In the last few years, a handful of small but provocative clinical studies have demonstrated that gene therapy can improve vision in patients with a rare type of inherited blindness called type 2 Leber congenital amaurosis (LCA2). Now that the clinical benefits from just one injection of the therapeutic gene have been shown to persist for several years, researchers are hoping to expand this approach to treat more common retinal diseases.
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Control treatment failed to prevent retinal degeneration (pink) in a mouse model for retinal disease (top); an eye treated with nanoparticles carrying a gene for glial cell line–derived neurotrophic factor (GDNF) was protected against retinal damage (bottom).
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