ANTISENSE OLIGONUCLEOTIDES AND USES THREREOF

摘要

The present invention refers to an antisense oligonucleotide having a L3 score of about ESE score of about > 70 %; and the antisense oligonucleotide binds to a target exon pre-mRNA starting in the first one third of the target exon pre-mRNA, counting from 5´ to 3' as well as method of a method of selecting at least one antisense oligonucleotide from a plurality of antisense oligonucleotides targeting a target sequence on one of a plurality of exons on a pre-mRNA.