Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease

Inherited retinal degenerations cause progressive loss of photoreceptor neurons with eventual blindness. Corrective or neuroprotective gene therapies under development could be delivered at a predegeneration stage to prevent the onset of disease, as well as at intermediate-degeneration stages to slo...

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Dettagli Bibliografici
Pubblicato in:Proc Natl Acad Sci U S A
Autori principali: Beltran, William A., Cideciyan, Artur V., Iwabe, Simone, Swider, Malgorzata, Kosyk, Mychajlo S., McDaid, Kendra, Martynyuk, Inna, Ying, Gui-Shuang, Shaffer, James, Deng, Wen-Tao, Boye, Sanford L., Lewin, Alfred S., Hauswirth, William W., Jacobson, Samuel G., Aguirre, Gustavo D.
Natura: Artigo
Lingua:Inglês
Pubblicazione: National Academy of Sciences 2015
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PNAS Plus
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4629324/
https://ncbi.nlm.nih.gov/pubmed/26460017
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1509914112
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