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

• Main Authors: Carmen F Bjurström (Author), Michelle Mojadidi (Author), John Phillips (Author), Caroline Kuo (Author), Stephen Lai (Author), Georgia R Lill (Author), Aaron Cooper (Author), Michael Kaufman (Author), Fabrizia Urbinati (Author), Xiaoyan Wang (Author), Roger P Hollis (Author), Donald B Kohn (Author)
• Format: Book
• Published: Elsevier, 2016-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...