Resistance to modulation of in vivo gene expression by genomic editing
Enhancer editing is widely proposed as a strategy to modulate gene dosage therapeutically. EKLF/KLF1, a master erythroid transcription factor, is an attractive target because haploinsufficiency is benign and associated with hereditary persistence of fetal hemoglobin (HPFH). Here we test whether enhancer disruption can...
A. Planutis, Sanjana Pillay, Babu Mia et al.
· Communications Biology · 0 citations