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Application of antisense technology to therapeutics

  • O Heidenreich
  • , S H Kang
  • , X Xu
  • , M Nerenberg

Research output: Contribution to journalReview articlepeer-review

Abstract

Antisense oligonucleotides inhibit gene expression by binding in a sequence-specific manner to an RNA target. Modern nucleotide chemistry has enabled the synthesis of chemically modified oligonucleotides that are highly resistant to nuclease degradation. Among other applications, these agents are currently being evaluated as potential antiviral and anticancer drugs. However, several unsolved problems remain. Poor efficiency of delivery to cells, tissue toxicity and antisense-independent biological effects of oligonucleotides currently limit the widespread application of antisense oligonucleotides to human disease. This article reviews some of the applications of antisense oligonucleotides and discusses problems associated with these applications.

Original languageEnglish
Pages (from-to)128-33
Number of pages6
JournalMolecular medicine today
Volume1
Issue number3
DOIs
Publication statusPublished - Jun 1995
Externally publishedYes

Keywords

  • Animals
  • Anti-HIV Agents/pharmacology
  • Drug Carriers
  • Gene Expression Regulation/drug effects
  • HIV-1/drug effects
  • Humans
  • Mammals
  • Oligonucleotides, Antisense/chemistry
  • Thionucleotides/chemistry

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