FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology, in particular to a gRNA molecule for mediating editing of promoter regions of hemoglobin gamma-1 (HBG1) subunit or hemoglobin gamma-2 (HBG2) subunit in a cell using Cas9 to increase expression of fetal hemoglobin in a subject, containing tracr and crRNA. Also disclosed is a composition for mediating editing of HBG1 or HBG2 promoter regions in a cell using Cas9. Invention also relates to a method of changing a cell in vitro in a target sequence or within 20 nucleotides of the target sequence in said cell and to a method of producing the cell.
EFFECT: invention makes it possible to efficiently edit promoter regions of hemoglobin gamma-1 (HBG1) subunit or hemoglobin gamma-2 (HBG2) subunit in a cell using Cas9 to increase expression of fetal hemoglobin in a subject.
34 cl, 41 dwg, 14 tbl, 9 ex
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