Multiplex base editing of BCL11A regulatory elements to treat sickle cell disease
We performed WES analysis in SCD CD34+ HSPCs (3 donors) that were preactivated for 2 days, then electroporated with a combination of base editors mRNA or Cas9 nuclease RNP and sgRNA targeting the +58-kb and +55-kb BCL11A enhancers, and then cultured for 6 additional days. Cells electroporated with TE buffer only or with epigenome editors mRNA only were used as controls.