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

Author(s):
Aartsma-Rus, A.; Dunnen, J.T. den (0000-0002-6304-1710); Ommen, G.J.B. van; Casanova, J.L.; Abel, L.; Lyonnet, S.
Published
2010
Publication type
Article / Letter to editor

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.

Date issued
2010
Language
English
Is part of
Annals of the New York Academy of Sciences
hdl
1887/98901
doi
10.1111/j.1749-6632.2010.05836.x