Compound, method and pharmaceutical composition for regulating expression of ataxin 3

A modified oligonucleotide having an activity of inhibiting Ataxin 3 (ATXN3) expression, comprising:

• a 3′ wing segment comprising nucleosides including a modified sugar;
• a 5′ wing segment comprising nucleosides including a modified sugar;
• and
• a gap segment positioned between the 5′ wing segment and the 3′ wing segment,
• wherein a nucleobase sequence of the modified oligonucleotide is a nucleobase sequence selected from the group consisting of
• (1) TCGGGTAAGTAGATTTTC (complementary sequence of 2159-2176 of SEQ ID NO:
• 1) (SEQ ID NO:
• 239 of the sequence listing),
• (5) AGGTTATAGGATGCAGGT (complementary sequence of 5845-5862 of SEQ ID NO:
• 1) (SEQ ID NO:
• 243 of the sequence listing),
• (6) GAAGCTAAGTAGGTGACT (complementary sequence of 15115-15132 of SEQ ID NO:
• 1) (SEQ ID NO:
• 244 of the sequence listing),
• (22) GTCATCCCTATGTCTTAT (complementary sequence of 36607-36624 of SEQ ID NO:
• 1) (SEQ ID NO:
• 259 of the sequence listing),
• (23) GTCATATGGTCAGGGTAT (complementary sequence of 40453-40470 of SEQ ID NO:
• 1) (SEQ ID NO:
• 260 of the sequence listing),
• (24) TGTCATATGGTCAGGGTA (complementary sequence of 40454-40471 of SEQ ID NO:
• 1) (SEQ ID NO:
• 261 of the sequence listing),
• (25) ATGTCATATGGTCAGGGT (complementary sequence of 40455-40472 of SEQ ID NO:
• 1) (SEQ ID NO:
• 262 of the sequence listing), and
• (26) TATGTCATATGGTCAGGG (complementary sequence of 40456-40473 of SEQ ID NO:
• 1) (SEQ ID NO:
• 263 of the sequence listing)
• or a nucleobase sequence of 17 contiguous bases contained in the nucleobase sequence.