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Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases

1 Jan 2016

Abstract excerpt

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 repressor BCL11A, in human K562 cells and human CD34 + progenitor cells. ZFNs and TALENs were delivered as in vitro...

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Identifiers

PMID: 28131278
DOI: 10.1038/mtna.2016.52

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