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

• Autores principales: Carmen F Bjurström (Autor), Michelle Mojadidi (Autor), John Phillips (Autor), Caroline Kuo (Autor), Stephen Lai (Autor), Georgia R Lill (Autor), Aaron Cooper (Autor), Michael Kaufman (Autor), Fabrizia Urbinati (Autor), Xiaoyan Wang (Autor), Roger P Hollis (Autor), Donald B Kohn (Autor)
• Formato: Libro
• Publicado: Elsevier, 2016-01-01T00:00:00Z.
• Materias: article
• Acceso en línea: 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...