USE OF LENTIVIRAL VECTORS EXPRESSING IX FACTOR Russian patent published in 2024 - IPC C12N15/86 

Abstract RU 2816873 C2

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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RU 2 816 873 C2

Authors

Liu, Tongyao

Patarroyo-White, Susannah

Drager, Douglas

Cantore, Alessio

Naldini, Luigi

Dates

2024-04-08Published

2019-12-05Filed