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Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases
We examined the efficiency, specificity, and mutational signatures of zinc finger nucleases (ZFNs), transcriptional activator-like effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 systems designed to target the gene encoding the transcriptional...
Autores principales: | Bjurström, Carmen F, Mojadidi, Michelle, Phillips, John, Kuo, Caroline, Lai, Stephen, Lill, Georgia R, Cooper, Aaron, Kaufman, Michael, Urbinati, Fabrizia, Wang, Xiaoyan, Hollis, Roger P, Kohn, Donald B |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5023398/ https://www.ncbi.nlm.nih.gov/pubmed/28131278 http://dx.doi.org/10.1038/mtna.2016.52 |
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