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Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical Evidence
Subretinal gene therapy trials began with the discovery of RPE65 variants and their association with Leber congenital amaurosis. The RPE65 protein is critical for the normal functioning of the visual phototransduction cascade. RPE65 gene knockout animal models were developed and showed similar disea...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Dove
2020
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7671481/ https://www.ncbi.nlm.nih.gov/pubmed/33223822 http://dx.doi.org/10.2147/OPTH.S231804 |