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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Dades bibliogràfiques
Publicat a:Proc Natl Acad Sci U S A
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: National Academy of Sciences 2015
Matèries:
PNAS Plus
Accés en línia: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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