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- Publisher
- Taylor & Francis
- Copyright
- © 2005 Ashley Publication Ltd.
- ISSN
- 1744-7593
- eISSN
- 1742-5247
- DOI
- 10.1517/17425247.2.1.3
- pmid
- 16296732
- Publisher site
- See Article on Publisher Site
Abstract
Antisense oligodeoxynucleotides, triplex-forming oligodeoxynucleotides and double-stranded small interfering RNAs have great potential for the treatment of many severe and debilitating diseases. Concerted efforts from both industry and academia have made significant progress in turning these nucleic acid drugs into therapeutics, and there is already one FDA-approved antisense drug in the clinic. Despite the success of one product and several other ongoing clinical trials, challenges still exist in their stability, cellular uptake, disposition, site-specific delivery and therapeutic efficacy. The principles, strategies and delivery consideration of these nucleic acids are reviewed. Furthermore, the ways to overcome the biological barriers are also discussed so that therapeutic concentrations at their target sites can be maintained for a desired period.
Journal
Expert Opinion on Drug Delivery – Taylor & Francis
Published: Jan 1, 2005
Keywords: antigene; antisense oligodeoxynucleotides; cellular uptake; nucleic acid delivery; phosphorothioates; RNA interference; siRNA; triple helix; triplex-forming oligodeoxynucleotides