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Exploring the Therapeutic Potential: Antisense RNA Delivery Via Bacteriophage Platform
Amir Jalali1, Majid Komijani1, Hussein Alyafuri1
1Department of Biology, Faculty of Science, Arak University, Arak, 38156-8-8349, Iran.
Iranian Journal of Biotechnology
|October 20, 2025
Summary
RNA therapeutics, including antisense oligonucleotides (ASOs), show promise for genetic diseases. Advanced delivery systems like bacteriophages and virus-like particles enhance ASO efficacy and overcome challenges for various applications.
Area of Science:
- Biochemistry
- Molecular Biology
- Therapeutics
Background:
- Nucleic acid therapeutics, particularly antisense oligonucleotides (ASOs), represent a significant advancement in treating genetic diseases.
- RNA medicine offers potential therapeutic interventions by targeting specific RNA molecules, but faces challenges in stability, delivery, and off-target effects.
Purpose of the Study:
- To review the mechanisms, applications, and delivery platforms of RNA therapeutics, with a focus on antisense oligonucleotides.
- To explore innovative delivery systems for enhancing the efficacy and overcoming the challenges associated with RNA-based therapeutics.
Main Methods:
- Comprehensive review of existing literature on RNA therapeutics, antisense oligonucleotides, and various delivery platforms.
- Analysis of mechanisms, applications, and clinical implications of RNA therapeutics, including bacteriophage and virus-like particle systems.
- Examination of FDA-approved ASO drugs and CRISPR-derived approaches.
Main Results:
- Various delivery platforms, including lipid nanoparticles, polymeric nanoparticles, cell-penetrating peptides, exosomes, polyplexes, and virus-like particles (VLPs), are employed to improve RNA stability and intracellular delivery.
- Bacteriophages and VLPs show significant potential as versatile delivery systems for RNA therapeutics, targeting bacterial infections, biofilm eradication, cancer, and viral infections.
- Targeted delivery using bacteriophages enhances therapeutic outcomes due to their ease of development, large cargo capacity, and safety.
Conclusions:
- RNA therapeutics, especially ASOs, hold substantial promise for treating genetic diseases and other conditions.
- Advanced delivery systems are crucial for overcoming the inherent challenges of RNA therapeutics, improving their clinical applicability.
- The exploration of novel platforms like bacteriophages and VLPs expands the therapeutic potential of RNA-based interventions, offering new avenues for disease treatment.
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