TARGETING BCL11A DISTAL REGULATORY ELEMENTS WITH A CAS9-CAS9 FUSION FOR FETAL HEMOGLOBIN REINDUCTION

摘要

Provided herein are methods and compositions for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.