ANTISENSE OLIGONUCLEOTIDES CAPABLE OF INDUCING EXON SKIPPING AND THE USE THEREOF AS A MEDICAMENT FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD)

摘要

The invention relates to the use of an antisense oligonucleotide directed against an exon sequence of the dystrophin gene for preparing a medicament capable of determining the skipping of said exon from the mRNA dystrophin transcript, thereby promoting the production of a functional or partially functional dystrophin protein in a patient carrying a small mutation in said exon. Such a use is characterised in that the exon is selected from the group consisting of exon 10, exon 16, exon 26, exon 33 and exon 34 of the dystrophin gene. Specific antisense oligonucleotides suitable to be used in the invention are also described.