In a study on inherited retinal degeneration, researchers developed a single gene therapy vector that prevented photoreceptor cell death and vision loss in a canine model of autosomal dominant retinitis pigmentosa caused by mutations in the rhodopsin (RHO) gene; retinal imaging and electroretinography analyses revealed that the treatment preserved retinal structure and function for more than 8 months, findings with potential implications for treating inherited retinal degeneration associated with RHO.
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Article #18-05055: "Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector," by Artur Cideciyan et al.
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Proceedings of the National Academy of Sciences