EXON SKIPPING THERAPY FOR FUNCTIONAL AMELIORATION OF SEMI FUNCTIONAL DYSTROPHIN IN BECKER AND DUCHENNE MUSCULAR DYSTROPHY

摘要

Methods for stabilizing unstable proteins or for restoring functionality to non-functional or poorly functioning (semi-functional) proteins using exon skipping technology are provided. The methods involve the administration of antisense oligonucleotides to cause exon skipping, thereby removing one or more exons responsible for protein instability or lack of functionality. For example, exons encoding protease recognition sites may be removed. The method is useful for treating diseases caused by protein instability, such as Becker Muscular Dystrophy, or for treating Duchenne Muscular Distrophy patients with semi-functional dystrophin due to treatment with other exon skipping or stop codon readthrough therapies.