New insights in gene-derived therapy: the example of Duchenne muscular dystrophy


Address for correspondence: Annemieke Aartsma-Rus, Leiden University Medical Center, Department of Human Genetics, Postzone S4-P, PO Box 9600, 2300 RC, Leiden, the Netherlands.


The two therapeutic approaches currently most advanced in clinical trials for Duchenne muscular dystrophy are antisense-mediated exon skipping and forced read-through of premature stop codons. Interestingly, these approaches target the gene product rather than the gene itself. This review will explain the rationale and current state of affairs of these approaches and will then discuss how these gene-derived therapies might also be applicable to other diseases.