FIELD: biotechnology.
SUBSTANCE: invention relates to the field of biotechnology, in particular to a lentiviral gene delivery vector containing an expression cassette containing a promotor sequence, a sequence encoding polypeptide of human pyruvate kinase of liver and red blood cells (PKLR) and a signal of ribonucleic acid (RNA) export, as well as to a composition containing it. A method for the expression of transgene in erythroid cells in vitro is also disclosed, providing for bringing into contact of one or more erythroid cells with the above-mentioned vector.
EFFECT: invention is effective for the treatment or prevention of pyruvate kinase deficiency.
17 cl, 22 dwg, 4 tbl, 11 ex
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