Targeting mRNA splicing as a potential treatment for Duchenne muscular dystrophy.

Several clinical trials have recently demonstrated that oligonucleotide-based drugs induced targeted exon skipping in dystrophin pre-mRNA in Duchenne muscular dystrophy patients, resulting in novel expression of a truncated but functional isoform of the dystrophin protein. Such exon skipping therapy has the potential to convert the lethal Duchenne phenotype into the less severe Becker phenotype. This splice switching technology has been shown to be very well tolerated and may become the first gene-specific therapy, if approved, for the treatment of Duchenne muscular dystrophy.