Stabile Zieleditierende Guide-Rna mit Darin Eingeführter Phosphatteilmodifikation

Details

Veröffentlichungs-Nr.

EP4768581

Anmeldetag

26. August 2024

Veröffentlichung

6. März 2025

Rechtsraum

EP

IPC

C12N15/11, C12N15/09

Abstract

Provided is an oligonucleotide capable of inducing the editing activity of ADAR in a cell and having excellent stability. The oligonucleotide includes a first oligonucleotide and a second oligonucleotide linked to the 5' side of the first oligonucleotide, and is capable of inducing the site-specific editing in the target RNA. The first oligonucleotide is composed of a target-corresponding nucleoside residue corresponding to an adenosine residue in the target RNA, an oligonucleotide containing 3 to 6 residues, linked to the 5' side of the target-corresponding nucleoside residue, and having a base sequence complementary to the target RNA and an oligonucleotide containing 10 to 24 residues, linked to the 3' side of the target-corresponding nucleoside residue, and having a base sequence complementary to the target RNA, and at least one phosphorus-containing linking group selected from the group consisting of a phosphorus-containing linking group linking a 1st nucleoside residue and a 2nd nucleoside residue and a phosphorus-containing linking group linking a 4th nucleoside residue and a 5th nucleoside residue, counting in the 3' direction from the target-corresponding nucleoside residue, is at least one selected from the group consisting of an alkylphosphonic residue, an alkyl phosphate residue, and a substituted phosphoramide residue. At the 3' end of the second oligonucleotide, a nucleoside residue corresponding to a nucleoside residue of the target RNA is deleted, or, at the 3' end, the second oligonucleotide has a nucleoside residue that does not form a complementary pair with the nucleoside residue of the target RNA, and contains 2 to 10 residues, and nucleoside residues at least other than at the 3' end are complementary to the target RNA.

Anmelder

Firmen

Fukuoka University
Daiichi Sankyo Company, Limited

Land

🇯🇵 Japan