Clinical Characteristics and Natural History of RHO-Associated RP
A multi-center, chart review of 100 patients with RHO-associated adRP. Based on central visual fields, the optimal window of intervention is
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RHO
Disease Category: autosomal dominant
Patient Population: unknown
Treatment Options: None known
Strategies to Preserve Eye Health: None known
Active Clinical Trials: 3 recruiting
Institution(s) Conducting Research: University of Michigan, UPMC Eye Center Pittsburgh, UCSD, ue Anschutz-Rogers Eye Center, University of Colorado, Duke Eye Center,
A FACE OF RP
Michael
Naugatuck, CT
IN THE NEWS
Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector
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Artur V. Cideciyan, Raghavi Sudharsan, Valérie L. Dufour, Michael, T. Massengill, Simone Iwabe, Malgorzata Swider, BriannaLisi, Alexander Sumaroka, Luis Felipe Marinho, Tatyana Appelbaum, Brian Rossmiller, William W. Hauswirth, Samuel G.Jacobson, Alfred S. Lewin, Gustavo D. Aguirre, William A. Beltran | Edited by Jeremy Nathans, Johns Hopkins University, Baltimore, MD | August 20, 2018 |
115 (36) E8547-E8556
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.
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