Genome editing of the BCL11A erythroid-specific enhancer in bone marrow-derived CD341 cells for the treatment of hemoglobinopathies

K. H. Chang,S. Tan, S. Smith, K. Chen, T. Sullivan,A. Reik, F. Urnov,E. Rebar,P. Gregory, O. Danos, H. Jiang

HUMAN GENE THERAPY(2015)

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