Antisense oligonucleotide of ATN1

Provided is an antisense oligonucleotide that controls the gene expression of ATN1 and can treat dentatorubropallidoluysian atrophy. A compound containing a modified oligonucleotide consisting of 8 to 80 linked nucleosides and having a nucleobase sequence comprising at least 8 consecutive nucleobase...

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Hauptverfasser: HASHIMOTO, Shinji, GOTO, Izumi, IRIYAMA, Yusuke
Format: Patent
Sprache:eng
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Beschreibung
Zusammenfassung:Provided is an antisense oligonucleotide that controls the gene expression of ATN1 and can treat dentatorubropallidoluysian atrophy. A compound containing a modified oligonucleotide consisting of 8 to 80 linked nucleosides and having a nucleobase sequence comprising at least 8 consecutive nucleobases that are complementary to a transcript of ATN1, or a pharmacologically acceptable salt of the compound.