Modulation of gene expression by antisense and antigene oligodeoxynucleotides and small interfering RNA

Ram I. Mahato, Kun Cheng, Ramareddy V. Guntaka

Research output: Contribution to journalReview article

55 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)3-28
Number of pages26
JournalExpert Opinion on Drug Delivery
Issue number1
Publication statusPublished - Jan 1 2005



  • Antigene
  • Antisense oligodeoxynucleotides
  • Cellular uptake
  • Nucleic acid delivery
  • Phosphorothioates
  • RNA interference
  • Triple helix
  • Triplex-forming oligodeoxynucleotides
  • siRNA

ASJC Scopus subject areas

  • Pharmaceutical Science

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