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Gene Editing Approach for Haemoglobinopathies

  • Novel targets have been identified in the BCL11A enhancer for gene editing to increase HbF
  • A method has been established to generate an HPFH-like deletion in the beta globin cluster in haematopoietic progenitors and has been tested in NBSGW mice to test its potential to increase HbF in human erythroid cells.

Post Author: cscr-nahd