Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression

HIGHLIGHTS

• who: Panagiotis Antoniou from the (UNIVERSITY) have published the paper: Base-editing-mediated dissection of a u03b3-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression, in the Journal: (JOURNAL) of 20/Oct/2022
• what: The delivery of the CBEs and ABEs used in this study to clinically relevant HSPCs could be further optimized with regard to the promoter editing frequency in_vitro (e_g, in both BFU-E and CFU-GM), the base-editing efficiency in repopulating HSCs, and the fitness of the edited HSCs. The authors have analyzed sgRNA-independent off-target . . .
  

   

  

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