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Nanoparticle Delivery of an Oligonucleotide Payload in a Glioblastoma Multiforme Animal Model
Published on: September 27, 2024
Oligonucleotide delivery in cancer therapy
Giuseppe De Rosa1, Daniela De Stefano, Aldo Galeone
1University of Naples Federico II, Department of Pharmaceutical and Toxicological Chemistry, Faculty of Pharmacy, Via D Montesano n 49, Naples, Italy. gderosa@unina.it
Importance Of The Field:
Cancer is frequently caused by altered protein expression. Oligonucleotides (ONs) are short synthetic nucleic acid fragments, able to selectively correct protein expression into cells by different mechanisms. However, biological barriers hamper the therapeutic use of ONs without suitable delivery strategies.
Areas Covered In This Review:
This review summarizes the most meaningful non-viral strategies for ON delivery, including the chemical modifications of the ON backbone and non-viral delivery systems.
What The Reader Will Gain:
The reader will gain an update of the main strategies for ON delivery in cancer. Advantages and limits of each approach are underlined. Emphasis is given to the delivery strategies that contributed to bringing ONs into clinical trials.
Take Home Message:
In the long story of ONs for cancer therapy, the development of delivery strategies has led, in the last few years, to different opportunities to use the high therapeutic potential of these molecules in humans.
Insights
Oligonucleotides (ONs) show therapeutic potential for cancer by correcting protein expression. Advanced delivery strategies are overcoming biological barriers, enabling ONs for human cancer therapy.
Area of Science:
- Biochemistry
- Molecular Biology
- Cancer Therapeutics
Background:
- Altered protein expression is a common driver of cancer.
- Oligonucleotides (ONs) are synthetic nucleic acid fragments with potential to modulate protein expression.
- Biological barriers impede the therapeutic application of ONs without effective delivery systems.
Purpose of the Study:
- To review non-viral delivery strategies for oligonucleotides (ONs) in cancer therapy.
- To provide an update on the main ON delivery approaches.
- To highlight strategies that have advanced ONs into clinical trials.
Main Methods:
- Review of chemical modifications of the oligonucleotide backbone.
- Analysis of non-viral delivery systems for ONs.
- Evaluation of advantages and limitations of different delivery strategies.
Main Results:
- Key non-viral strategies for ON delivery in cancer are summarized.
- The review details the benefits and drawbacks of each approach.
- Emphasis is placed on delivery methods that have facilitated clinical translation of ONs.
Conclusions:
- Delivery strategy development has been crucial for harnessing the therapeutic potential of ONs in cancer.
- Recent advancements offer new opportunities for using ONs in human cancer treatment.
- The progress in ON delivery marks a significant step towards effective oligonucleotide-based cancer therapies.
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