New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.
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.
Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands. a.m.rus@lumc.nl
Annemieke
A
Aartsma-Rus
Johan T
JT
den Dunnen
Gert-Jan B
GJ
van Ommen
D000818:Animals; D018388:Codon, Terminator / Q000235:genetics; D005091:Exons / Q000235:genetics; D018014:Gene Transfer Techniques / Q000639:trends; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics* / Q000628:therapy*
D018388:Codon, Terminator