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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...
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| Veröffentlicht in: | Clin Ophthalmol |
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| Hauptverfasser: | , , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Dove
2020
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7671481/ https://ncbi.nlm.nih.gov/pubmed/33223822 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/OPTH.S231804 |
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