Article
Long-term effect of gene therapy on Leber's congenital amaurosis.
The New England journal of medicine - 14 May 2015
Bainbridge James W B, Mehat Manjit S, Sundaram Venki, Robbie Scott J, Barker Susie E, Ripamonti Caterina, Georgiadis Anastasios, Mowat Freya M, Beattie Stuart G, Gardner Peter J, Feathers Kecia L, Luong Vy A, Yzer Suzanne, Balaggan Kamaljit, Viswanathan Ananth, de Ravel Thomy J L, Casteels Ingele, Holder Graham E, Tyler Nick, Fitzke Fred W, Weleber Richard G, Nardini Marko, Moore Anthony T, Thompson Debra A, Petersen-Jones Simon M, Michaelides Michel, van den Born L Ingeborgh, Stockman Andrew, Smith Alexander J, Rubin Gary, Ali Robin R
Abstract excerpt
BACKGROUND: Mutations in RPE65 cause Leber's congenital amaurosis, a progressive retinal degenerative disease that severely impairs sight in children. Gene therapy can result in modest improvements in night vision, but knowledge of its efficacy in humans is limited. METHODS: We performed a phase 1-2 open-label trial involving 12 participants to evaluate the safety and efficacy of gene therapy with a recombinant...
Topics
- Adolescent
- Animals
- Child
- DNA, Complementary
- Dependovirus
- Disease Models, Animal
- Disease Progression
- Dogs