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Updated: Sep 25, 2026

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
Published on: September 21, 2017
Antisense oligonucleotide therapy in cancer
Alick C Stephens1, Rodney P A Rivers
1Academic Department of Paediatrics, Imperial College London, St Mary's Campus, Norfolk Place, Paddington, London, W2 1PG, UK. a.stephens@imperial.ac.uk
Abstract:
The sequencing of the human genome has highlighted some of the genes that are of importance in disease states. This has provided opportunities for the development of new therapeutics to target a wide range of human diseases. These new drugs are intended to be highly specific; antisense oligonucleotides (ONs) are one such class of new drugs. ONs are short pieces of DNA which hybridize to a specific target mRNA blocking its translation to protein, thereby inhibiting the action of the gene. Several genes known to be of importance in the regulation of apoptosis, cell growth, metastasis and angiogenesis provide a tantalizing prospect for the development of anticancer agents. The phosphorothioate antisense ONs are the current choice for antisense therapy. This article reviews the current strategies for antisense targets in cancer therapy.
Insights
Antisense oligonucleotides (ONs) are novel DNA-based therapeutics targeting specific gene activity. This review explores current strategies for using ONs in cancer therapy by targeting genes involved in apoptosis and cell growth.
Area of Science:
- Genomics and Molecular Biology
- Pharmacology and Therapeutics
- Oncology
Background:
- Human genome sequencing reveals disease-associated genes.
- New targeted therapeutics offer treatment opportunities.
- Antisense oligonucleotides (ONs) are a promising class of drugs.
Purpose of the Study:
- To review current strategies for antisense oligonucleotide (ON) targets in cancer therapy.
- To highlight the potential of ONs in developing novel anticancer agents.
Main Methods:
- Review of scientific literature on antisense oligonucleotide therapy.
- Focus on ONs targeting genes regulating apoptosis, cell growth, metastasis, and angiogenesis.
- Discussion of phosphorothioate antisense ONs as the current standard.
Main Results:
- Antisense oligonucleotides (ONs) hybridize to target mRNA, blocking protein translation.
- Specific gene targets in cancer regulation offer therapeutic potential.
- Phosphorothioate ONs are the prevalent choice for current antisense therapy.
Conclusions:
- Antisense oligonucleotides represent a highly specific therapeutic approach for cancer.
- Targeting key genes in cancer pathways with ONs is a viable strategy.
- Further development of ON-based therapies holds promise for cancer treatment.
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