The potential of oligonucleotides for therapeutic applications.
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.
Fichou
Yann
Y
Inserm U613, Université de Bretagne Occidentale, 46 rue Félix Le Dantec, 29275 Brest Cedex, France.
Férec
Claude
C
D000818:Animals; D020868:Gene Silencing; D018390:Gene Targeting / Q000379:methods* / Q000639:trends*; D005818:Genetic Engineering / Q000379:methods* / Q000639:trends*; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends*; D006801:Humans; D009841:Oligonucleotides / Q000627:therapeutic use*
D009841:Oligonucleotides