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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Detaylı Bibliyografya
Asıl Yazarlar: 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.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: National Academy of Sciences 2013
Konular:
PNAS Plus
Online Erişim: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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