FIELD: biotechnology.
SUBSTANCE: presented are: an isolated human haemopoietic stem cell and a precursor cell transduced by a lentiviral vector which contains a coding nucleotide sequence coding a functional version of a polypeptide selected from gp91phox, p22phox, p40phox, p47phox, p67phox and Rac2, under transcriptional control of a promoter sequence, which consists of a sequence of the miR223 promoter, that is SEQ ID NO 01. Also disclosed is a lentiviral vector for human gene therapy and a method for preparing a therapeutic cell preparation for use in treating or preventing chronic granulomatous disease.
EFFECT: invention can be used for treating or preventing chronic granulomatous disease in a patient.
9 cl, 6 dwg, 2 tbl, 1 ex
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