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Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice

Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated AAV2tYF-GRK1-RPGRco, to treat retinitis pigmentosa (RP) in patients with mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. The vector contains...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther Clin Dev
Main Authors: Song, Chunjuan, Conlon, Thomas J, Deng, Wen-Tao, Coleman, Kirsten E., Zhu, Ping, Plummer, Cayrn, Mandapati, Savitri, Hoosear, Mailin Van, Green, Kari B., Sonnentag, Peter, Sharma, Alok K., Timmers, Adrian, Robinson, Paulette M., Knop, David R., Hauswirth, William W., Chulay, Jeffrey D., Shearman, Mark S., Ye, Guo-jie
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc., publishers 2018
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6421992/
https://ncbi.nlm.nih.gov/pubmed/30280954
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2018.168
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