Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector
Year: 2018
Type: article
Abstract: Significance A number of gene-augmentation strategies are entering clinical trials for the treatment of inherited retinal blindness. Gene therapy for autosomal dominant diseases faces significant obstacles that include allelic heterogeneity and the potential need to silence the mutated gene. Here we show that a single-gene therapy vector that combines knockdown of the causative gene with its replacement by a resistant wild-type copy can prevent photoreceptor cell death and vision loss in a canine model of autosomal dominant retinitis pigmentosa.
Authors: Artur V. Cideciyan, Raghavi Sudharsan, Valérie L. Dufour, Michael T. Massengill, Simone Iwabe, Malgorzata Swider, Brianna Lisi, Alexander Sumaroka, Luis Felipe Marinho, Tatyana Appelbaum, Brian Rossmiller, William W. Hauswirth, Samuel G. Jacobson, Alfred S. Lewin, Gustavo D. Aguirre, William A. Beltran
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: bronze
Awards: G1390634746, G1481536873, Vision Research Core, G2000954033, G2606015775, Development of Allele Independent Gene Therapy Strategies for Autosomal Dominant Retinitis Pigmentsa, G495111706, G5246180421, Translational Gene Therapy for Rhodopsin Autosomal Dominant Retinitis Pigmentosa, G9000024960, G9000256854, Models for Therapy of Hereditary Retinal Degeneration, P30 Core Grant For Vision Research
OpenAlex ID: W2888444358