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Related Concept Videos

RNA Splicing01:32

RNA Splicing

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Splicing is the process by which eukaryotic RNA is edited before its translation into protein. The RNA strand transcribed from eukaryotic DNA is called the primary transcript. The primary transcripts that become mRNAs are called precursor messenger RNAs (pre-mRNAs). Eukaryotic pre-mRNA contains alternating sequences of exons and introns. Exons are nucleotide sequences that code for proteins, whereas introns are the non-coding regions. In RNA splicing, introns are removed and exons are bonded...
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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.
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Alternative RNA Splicing02:18

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Alternative RNA splicing is the regulated splicing of exons and introns to produce different mature mRNAs from a single pre-mRNA. Unlike in constitutive splicing where a single gene produces a single type of mRNA, alternative splicing allows an organism to produce multiple proteins from a single gene and plays an important role in protein diversity.
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Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
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Cancer therapies are various modes of treatment, such as surgery, radiation therapy, and chemotherapy that are administered to cancer patients.
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Related Experiment Video

Updated: Aug 12, 2025

Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells
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Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells

Published on: April 26, 2017

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Targeting splicing factors for cancer therapy.

Ariel Bashari1, Zahava Siegfried1, Rotem Karni2

  • 1Department of Biochemistry and Molecular Biology, the Institute for Medical Research Israel-Canada, Hebrew University Hadassah Medical School, Jerusalem 9112001, Israel.

RNA (New York, N.Y.)
|January 25, 2023
PubMed
Summary

Alternative splicing (AS) misregulation drives cancer, making splicing factors therapeutic targets. New strategies focus on specific factors for precise cancer treatment, improving drug specificity and reducing toxicity.

Keywords:
cancer therapydecoy oligonucleotidessmall moleculessplicingsplicing factor

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Area of Science:

  • Molecular Biology
  • Cancer Biology
  • Pharmacology

Background:

  • Alternative splicing (AS) is a key regulator of eukaryotic gene expression.
  • Misregulation of AS, often due to splicing factor dysfunction, contributes to cancer development and progression.
  • Splicing factors are emerging as promising therapeutic targets for cancer treatment.

Purpose of the Study:

  • To review current approaches for targeting splicing factors in cancer therapy.
  • To discuss the advantages of targeting specific splicing factors over core spliceosome components.
  • To suggest methods for enhancing the specificity of splicing factor modulators.

Main Methods:

  • Review of existing literature on pharmacological modulators of splicing factors.
  • Analysis of strategies targeting distinct splicing machinery components.
  • Discussion of novel research directions in precision cancer therapy.

Main Results:

  • Pharmacological modulators (small molecules, oligonucleotides) targeting splicing factors are under development.
  • Targeting core spliceosome components can cause nonspecific and toxic effects.
  • Focusing on specific splicing factors offers a more precise therapeutic approach.

Conclusions:

  • Targeting specific splicing factors presents a promising avenue for developing more effective and less toxic cancer therapies.
  • Improving the specificity of these targeted therapies is crucial for clinical success.
  • Further research into novel strategies for modulating splicing factor function is warranted.