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Gene therapy rescues cone function in congenital achromatopsia

The successful restoration of visual function with recombinant adeno-associated virus (rAAV)-mediated gene replacement therapy in animals and humans with an inherited disease of the retinal pigment epithelium has ushered in a new era of retinal therapeutics. For many retinal disorders, however, targ...

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Main Authors: Komáromy, András M., Alexander, John J., Rowlan, Jessica S., Garcia, Monique M., Chiodo, Vince A., Kaya, Asli, Tanaka, Jacqueline C., Acland, Gregory M., Hauswirth, William W., Aguirre, Gustavo D.
格式: Artigo
語言:Inglês
出版: Oxford University Press 2010
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC2883338/
https://ncbi.nlm.nih.gov/pubmed/20378608
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddq136
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