Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa

Our collaborative successful gene replacement therapy using AAV vectors expressing a variant of human RPGR-ORF15 in two canine models provided therapeutic proof of concept for translation into human treatment. The ORF15 sequence contained within this AAV vector, however, has ORF15 DNA sequence varia...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: Deng, Wen-Tao, Dyka, Frank M., Dinculescu, Astra, Li, Jie, Zhu, Ping, Chiodo, Vince A., Boye, Sanford L., Conlon, Thomas J., Erger, Kirsten, Cossette, Travis, Hauswirth, William W.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2015
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4575541/
https://ncbi.nlm.nih.gov/pubmed/26076799
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.035
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