Article
Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog.
Investigative ophthalmology & visual science - 1 Apr 2003
Narfström Kristina, Katz Martin L, Bragadottir Ragnheidur, Seeliger Mathias, Boulanger Ana, Redmond T Michael, Caro Lynette, Lai Chooi-May, Rakoczy P Elizabeth
Abstract excerpt
PURPOSE: To assess the efficacy of AAV-mediated gene therapy to restore vision in a large number of RPE65(-/-) dogs and to determine whether systemic and local side effects are caused by the treatment. METHODS: Normal RPE65 dog cDNA was subcloned into an rAAV vector under control of a cytomegalovirus promoter, and an AAV.GFP control vector was also produced with the titers 2 x 10(12) particles/mL and 2 x 10(10)...
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