Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

BACKGROUND: Mutations in RPE65 cause Leber’s congenital amaurosis, a progressive retinal degenerative disease that severely impairs sight in children. Gene therapy can result in modest improvements in night vision, but knowledge of its efficacy in humans is limited. METHODS: We performed a phase 1–2...

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Udgivet i:N Engl J Med
Main Authors: Bainbridge, J.W.B., Mehat, M.S., Sundaram, V., Robbie, S.J., Barker, S.E., Ripamonti, C., Georgiadis, A., Mowat, F.M., Beattie, S.G., Gardner, P.J., Feathers, K.L., Luong, V.A., Yzer, S., Balaggan, K., Viswanathan, A., de Ravel, T.J.L., Casteels, I., Holder, G.E., Tyler, N., Fitzke, F.W., Weleber, R.G., Nardini, M., Moore, A.T., Thompson, D.A., Petersen-Jones, S.M., Michaelides, M., van den Born, L.I., Stockman, A., Smith, A.J., Rubin, G., Ali, R.R.
Format: Artigo
Sprog:Inglês
Udgivet: 2015
Fag:
Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4497809/
https://ncbi.nlm.nih.gov/pubmed/25938638
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1414221
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