Oligonucleotide for the Treatment of Muscular Dystrophy Patients

摘要

The invention relates to an oligonucleotide and to a pharmaceutical composition comprising said oligonucleotide. This oligonucleotide is able to bind to a region of a first exon from a dystrophin pre-mRNA and to a region of a second exon within the same pre-mRNA, wherein said region of said second exon has at least 50% identity with said region of said first exon, wherein said oligonucleotide is suitable for the skipping of said first and second exons of said pre-mRNA, and preferably the entire stretch of exons in between.

主权项

1. A method for designing an oligonucleotide for producing an at least partially functional protein, wherein said method comprises the following steps:
(a) identifying an in-frame combination of a first and a second exon in a same pre-mRNA, wherein a region of said second exon has at least 50% identity with a region of said first exon; (b) designing an oligonucleotide that is functional to bind to said region of said first exon and said region of said second exon, and (c) wherein said binding results in the skipping of said first and said second exon.