The potential of oligonucleotides for therapeutic applications

The potential of oligonucleotides for therapeutic applications
2006 Oct 11
Fichou Y, Ferec C.
Inserm U613, Universite de Bretagne Occidentale, 46 rue Felix Le Dantec, 29275 Brest Cedex, France
Trends Biotechnol.
Viral-derived particles have been widely used and described in gene therapy clinical trials. Although substantial results have been achieved, major safety issues have also arisen. For more than a decade, oligonucleotides have been seen as an alternative to gene complementation by viral vectors or DNA plasmids, either to correct the genetic defect or to silence gene expression. The development of RNA interference has strengthened the potential of this approach. Recent clinical trials have also tested the ability of aptamer molecules and decoy oligonucleotides to sequestrate pathogenic proteins. Here, we review the potential of oligonucleotides in gene therapy, outline what has already been accomplished, and consider what remains to be done.
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