SYSTEM OF TARGETED SPLICING CHANGES IN MARK2 GENE Russian patent published in 2023 - IPC C12N15/113 C07H21/04 A61K48/00 

Abstract RU 2810907 C1

FIELD: biotechnology; medicine.

SUBSTANCE: antisense oligonucleotide is proposed that is capable of increasing the frequency of inclusion of exon 17 in the human MARK2 gene mRNA during the formation of the said mRNA by splicing from pre-mRNA in the cell. The antisense oligonucleotide has the sequence 5'-CGCTCAGCGAGAC-3'P, in which every second nucleotide is a locked nucleic acid (LNA) and every internucleotide bond is a phosphorothioate bond.

EFFECT: invention provides a targeted change in splicing in MARK2 gene and can be used to correct possible deregulation of splicing in MARK2 gene and, accordingly, for the production of drugs aimed at such correction.

1 cl, 1 dwg, 2 tbl, 3 ex

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Authors

Pervushin Dmitrij Davidovich

Skvortsov Dmitrij Aleksandrovich

Margasyuk Sergej Dmitrievich

Petrova Marina Viktorovna

Vlasenok Mariya Aleksandrovna

Zavilejskij Lev Georgievich

Dates

2023-12-29Published

2023-09-19Filed