Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

Leber congenital amaurosis (LCA) associated with retinal pigment epithelium-specific protein 65 kDa (RPE65) mutations is a severe hereditary blindness resulting from both dysfunction and degeneration of photoreceptors. Clinical trials with gene augmentation therapy have shown partial reversal of the...

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Bibliographische Detailangaben
Hauptverfasser: Cideciyan, Artur V., Jacobson, Samuel G., Beltran, William A., Sumaroka, Alexander, Swider, Malgorzata, Iwabe, Simone, Roman, Alejandro J., Olivares, Melani B., Schwartz, Sharon B., Komáromy, András M., Hauswirth, William W., Aguirre, Gustavo D.
Format: Artigo
Sprache:Inglês
Veröffentlicht: National Academy of Sciences 2013
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PNAS Plus
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3568385/
https://ncbi.nlm.nih.gov/pubmed/23341635
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1218933110
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