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Treatment Potential for LCA5-Associated Leber Congenital Amaurosis
PURPOSE: To determine the therapeutic window for gene augmentation for Leber congenital amaurosis (LCA) associated with mutations in LCA5. METHODS: Five patients (ages 6–31) with LCA and biallelic LCA5 mutations underwent an ophthalmic examination including optical coherence tomography (SD-OCT), ful...
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| Publié dans: | Invest Ophthalmol Vis Sci |
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| Auteurs principaux: | , , , , , , , , , , , |
| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
The Association for Research in Vision and Ophthalmology
2020
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| Sujets: | |
| Accès en ligne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7405811/ https://ncbi.nlm.nih.gov/pubmed/32428231 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1167/iovs.61.5.30 |
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