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

Molecular Therapy Methods and Clinical Development

Volumn 4, Issue , 2017, Pages 137-148

  Chang, Kai Hsin ^a   Smith, Sarah E ^a   Sullivan, Timothy ^a   Chen, Kai ^a   Zhou, Qianhe ^a   West, Jason A ^a   Liu, Mei ^a   Liu, Yingchun ^a   Vieira, Benjamin F ^a   Sun, Chao ^a   Hong, Vu P ^a   Zhang, Mingxuan ^a   Yang, Xiao ^a   Reik, Andreas ^b   Urnov, Fyodor D ^b   Rebar, Edward J ^b   Holmes, Michael C ^b   Danos, Olivier ^a   Jiang, Haiyan ^a   Tan, Siyuan ^a  

^a BIOGEN   (United States)

^b SANGAMO BIOSCIENCES INC   (United States)

Author keywords

BCL11A; fetal globin reactivation; fetal hemoglobin; gene therapy; genome editing; hemoglobinopathies; zinc finger nucleases

Indexed keywords

CD34 ANTIGEN; GLOBIN; HEMOGLOBIN S;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; AUTOLOGOUS STEM CELL TRANSPLANTATION; BCL11A GENE; BURST FORMING UNIT E; CELL DIFFERENTIATION; CELL MATURATION; CELLS; CONTROLLED STUDY; DNA END JOINING REPAIR; ELECTROPORATION; ENGRAFTMENT; ENHANCER REGION; ERYTHROID CELL; ERYTHROID PRECURSOR CELL; EX VIVO STUDY; FETUS; FRAMESHIFT MUTATION; GENE EDITING; GENE SEQUENCE; GLOBIN GENE; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; IN VITRO STUDY; INDEL MUTATION; MOUSE; NONHUMAN; PRIORITY JOURNAL; REVERSED PHASE HIGH PERFORMANCE LIQUID CHROMATOGRAPHY; SICKLE CELL ANEMIA; ULTRA PERFORMANCE LIQUID CHROMATOGRAPHY; UPREGULATION;

EID: 85015655808     PISSN: None     EISSN: 23290501     Source Type: Journal    
DOI: 10.1016/j.omtm.2016.12.009     Document Type: Article

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