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TARGETING BCL11A DISTAL REGULATORY ELEMENTS FOR FETAL HEMOGLOBIN REINDUCTION
TARGETING BCL11A DISTAL REGULATORY ELEMENTS FOR FETAL HEMOGLOBIN REINDUCTION
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机译:针对胎儿血红蛋白再生的BCL11A远端调节元件
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摘要
Methods and compositions are provided herein for increasing fetal hemoglobin levels in cells by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions for the treatment of hemoglobinopathy by re-induction of fetal hemoglobin levels.
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