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Gene Therapy For Blindness Improves Vision, Safety Study Indicates

Posted on: 8 September 2008, source: ScienceDaily
All three people who received gene therapy at the University of Florida to treat a rare, incurable form of blindness have regained some of their vision, according to a paper published online today in Human Gene Therapy.
The patients with a type of hereditary blindness called Leber congenital amaurosis type 2, volunteered to test the safety of an experimental gene-transfer technique in a phase 1 clinical research study. In this form of LCA disease, photoreceptor cells cannot respond to light because a gene called RPE65 does not properly produce a protein necessary for healthy vision. In the study, researchers used an adeno-associated virus to deliver RPE65 to a small area of the retina. Not only were there no ill effects other than routine postsurgical soreness, the subjects said the vision in their treated eyes was slightly improved in dim lighting conditions.