Antisense oligonucleotides for treating dystrophic epidermolysis bullosa

摘要

The problem is that antisense oligonucleotides that exon 80 can prevent or reduce the inclusion of human COL7A1 mRNA, and exon 80 are included in human COL7A1 mRNA Or to provide a method for reducing.Solution: when human COL7A1 mRNA is generated by splicing from pre mRNA in the cellPrevent exon 80 from being contained in the mRNA orAn antisense oligonucleotide (AON) that can be reduced or- complementary to at least a portion of the exon 80 of the COL7A1 gene;Nucleotide sequences not complementary to the upstream introns of exons 80Or - complementary to at least part of the exonAn AON characterized by comprising a nucleotide sequence that is less than 24 nucleotide length.No selection