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.
###
Article #18-05055: "Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector," by Artur Cideciyan et al.
MEDIA CONTACT: William Beltran, University of Pennsylvania, Philadelphia, PA; tel: 215-898-4692; e-mail: wbeltran@vet.upenn.edu
Journal
Proceedings of the National Academy of Sciences