MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE

摘要

The invention relates to oligonucleotides for inducing skipping of exon 55 of the dystrophin gene. The invention also relates to methods of inducing exon 55 skipping using the oligonucleotides.

主权项

1. A method for inducing the skipping of exon 51 of the human dystrophin pre-mRNA, said method comprising providing an oligonucleotide of 20 to 50 nucleotides in length to a cell, wherein said oligonucleotide is capable of binding to an exon-internal sequence of exon 51 of the human dystrophin pre-mRNA and inducing skipping of exon 51, wherein h51AON1 (UCAAGGAAGAUGGCAUUUCU) (SEQ ID NO: 27) is capable of binding to said exon-internal sequence.