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(19)

(11)

2 831 087

(13)

(51)

IPC

C12N15/113(2010-01-01)

A61K31/7088(2006-01-01)

A61K48/00(2006-01-01)

A61P21/00(2006-01-01)

A61P43/00(2006-01-01)

(21) (22)

Application

2021131553, 2020-03-27

(24)

Start date

2020-03-27

(22)

Actual filing date

2020-03-27

(45)

Published

2024-12-02

(72)

Inventor

Kumagai, ShinjiYashiro, TakashiAraki, TomoKanagawa, TakayukiOkagaki, ChiekoFurukawa, Hiroyuki

(73)

Holder

Mitsubishi Tanabe Pharma Corporation

COMPOUND, METHOD AND PHARMACEUTICAL COMPOSITION FOR MODULATING DUX4 EXPRESSION Russian patent published in 2024 - IPC C12N15/113 A61K31/7088 A61K48/00 A61P21/00 A61P43/00

Abstract RU 2831087 C2

FIELD: biotechnology.

SUBSTANCE: presented group of inventions can be used in medicine. There are disclosed modified oligonucleotides capable of inhibition of double homeobox 4 (DUX4) gene expression.

EFFECT: invention can be used in treating diseases associated with abnormal expression of said gene in an individual, in particular, in antisense gene therapy of facioscapulohumeral muscular dystrophy (FSHD), B-cell acute lymphocytic leukemia, differentiated round-cell sarcoma and fetal rhabdomyosarcoma.

29 cl, 6 dwg, 5 tbl, 12 ex

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RU 2 831 087 C2

Authors

Kumagai, Shinji

Yashiro, Takashi

Araki, Tomo

Kanagawa, Takayuki

Okagaki, Chieko

Furukawa, Hiroyuki

Dates

2024-12-02—Published

2020-03-27—Filed