Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65

Leber congenital amaurosis is a group of inherited retinal dystrophies that cause severe sight impairment in childhood; RPE65-deficiency causes impaired rod photoreceptor function from birth and progressive impairment of cone photoreceptor function associated with retinal degeneration. In animal mod...

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Detalles Bibliográficos
Publicado en:Gene Ther
Main Authors: Georgiadis, A, Duran, Y, Ribeiro, J, Abelleira-Hervas, L, Robbie, S J, Sünkel-Laing, B, Fourali, S, Gonzalez-Cordero, A, Cristante, E, Michaelides, M, Bainbridge, J W B, Smith, A J, Ali, R R
Formato: Artigo
Idioma:Inglês
Publicado: Nature Publishing Group 2016
Assuntos:
Short Communication
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC5143366/
https://ncbi.nlm.nih.gov/pubmed/27653967
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2016.66
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