Related Experiment Video

Updated: Aug 17, 2026

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

Nucleic acid modulation of gene expression: approaches for nucleic acid therapeutics against cancer

Yuji Nakata1, Tae-Kon Kim, Susan Shetzline

  • 1Hematology/Oncology, University of Pennsylvania School of Medicine, Philadelphia, PA 19104, USA. nakatay@mail.med.upenn.edu

Critical Reviews in Eukaryotic Gene Expression
|July 19, 2005
PubMed

Insights

Gene silencing using anti-messenger RNA (mRNA) strategies offers a promising approach to cancer therapy by targeting abnormal gene expression. This review covers antisense oligonucleotides, ribozymes, DNAzymes, and small interfering RNA (siRNA) for cancer treatment.

Area of Science:

  • Molecular Biology
  • Oncology
  • Biochemistry

Background:

  • Cancers exhibit abnormal gene expression critical for malignant phenotypes like uncontrolled proliferation and apoptosis.
  • Targeting and silencing these aberrant genes represent a rational therapeutic strategy for cancer treatment.

Purpose of the Study:

  • To review anti-messenger RNA (mRNA) gene silencing agents for cancer therapeutics.
  • To provide an overview of the development of these agents.

Main Methods:

  • Review of anti-mRNA gene silencing strategies including antisense oligonucleotides, ribozymes, DNAzymes, and small interfering RNA (siRNA).
  • Focus on agents that induce RNA interference (RNAi).

Main Results:

  • Antisense oligonucleotides, particularly phosphorothioate (PS) oligonucleotides, are the most utilized agents in clinical trials.
  • Various anti-mRNA agents show potential for cancer therapy.

Conclusions:

  • Anti-mRNA gene silencing is a developing therapeutic approach for cancers.
  • Further development of these agents, especially antisense oligonucleotides, is crucial for clinical application.

More Related Videos

Dual CRISPR-Interference Strategy for Targeting Synthetic Lethal Interactions Between Non-Coding RNAs in Cancer Cells07:23

Dual CRISPR-Interference Strategy for Targeting Synthetic Lethal Interactions Between Non-Coding RNAs in Cancer Cells

Published on: May 30, 2025

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...
MicroRNAs01:22

MicroRNAs

MicroRNA (miRNA) are short, regulatory RNA transcribed from introns (non-coding regions of a gene) or intergenic regions (stretches of DNA present between genes). Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself, forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA...
MicroRNAs01:22

MicroRNAs

MicroRNA (miRNA) are short, regulatory RNA transcribed from introns—non-coding regions of a gene—or intergenic regions—stretches of DNA present between genes. Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA ends...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Regulation of Expression at Multiple Steps01:23

Regulation of Expression at Multiple Steps

The gene expression in cells is regulated at different stages: (i) transcription, (ii) RNA processing, (iii) RNA localization, and (iv) translation. Transcriptional regulation is mediated by regulatory proteins such as transcription factors, activators, or repressors—these control gene expression by initiating or inhibiting the transcription of genes. Once a precursor or pre-mRNA is produced, it undergoes post-transcriptional modification, including 5' capping, splicing, and the addition of a...