Therapeutic implications of antisense oligonucleotides

D. Lattuada, M. Mazzei, R. Meazza, A. Nicolin

Research output: Contribution to journalArticle

Abstract

Antisense oligonucleotides and their derivatives have been shown to be specific inhibitors of gene expression. They are considered a very promising new generation of drugs, potentially useful in most human diseases, including cancers and viral infections. The elegance of the antisense oligonucleotides lies in their ability to bind, via standard Watson-Crick base pairing, a complementary region within a target mRNA. Although easily synthesized, therapeutic applications have been restricted by a number of difficulties including: stability, pharmacokinetic behavior (both a cellular and at systemic level), and the high cost of industrial production. The object of this review is to briefly describe the major properties of antisense oligonucleotides, the modalities currently under investigation to circumvent the difficulties in their use, and the up-to-date experimental applications, including findings from our own laboratory. As very few oligonucleotides need to be synthesized in order to obtain an active compound, compared with an average of 10000 new standard compounds, prospects are extremely exciting and worthy of maximum attention.

Original languageEnglish
Pages (from-to)296-299
Number of pages4
JournalInternational Journal of Clinical & Laboratory Research
Volume21
Issue number2-4
DOIs
Publication statusPublished - Jun 1992

Keywords

  • Antisense oligonucleotides
  • Therapeutic applications

ASJC Scopus subject areas

  • Clinical Biochemistry

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