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Updated: Jun 13, 2025
Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System
Published on: October 29, 2019
Therapeutic Antisense Oligonucleotides in Oncology: From Bench to Bedside
Elif Çakan1,2, Olivia D Lara3,4, Anna Szymanowska1
1Department of Experimental Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77054, USA.
Abstract:
Advancements in our comprehension of tumor biology and chemoresistance have spurred the development of treatments that precisely target specific molecules within the body. Despite the expanding landscape of therapeutic options, there persists a demand for innovative approaches to address unmet clinical needs. RNA therapeutics have emerged as a promising frontier in this realm, offering novel avenues for intervention such as RNA interference and the utilization of antisense oligonucleotides (ASOs). ASOs represent a versatile class of therapeutics capable of selectively targeting messenger RNAs (mRNAs) and silencing disease-associated proteins, thereby disrupting pathogenic processes at the molecular level. Recent advancements in chemical modification and carrier molecule design have significantly enhanced the stability, biodistribution, and intracellular uptake of ASOs, thereby bolstering their therapeutic potential. While ASO therapy holds promise across various disease domains, including oncology, coronary angioplasty, neurological disorders, viral, and parasitic diseases, our review manuscript focuses specifically on the application of ASOs in targeted cancer therapies. Through a comprehensive examination of the latest research findings and clinical developments, we delve into the intricacies of ASO-based approaches to cancer treatment, shedding light on their mechanisms of action, therapeutic efficacy, and prospects.
Insights
Antisense oligonucleotides (ASOs) are a novel RNA therapeutic targeting messenger RNAs (mRNAs) to silence disease-causing proteins. This review explores ASO applications in targeted cancer therapies, highlighting their potential to address unmet clinical needs.
Area of Science:
- Biochemistry
- Molecular Biology
- Oncology
Background:
- Tumor biology and chemoresistance advancements drive targeted molecular therapies.
- Despite progress, unmet clinical needs persist, necessitating innovative therapeutic strategies.
- RNA therapeutics, including antisense oligonucleotides (ASOs), offer novel intervention methods.
Purpose of the Study:
- To review the application of ASOs in targeted cancer therapies.
- To examine the mechanisms of action, therapeutic efficacy, and prospects of ASO-based cancer treatments.
- To provide a comprehensive overview of recent research and clinical developments in ASO oncology.
Main Methods:
- Literature review of recent research findings and clinical developments in ASO therapy.
- Analysis of ASO mechanisms targeting messenger RNAs (mRNAs) and protein silencing.
- Examination of advancements in ASO chemical modification and delivery systems.
Main Results:
- ASOs selectively target mRNAs to silence disease-associated proteins, disrupting pathogenic processes.
- Chemical modifications and carrier molecules enhance ASO stability, biodistribution, and cellular uptake.
- ASO therapy shows promise across oncology, neurological disorders, and infectious diseases.
Conclusions:
- ASOs represent a promising frontier in RNA therapeutics for targeted cancer treatment.
- Advancements in ASO design have significantly improved their therapeutic potential.
- ASO-based strategies offer a novel approach to address unmet needs in cancer therapy.
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