The Human Rhodopsin Kinase Promoter in an AAV5 Vector Confers Rod- and Cone-Specific Expression in the Primate Retina

Adeno-associated virus (AAV) has proven an effective gene delivery vehicle for the treatment of retinal disease. Ongoing clinical trials using a serotype 2 AAV vector to express RPE65 in the retinal pigment epithelium have proven safe and effective. While many proof-of-concept studies in animal mode...

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Detalles Bibliográficos
Main Authors: Boye, Shannon E., Alexander, John J., Boye, Sanford L., Witherspoon, Clark D., Sandefer, Kristen J., Conlon, Thomas J., Erger, Kirsten, Sun, Jingfen, Ryals, Renee, Chiodo, Vince A., Clark, Mark E., Girkin, Christopher A., Hauswirth, William W., Gamlin, Paul D.
Formato: Artigo
Idioma:Inglês
Publicado: Mary Ann Liebert, Inc. 2012
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Research Articles
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3472519/
https://ncbi.nlm.nih.gov/pubmed/22845794
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2012.125
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