Section review

Biologicals & immunologicals: Applications of antisense technology to both basic and clinical Research

Graeme L. Fraser, Claes R Wahlestedt

Research output: Contribution to journalArticle

4 Citations (Scopus)

Abstract

Antisense oligodeoxynucleotides (ODNs) are synthetic molecules typically designed to hybridise to the sense strand of the target gene messenger RNA (mRNA). The activity of these molecules reduces gene expression by disrupting the flow of information from gene to protein. The specificity of this interaction arises from the antisense ODN having a unique affinity for the primary sequence of the target gene due to nucleotide base complementarity. Specific inhibition of target gene products allows a rapidly increasing number of researchers to investigate gene function. Additionally, the reversible and specific nature of this interaction has initiated interest in antisense ODNs as a potentially novel class of therapeutics. Antisense molecules are already undergoing clinical trials as candidate therapeutics for several indications. Recent advances in our understanding of the activity of antisense ODNs, together with the advances in ODN chemistry, has made the notion of therapeutically active antisense molecules realistic. However, there remain many challenges in this field which must be overcome. Notably, the most commonly used chemical class of antisense ODNs, the phosphorothioates, has been associated with significant non-specific activities. This brief review aims to present in vivo applications of antisense in the domains of both basic and clinical research. 1995

Original languageEnglish
Pages (from-to)637-646
Number of pages10
JournalExpert Opinion on Investigational Drugs
Volume4
Issue number7
DOIs
StatePublished - Jul 1 1995
Externally publishedYes

Fingerprint

Oligodeoxyribonucleotides
Technology
Research
Genes
Nucleotides
Research Personnel
Clinical Trials
Gene Expression
Messenger RNA
Therapeutics
Proteins

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)

Cite this

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abstract = "Antisense oligodeoxynucleotides (ODNs) are synthetic molecules typically designed to hybridise to the sense strand of the target gene messenger RNA (mRNA). The activity of these molecules reduces gene expression by disrupting the flow of information from gene to protein. The specificity of this interaction arises from the antisense ODN having a unique affinity for the primary sequence of the target gene due to nucleotide base complementarity. Specific inhibition of target gene products allows a rapidly increasing number of researchers to investigate gene function. Additionally, the reversible and specific nature of this interaction has initiated interest in antisense ODNs as a potentially novel class of therapeutics. Antisense molecules are already undergoing clinical trials as candidate therapeutics for several indications. Recent advances in our understanding of the activity of antisense ODNs, together with the advances in ODN chemistry, has made the notion of therapeutically active antisense molecules realistic. However, there remain many challenges in this field which must be overcome. Notably, the most commonly used chemical class of antisense ODNs, the phosphorothioates, has been associated with significant non-specific activities. This brief review aims to present in vivo applications of antisense in the domains of both basic and clinical research. 1995",
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