Toxicity and Efficacy Evaluation of an AAV Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa

Valerie Dufour,Artur V. Cideciyan,Guo-jie Ye,Chunjuan Song,Adrian M. Timmers,Perry L. Habecker,Wei Pan,Nicole M. Weinstein,Malgorzata Swider,Amy C. Durham,Gui-Shuang Ying,Paulette M. Robinson,Samuel G. Jacobson,David R. Knop,Jeffrey D. Chulay,Mark S. Shearman,Gustavo D. Aguirre,William A. Beltran

Toxicity and Efficacy Evaluation of an AAV Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa

2020

Valerie Dufour
Artur V. Cideciyan
Guo-jie Ye
Chunjuan Song
Adrian M. Timmers
Perry L. Habecker
Wei Pan
Nicole M. Weinstein
Malgorzata Swider
Amy C. Durham
Gui-Shuang Ying
Paulette M. Robinson
Samuel G. Jacobson
David R. Knop
Jeffrey D. Chulay
Mark S. Shearman
Gustavo D. Aguirre
William A. Beltran

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 pigment...

Keywords:

Retinitis
Virus
Biology
Molecular biology
X-linked retinitis pigmentosa
Recombinant DNA
Toxicity
Virology
Retinal degeneration
Genetic enhancement
canine model
Adeno-associated virus

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