FIELD: medicine.
SUBSTANCE: invention relates to the treatment of haemophilia B. Method of preventing or treating haemophilia involves administering to a subject an effective dose of a lentiviral vector containing a nucleotide sequence at least 99% identical to SEQ ID NO: 1, which codes a polypeptide with factor IX activity (FIX). Lentiviral vector is packaged in HEK293T cells, super-expressing CD47, which have a higher level of expression of the CD47 surface protein than the control lentiviral vector produced in unmodified HEK293T cells (ATCC® CRL-11268TM). Effective dose is reduced relative to the reference dose of the control lentiviral vector required to induce the same FIX activity as the lentiviral vector.
EFFECT: implementation of the invention provides prevention or treatment of haemophilia B.
13 cl, 23 dwg, 5 ex
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