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Gene therapy for Leber congenital amaurosis advances and future directions

BACKGROUND: Leber congenital amaurosis (LCA) is a congenital retinal dystrophy that results in significant and often severe vision loss at an early age. Comprehensive analysis of the genetic mutations and phenotypic correlations in LCA patients has allowed for significant improvements in understandi...

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Bibliographic Details
Published in:Graefes Arch Clin Exp Ophthalmol
Main Authors: Hufnagel, Robert B., Ahmed, Zubair M., Corrêa, Zélia M., Sisk, Robert A.
Format: Artigo
Language:Inglês
Published: 2012
Subjects:
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC8108009/
https://ncbi.nlm.nih.gov/pubmed/22644094
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00417-012-2028-2
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