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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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Bibliografische gegevens
Gepubliceerd in:Graefes Arch Clin Exp Ophthalmol
Hoofdauteurs: Hufnagel, Robert B., Ahmed, Zubair M., Corrêa, Zélia M., Sisk, Robert A.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2012
Onderwerpen:
Online toegang: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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