COL7A1 73 Oligonucleotides matching COL7A1 exon 73 for epidermolysis bullosa therapy

摘要

Antisense oligonucleotides capable of preventing or reducing exon 73 inclusion in human COL7A1 mRNA are characterized in various ways: (a) the sequence of the oligonucleotide comprises up to two CpG sequences; (b) the oligonucleotide has a length of up to 24 nucleotides; (c) The oligonucleotide can anneal to the (SRp40 / SC35 binding / ESE) element at exon 73. These oligonucleotides may be, for example, oligoribonucleotides having modified nucleoside linkages such as phosphorothioate linkages.