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Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA
Ocular gene therapy with recombinant adeno-associated virus (AAV) has shown vector-mediated gene augmentation to be safe and efficacious in the retina in one set of diseases (retinitis pigmentosa and Leber congenital amaurosis (LCA) caused by RPE65 deficiency), with excellent safety profiles to date...
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| Опубликовано в: : | Mol Ther Nucleic Acids |
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| Главные авторы: | , , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
American Society of Gene & Cell Therapy
2018
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6023944/ https://ncbi.nlm.nih.gov/pubmed/30195768 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2018.05.014 |
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