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Neovascular, or wet, age-related macular degeneration causes central vision loss and represents a major health problem in elderly people, and is currently treated with frequent intraocular injections of anti-VEGF protein. Gene therapy might enable long-term anti-VEGF therapy from a single treatment. We tested the safety of rAAV.sFLT-1 in treatment of wet age-related macular degeneration with a single...
The site-specific integrase from bacteriophage ϕC31 functions in mammalian cells and is being applied for genetic engineering, including gene therapy. The ϕC31 integrase catalyzes precise, unidirectional recombination between its 30–40-bp attP and attB recognition sites. In mammalian cells, the enzyme also mediates integration of plasmids bearing attB into native sequences that have partial sequence...
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