Antisense molecules for targeted cancer therapy

V Wacheck1, U Zangemeister-Wittke

  • 1Department of Clinical Pharmacology, Experimental Oncology/Molecular Pharmacology, Medical University Vienna/AKH, A-1090 Vienna, Austria.

Insights

Novel antisense molecules, including antisense oligonucleotides (ASO) and small interfering RNA (siRNA), offer targeted cancer therapy by inhibiting gene expression. Despite past challenges, advancements in targeting and delivery show promise for future clinical efficacy.

Area of Science:

  • Molecular oncology
  • Gene therapy
  • Drug discovery

Background:

  • Traditional chemotherapy lacks specificity, causing toxicity to normal tissues.
  • Understanding cancer genetics reveals specific molecular targets for therapy.
  • Novel biological agents offer improved specificity over cytotoxic drugs.

Purpose of the Study:

  • To review the potential of antisense molecules in cancer treatment.
  • To highlight the mechanisms and advantages of antisense oligonucleotides (ASO) and small interfering RNA (siRNA).
  • To discuss the future prospects of antisense-based cancer therapies.

Main Methods:

  • Review of current literature on antisense technology in oncology.
  • Analysis of gene expression inhibition by ASO and siRNA.
  • Discussion of challenges and advancements in targeted delivery systems.

Main Results:

  • ASO and siRNA specifically target gene expression at the transcript level.
  • These agents are effective against oncogenes, including those with inaccessible protein products.
  • Recent trials faced challenges, but technological improvements are ongoing.

Conclusions:

  • Antisense molecules represent a promising strategy for targeted cancer therapy.
  • Continued research in target identification, ASO/siRNA technology, and delivery systems is crucial.
  • These advancements offer renewed hope for improved clinical efficacy in cancer treatment.

Related Concept Videos

Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
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...
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...
Cancer02:18

Cancer

Cancers arise due to mutations in genes involved in the regulation of cell division, which leads to unrestricted cell proliferation. Modern science and medicine have made great strides in the understanding and treatment of cancer, including eradicating cancer in some patients. However, there is still no cure for cancer. This is largely due to the fact that cancer is a large group of many diseases.