| abstract |
The present disclosure targets, for example, ATP7B exon 6 or adjacent introns to provide therapy for Wilson's disease, thereby regulating the splicing of ATP7B pre-mRNA to reduce the level of an ATP7B mRNA molecule having exon 6. Provided are antisense oligonucleotides, compositions, and methods of increasing. The present disclosure is a nucleobase sequence that is at least 70% complementary to an ATP7B target sequence in an exon 6, 5'-adjacent intron, 3'-adjacent intron, or a combination of exons 6 and 5'-adjacent or 3'-adjacent introns. To provide an antisense oligonucleotide containing. [Selection diagram] None |