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

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 vec...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc., publishers 2020
Assuntos:
Research Article—Clinical Development
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7047101/
https://ncbi.nlm.nih.gov/pubmed/31910043
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2019.297
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