EXON SKIPPING THERAPY FOR DYSTROPHIC EPIDERMOLYSIS BULLOSA

摘要

The present invention relates to the treatment of Dystrophic Epidermolysis Bullosa. The inventors showed that exon skipping therapy may be performed using antisense oligonucleotides targeting specifically exon 73, 74 or 80. In particular, the invention relates to a tricyclo-DNA-antisense oligonucleotide complementary to a nucleic acid sequence of COL7A1 gene that is necessary for correct splicing of one or more exons which encode amino acid sequence of type VII collagen implicated in dysfunction of a mutated type VII collagen wherein said exons are selected from the group consisting of exon 73, 74 or 80 of the COL7A1 gene.