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US 2025/0084411 A1Published application
Antisense Nucleic Acid
AssigneeSumitomo Pharma Co., Ltd.
InventorsZhiqiu MAN, Shigehiro ASANO, Kyosuke HINO
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Abstract
The present disclosure relates to an oligonucleotide that expresses functional human WRN protein against skip mutation in the 26th or 28th exon of human WRN gene, or a pharmaceutically acceptable salt thereof, and use thereof.
Claims29
An oligonucleotide that expresses a functional human WRN protein against a skip mutation in a 26th or 28th exon of a human WRN gene, or a pharmaceutically acceptable salt thereof, wherein
- one nucleotide is bound to another via a phosphate group and/or modified phosphate group in the oligonucleotide,
- the oligonucleotide contains a modified nucleic acid having at least one modified sugar,
- the oligonucleotide is 10 to 30 mer in nucleotide length, and
- the nucleotide sequence of the oligonucleotide is:
- a nucleotide sequence having a sequence identity of 90% or more and 100% or less with a nucleotide sequence complementary to at least one target region having the same nucleotide length as the oligonucleotide in a nucleotide sequence set forth in SEQ ID NO:
- 1, SEQ ID NO:
- 2, SEQ ID NO:
- 3, SEQ ID NO:
- 4, SEQ ID NO:
- 5, or SEQ ID NO:
- 6;
- a nucleotide sequence complementary to a nucleotide sequence formed by deleting, substituting, inserting, or adding one or several nucleotides in the target region;
- or
- a nucleotide sequence hybridizable with an oligonucleotide having the target region under stringent conditions.
11 more elements
Dependencies are inferred from the claim text.