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Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
09:04

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-based therapeutics for cancer

Nicholas M Dean1, C Frank Bennett

  • 1ISIS Pharmaceuticals, 2282 Faraday Ave, Carlsbad, CA 92008, USA.

Oncogene
|December 10, 2003
PubMed

Insights

Antisense technology has advanced significantly, enabling gene inhibition in various species. With over 20 drugs in clinical trials, this technology shows promise for drug discovery and gene functionalization.

Area of Science:

  • Molecular Biology
  • Pharmacology
  • Genetics

Background:

  • Antisense technology has undergone substantial development over the last 14 years.
  • A deeper understanding of its capabilities and constraints has been achieved.
  • Antisense oligonucleotides (ASOs) are established tools for gene inhibition.

Purpose of the Study:

  • To review the progress and current status of antisense technology.
  • To highlight its utility as a drug discovery platform and research tool.
  • To discuss future potential based on medicinal chemistry and formulation advances.

Main Methods:

  • Selective gene inhibition in mammalian cells, rodents, and humans using antisense oligonucleotides.
  • Clinical trial progression of over 20 antisense drugs.
  • Evaluation of therapeutic and commercial potential through medicinal chemistry and formulation.

Main Results:

  • ASOs have successfully inhibited thousands of genes across multiple species.
  • Numerous antisense drugs are in clinical trials, with some demonstrating positive outcomes.
  • The technology is recognized as a valid platform for both drug discovery and gene functionalization.

Conclusions:

  • Antisense technology is a validated approach for drug discovery and experimental gene functionalization.
  • Continued advancements in medicinal chemistry and formulation are expected to boost its therapeutic and commercial viability.
  • The field anticipates both successes and failures, characteristic of drug development.

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