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

Abstract

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.

• Authors: Annemieke Aartsma-Rus, Johan T den Dunnen, Gert-Jan B van Ommen
• Journal: Annals of the New York Academy of Sciences (Ann N Y Acad Sci) Vol. 1214 Pg. 199-212 (Dec 2010) ISSN: 1749-6632 [Electronic] United States
• PMID: 21121926 (Publication Type: Journal Article, Review)
• Copyright: © 2010 New York Academy of Sciences.

Chemical References

• Codon, Terminator

Topics

• Animals
• Codon, Terminator (genetics)
• Exons (genetics)
• Gene Transfer Techniques (trends)
• Genetic Therapy (methods, trends)
• Humans
• Muscular Dystrophy, Duchenne (genetics, therapy)