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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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Détails bibliographiques
Publié dans:Graefes Arch Clin Exp Ophthalmol
Auteurs principaux: Hufnagel, Robert B., Ahmed, Zubair M., Corrêa, Zélia M., Sisk, Robert A.
Format: Artigo
Langue:Inglês
Publié: 2012
Sujets:
Accès en ligne: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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