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Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells

To develop an effective and sustainable cell therapy for sickle cell disease (SCD), we investigated the feasibility of targeted disruption of the BCL11A gene, either within exon 2 or at the GATAA motif in the intronic erythroid-specific enhancer, using zinc finger nucleases in human bone marrow (BM)...

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Bibliografiske detaljer
Main Authors: Kai-Hsin Chang, Sarah E. Smith, Timothy Sullivan, Kai Chen, Qianhe Zhou, Jason A. West, Mei Liu, Yingchun Liu, Benjamin F. Vieira, Chao Sun, Vu P. Hong, Mingxuan Zhang, Xiao Yang, Andreas Reik, Fyodor D. Urnov, Edward J. Rebar, Michael C. Holmes, Olivier Danos, Haiyan Jiang, Siyuan Tan
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2017-03-01
Serier:Molecular Therapy: Methods & Clinical Development
Fag:
genome editing
gene therapy
BCL11A
hemoglobinopathies
fetal hemoglobin
fetal globin reactivation
zinc finger nucleases
Online adgang:http://www.sciencedirect.com/science/article/pii/S2329050117300037
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