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...

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Vydáno v:Mol Ther Nucleic Acids
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2016
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5023398/
https://ncbi.nlm.nih.gov/pubmed/28131278
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2016.52
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