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CRISPR RNA-guided FokI nucleases repair a PAH variant in a phenylketonuria model
The CRISPR/Cas9 system is a recently developed genome editing technique. In this study, we used a modified CRISPR system, which employs the fusion of inactive Cas9 (dCas9) and the FokI endonuclease (FokI-dCas9) to correct the most common variant (allele frequency 21.4%) in the phenylalanine hydroxyl...
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| Опубликовано в: : | Sci Rep |
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| Главные авторы: | , , , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
Nature Publishing Group
2016
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5081532/ https://ncbi.nlm.nih.gov/pubmed/27786189 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep35794 |
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