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Updated: Aug 29, 2026

Polymalic Acid-based Nano Biopolymers for Targeting of Multiple Tumor Markers: An Opportunity for Personalized Medicine?
Published on: June 13, 2014
Progress in the development of nucleic acid therapeutics for cancer
Anna Kalota1, Susan E Shetzline, Alan M Gewirtz
1Department of Medicine, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania 19104, USA.
Abstract:
Many cancers are characterized by abnormal gene expression. Silencing these aberrantly expressed genes could therefore have therapeutic utility and by virtue of specific targeting, prove less toxic than conventional cancer therapies. A number of strategies for inhibiting gene expression have been developed. Some, such as triple helix forming, or decoy transcription factor binding, oligodeoxynucleotides seek to disrupt gene expression at the level of transcription. Others, such as antisense oligonucleotides (ODN) and short interfering RNA (siRNA) molecules attempt to disrupt expression at the level of mRNA translation. In this review, we provide an overview of gene silencing agents and their development for use as cancer therapeutics. We will focus on mRNA targeting methodologies and discuss issues core to the clinical success of these molecules including cellular delivery, and successful targeting. The potential utility of nucleic acid based therapeutics in the clinic will also be addressed.
Insights
Gene silencing agents offer targeted cancer therapy by inhibiting abnormal gene expression. This review focuses on messenger RNA (mRNA) targeting strategies like antisense oligonucleotides (ODN) and short interfering RNA (siRNA) for potential clinical use.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- Aberrant gene expression is a hallmark of many cancers.
- Targeted gene silencing offers a potentially less toxic alternative to conventional chemotherapy.
- Several strategies exist to inhibit gene expression at transcriptional or translational levels.
Purpose of the Study:
- To review gene silencing agents for cancer therapeutics.
- To focus on messenger RNA (mRNA) targeting methodologies.
- To address challenges in clinical application, including cellular delivery and targeting efficacy.
Main Methods:
- Overview of gene silencing strategies, including triple helix forming oligonucleotides, decoy transcription factor binding oligonucleotides, antisense oligonucleotides (ODN), and short interfering RNA (siRNA).
- Focus on mRNA targeting agents.
- Discussion of clinical considerations for nucleic acid-based therapeutics.
Main Results:
- Various nucleic acid-based agents target gene expression at transcription or mRNA translation.
- Antisense ODN and siRNA are key mRNA targeting modalities.
- Cellular delivery and successful targeting are critical for clinical success.
Conclusions:
- Gene silencing agents, particularly mRNA-targeting ones, show therapeutic promise for cancer.
- Further development is needed to overcome challenges in delivery and targeting for clinical translation.
- Nucleic acid-based therapeutics represent a potential new frontier in cancer treatment.
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