Related Experiment Video

Updated: Feb 5, 2026

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System
07:47

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

Published on: October 29, 2019

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Advances in antisense oligonucleotide treatment for cancer

Lu Zhu1,2, Pariha Muhtar1,2,3, Susumu Goyama2,4

  • 1Division of Cancer RNA Research, National Cancer Center Research Institute, 5-1-1 Tsukiji, Chuo-ku, Tokyo 104-0045, Japan.

Japanese Journal of Clinical Oncology
|February 3, 2026
PubMed

Insights

Antisense oligonucleotides (ASOs) are a promising RNA therapeutic class. Research is accelerating ASO development for cancer by targeting aberrant RNA splicing, with many clinical trials underway.

Area of Science:

  • Biomedical Sciences
  • Molecular Biology
  • Oncology

Background:

  • RNA therapeutics, including antisense oligonucleotides (ASOs), represent a significant advancement in drug development, with several already approved for clinical use.
  • While most approved ASO therapies target non-malignant conditions like neurodegenerative diseases, only one is currently approved for cancer treatment.
  • Aberrant RNA splicing is increasingly recognized as a key factor in cancer development, highlighting a therapeutic opportunity.

Purpose of the Study:

  • To review recent advancements in understanding cancer biology relevant to ASO-based therapies.
  • To highlight the progress in developing RNA-targeted approaches for cancer treatment.
  • To underscore the potential of ASO therapeutics in oncology.

Main Methods:

  • Literature review of recent scientific publications and clinical trial data.
  • Analysis of the role of aberrant RNA splicing in cancer pathogenesis.
  • Synthesis of current research on ASO development for oncologic indications.

Main Results:

  • Nearly half of current RNA therapeutics modulate RNA splicing.
  • Over 250 clinical trials have investigated oligonucleotide agents for various cancer-associated molecules.
  • Early clinical trial results for ASO-based cancer therapies show encouraging outcomes.

Conclusions:

  • The development of ASO-based therapeutics for cancer is poised for acceleration due to the role of RNA splicing in oncogenesis.
  • Ongoing research and clinical trials are paving the way for novel RNA-targeted cancer treatments.
  • ASO technology offers a promising avenue for addressing unmet needs in cancer therapy.
Keywords:
RNA splicingantisenseclinical trialmolecular targeted therapyneoplasmsoligonucleotides

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