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Updated: Jun 17, 2026

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery
Published on: May 2, 2019
Nanomedicine based approaches for the delivery of siRNA in cancer
B Ozpolat1, A K Sood, G Lopez-Berestein
1Department of Experimental Therapeutics, The University of Texas M. D. Anderson Cancer Center, 1515 Holcombe Blvd., Houston, TX 77030-4009, USA.
Abstract:
Small interfering RNA (siRNA) technology holds great promise as a therapeutic intervention for targeted gene silencing in cancer and other diseases. However, in vivo systemic delivery of siRNA-based therapeutics to tumour tissues/cells remains a challenge. The major limitations against the use of siRNA as a therapeutic tool are its degradation by serum nucleases, poor cellular uptake and rapid renal clearance following systemic administration. Several siRNA-based loco-regional therapeutics are already in clinical trials. Further development of siRNAs for anti-cancer therapy depends on the development of safe and effective nanocarriers for systemic administration. To overcome these hurdles, nuclease-resistant chemically modified siRNAs and variety of synthetic and natural biodegradable lipids and polymers have been developed to systemically deliver siRNA with different efficacy and safety profiles. Cationic liposomes have emerged as one of the most attractive carriers because of their ability to form complexes with negatively charged siRNA and high in vitro transfection efficiency. However, their effectiveness as potential therapeutic carriers is limited by potential for pulmonary toxicity. Recently, our laboratories described the use of neutral 1,2-dioleoyl-sn-glycero-3-phosphatidylcholine based nanoliposomes in murine tumour models. We found this approach to be safe and 10- and 30-fold more effective than cationic liposomes and naked siRNA, respectively, for systemic delivery of siRNA into tumour tissues. Here, we review potential approaches for systemic delivery of siRNA for cancer therapy.
Insights
Systemic delivery of small interfering RNA (siRNA) for cancer therapy faces challenges. Neutral nanoliposomes show promise as a safe and effective delivery system, outperforming cationic liposomes and naked siRNA in tumor targeting.
Area of Science:
- Biotechnology
- Nanomedicine
- Cancer Therapeutics
Background:
- Small interfering RNA (siRNA) offers targeted gene silencing for diseases like cancer.
- Systemic delivery of siRNA therapeutics to tumors is hindered by nuclease degradation, poor uptake, and rapid clearance.
- Current research focuses on developing safe and effective nanocarriers for enhanced siRNA delivery.
Purpose of the Study:
- To review potential approaches for systemic delivery of siRNA for cancer therapy.
- To highlight the challenges and advancements in siRNA delivery systems.
- To discuss the potential of novel nanocarriers in overcoming current limitations.
Main Methods:
- Review of existing literature on siRNA delivery systems.
- Analysis of various nanocarrier strategies, including liposomes and polymers.
- Evaluation of safety and efficacy profiles of different delivery approaches.
Main Results:
- Neutral 1,2-dioleoyl-sn-glycero-3-phosphatidylcholine based nanoliposomes demonstrated safety and superior efficacy in murine tumor models.
- This approach was 10-fold more effective than cationic liposomes and 30-fold more effective than naked siRNA.
- Chemically modified siRNAs and biodegradable lipids/polymers have been developed with varying profiles.
Conclusions:
- Neutral nanoliposomes represent a promising strategy for safe and effective systemic siRNA delivery in cancer therapy.
- Overcoming delivery challenges is crucial for realizing the full therapeutic potential of siRNA.
- Further development of nanocarrier systems is essential for advancing siRNA-based cancer treatments.
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