the patient's eyes using a harmless virus. One of the three patients in the trial had significantly improved night vision after the gene therapy, and no side-effects were seen. The team has already begun to trial the technique in younger patients, where they hope to achieve even better results. There are currently no effective treatments for LCA. The findings will appear in the May 22 issue of the New England Journal of Medicine.

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