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(19)

RU

(11)

2 674 600

(13)

C2

(51)

IPC

C12N15/113(2010-01-01)

A61K31/7088(2006-01-01)

A61K48/00(2006-01-01)

A61P21/00(2006-01-01)

(21) (22)

Application

2015103227, 2013-07-03

(24)

Start date

2013-07-03

(22)

Actual filing date

2013-07-03

(45)

Published

2018-12-11

(72)

Inventor

Van Detekom Yudit Kristina Teodora

(73)

Holder

Prosensa Teknolodzhiz B.V.

OLIGONUCLEOTIDE FOR TREATMENT OF MUSCULAR DYSTROPHY PATIENTS Russian patent published in 2018 - IPC C12N15/113 A61K31/7088 A61K48/00 A61P21/00 

Abstract RU 2674600 C2

FIELD: biotechnology.

SUBSTANCE: present invention relates to biotechnology. Disclosed is an oligonucleotide to allow skipping of two or more exons of dystrophin pre-mRNA. Also disclosed is a composition to enable skipping of two or more exons of dystrophin pre-mRNA, a method and use of the oligonucleotide and composition for preventing, slowing down, alleviating and / or treating Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).

EFFECT: present invention may find further application in the treatment of various diseases caused by mutations in the dystrophin gene.

5 cl, 5 dwg, 6 tbl, 5 ex

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RU 2 674 600 C2

Authors

Van Detekom Yudit Kristina Teodora

Dates

2018-12-11—Published

2013-07-03—Filed

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