Functional and Structural Recovery of the Retina after Gene Therapy in the RPE65 Null Mutation Dog
Investigative Ophthalmology & Visual Science2003Vol. 44(4), pp. 1663–1663
Citations Over TimeTop 10% of 2003 papers
Kristina Narfstro ̈m, Martin L. Katz, Ragnheiður Bragadóttir, Mathias W. Seeliger, Ana Boulanger, T. Michael Redmond, Lynette Caro, Chooi‐May Lai, P. Elizabeth Rakoczy
Abstract
In 10 of 11 treated RPE65(-/-) eyes, gene transfer resulted in development of vision, both subjectively apparent by loss of nystagmus, and objectively recorded by ERG. Structurally, there was reversal of lipid droplet accumulation in the RPE. Uveitis developed in 75% of the transgene-treated eyes, a complication possibly due to an immunopathogenic response to the RPE65 molecule.
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