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Related Concept Videos

RNA Interference01:23

RNA Interference

RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
RNA Interference01:23

RNA Interference

RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the ATP-dependent...
Experimental RNAi02:15

Experimental RNAi

RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
Small interfering RNAs (siRNA)02:30

Small interfering RNAs (siRNA)

Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the ATP-dependent...
Upstream Processing01:27

Upstream Processing

Upstream processing represents a critical phase in biomanufacturing, wherein biological systems such as microorganisms, mammalian cells, or insect cells are cultivated to produce therapeutic proteins, vaccines, enzymes, or other biologically derived products. This phase encompasses all steps from the selection and genetic manipulation of the production organism to the cultivation of cells in bioreactors under tightly controlled environmental conditions.Host Selection and Genetic OptimizationThe...

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Direct Intraventricular Delivery of Drugs to the Rodent Central Nervous System
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Direct Intraventricular Delivery of Drugs to the Rodent Central Nervous System

Published on: May 12, 2013

Progress in antisense technology.

Stanley T Crooke1

  • 1Isis Pharmaceuticals, 2292 Faraday Avenue, Carlsbad, California 92008, USA. scrooke@isisph.com

Annual Review of Medicine
|January 30, 2004
PubMed
Summary
This summary is machine-generated.

Antisense technology uses oligonucleotide analogs to target RNA, disabling or degrading it. This approach is advancing rapidly, with approved drugs and many in development for various diseases.

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Last Updated: Jun 25, 2026

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

  • Biotechnology
  • Molecular Biology
  • Pharmacology

Background:

  • Antisense technology utilizes oligonucleotide analogs for targeted RNA interaction.
  • These agents bind to RNA via Watson-Crick hybridization, modulating gene expression.
  • Significant advancements have been made in understanding antisense mechanisms and properties.

Purpose of the Study:

  • To review the progress and potential of antisense technology.
  • To highlight its applications in gene function studies and target validation.
  • To discuss the therapeutic implications of antisense-based drugs.

Main Methods:

  • Review of basic mechanisms of antisense technology.
  • Analysis of medicinal chemistry, pharmacology, pharmacokinetics, and toxicology.
  • Examination of clinical development of antisense drugs.

Main Results:

  • Antisense agents can disable or degrade target RNA and alter splicing.
  • The technology has proven valuable for gene functionalization and target validation.
  • One antisense drug (Vitravene) is marketed, with approximately 20 in clinical trials.

Conclusions:

  • Antisense technology has matured significantly over the past decade.
  • Antisense drugs show promise for treating a wide range of diseases.
  • The field is poised for continued growth and therapeutic impact.