Article
Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa.
Human gene therapy - 1 Feb 2020
Dufour Valérie L, Cideciyan Artur V, Ye Guo-Jie, Song Chunjuan, Timmers Adrian, Habecker Perry L, Pan Wei, Weinstein Nicole M, Swider Malgorzata, Durham Amy C, Ying Gui-Shuang, Robinson Paulette M, Jacobson Samuel G, Knop David R, Chulay Jeffrey D, Shearman Mark S, Aguirre Gustavo D, Beltran William A
Abstract excerpt
Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated rAAV2tYF-GRK1-hRPGRco, to treat X-linked retinitis pigmentosa (XLRP) in patients with mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. The vector contains a codon-optimized human RPGR cDNA (hRPGRco) driven by a photoreceptor-specific promoter (G...
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