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- Title
Visible improvement.
- Authors
Nicholson, Craig
- Abstract
The article reports on the two independent studies which show tremendous improvements in the vision of legally blind individuals after gene therapy. The subjects are all sick of Leber's congenital amaurosis (LCA), a condition caused by a defect in a gene (RPE65) that encodes an enzyme required for rhodopsin production in the retina. Studies used genetically engineered viruses to deliver normal copies of RPE65 gene. Patients showed improved visual acuity and increased light-sensitivity.
- Subjects
GENETIC research; BLINDNESS; PEOPLE with visual disabilities; VISUAL acuity; GENE therapy; RHODOPSIN; RETINA
- Publication
Nature Reviews Neuroscience, 2008, Vol 9, Issue 6, p410
- ISSN
1471-003X
- Publication type
Article
- DOI
10.1038/nrn2409