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Researchers report three patients maintained vision 1 year after gene therapy trial
N Engl J Med. 2009;361(7):725-727.
One year after the release of a study on the use of gene therapy to treat Leber's congenital amaurosis, the National Eye Institute has reported that three of the patients from the study have maintained their vision gains.
In a letter to the editor in the Aug. 13 issue of the New England Journal of Medicine, investigators in the trial also described visual improvements in one patient that helped her perform daily tasks.
The three patients were involved in a phase 1 trial supported by the National Eye Institute and conducted at the University of Pennsylvania, Philadelphia, and at the University of Florida, Gainesville. The trial was one of three separate trials investigating various RPE65 gene replacement strategies using an adenovirus-associated viral vector.
"These results are very significant because they represent one of the first steps toward the clinical use of gene therapy for an inherited form of blindness," Paul A. Sieving, MD, PhD, director of the NEI, said in a press release.