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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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. |
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