EXON SKIP WITH PEPTIDONUCLEIC ACID DERIVATIVES Russian patent published in 2022 - IPC C12N15/113 C07K14/00 A61K38/00 C07K7/02 A61P35/00 

Abstract RU 2786637 C2

FIELD: biotechnology.

SUBSTANCE: invention relates to the field of biotechnology, specifically to new derivatives of peptido-nucleic acids, and can be applied in medicine. The invention discloses a peptidonucleic acid molecule capable of specific, strong binding to a splicing site in a pre-mRNA target. Due to the improved ability to penetrate the cell membrane, as well as the strong affinity for the target RNA, said peptidonucleic acid is able to induce exon skipping in cells.

EFFECT: invention can be used as an alternative to antisense oligonucleotides in vaccination and in the treatment of various diseases.

19 cl, 66 ex, 14 tbl, 56 dwg

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RU 2 786 637 C2

Authors

Chung, Shin

Jung, Daram

Cho, Bongjun

Jang, Kangwon

Yoon, Heungsik

Dates

2022-12-23Published

2017-12-29Filed