Agent for Regulating Expression and/or Function of RPS25 Gene

    公开(公告)号:US20240018516A1

    公开(公告)日:2024-01-18

    申请号:US18035300

    申请日:2021-11-05

    摘要: A single-stranded antisense oligonucleotide, or a pharmaceutically acceptable salt thereof, capable of modulating expression and/or function of RPS25 gene, wherein nucleotides of the single-stranded antisense oligonucleotide are bonded to each other via a phosphate group and/or a modified phosphate group, the single-stranded antisense oligonucleotide includes a gap region, a 3′ wing region bonded to a 3′ end of the gap region, and a 5′ wing region bonded to a 5′ end of the gap region, the gap region is a deoxyribose-based nucleic acid optionally including a nucleic acid having a modified sugar moiety, each of the 3′ wing region and the 5′ wing region is a modified nucleotide, the single-stranded antisense oligonucleotide has a base length of 12- to 30-mer, and a base sequence of the antisense oligonucleotide is: a base sequence with a sequence identity of 90% to 100% to a base sequence complementary to at least one target region of the same base length as the antisense oligonucleotide present in the base sequence as set forth in SEQ ID NO: 1 or SEQ ID NO: 2; a base sequence complementary to a base sequence of the target region with deletion, substitution, insertion, or addition of one or several bases; or a base sequence capable of hybridizing under stringent conditions with an oligonucleotide having the target region.