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

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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Bibliographic Details
Main Authors: Carmen F Bjurström, Michelle Mojadidi, John Phillips, Caroline Kuo, Stephen Lai, Georgia R Lill, Aaron Cooper, Michael Kaufman, Fabrizia Urbinati, Xiaoyan Wang, Roger P Hollis, Donald B Kohn
Format: Article
Language:English
Published: Elsevier 2016-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
zinc finger nucleases
TALENs
CRISPR/Cas9
nonhomologous end joining
BCL11A
fetal hemoglobin
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117300690

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http://www.sciencedirect.com/science/article/pii/S2162253117300690

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