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T7 peptide-functionalized nanoparticles utilizing RNA interference for glioma dual targeting
Yuyang Kuang1, Sai An, Yubo Guo
1Department of Pharmaceutics, School of Pharmacy, Fudan University, Shanghai, China.
Abstract:
Among all the malignant brain tumors, glioma is the deadliest and most common form with poor prognosis. Gene therapy is regarded as a promising way to halt the progress of the disease or even cure the tumor and RNA interference (RNAi) stands out. However, the existence of the blood-brain barrier (BBB) and blood tumor barrier (BTB) limits the delivery of these therapeutic genes. In this work, the delivery system targeting to the transferrin (Tf) receptor highly expressed on both BBB and glioma was successfully synthesized and would not compete with endogenous Tf. U87 cells stably express luciferase were employed here to simulate tumor and the RNAi experiments in vitro and in vivo validated that the gene silencing activity was 2.17-fold higher with the targeting ligand modification. The dual-targeting gene delivery system exhibits a series of advantages, such as high efficiency, low toxicity, stability and high transaction efficiency, which may provide new opportunities in RNAi therapeutics and nanomedicine of brain tumors.
Insights
This study developed a novel dual-targeting gene delivery system to overcome the blood-brain barrier for glioma treatment. The system enhances RNA interference (RNAi) efficacy, offering a promising nanomedicine approach for brain tumors.
Area of Science:
- Neuro-oncology
- Nanomedicine
- Gene Therapy
Background:
- Glioma is a deadly brain tumor with poor prognosis.
- Gene therapy, particularly RNA interference (RNAi), shows therapeutic potential.
- The blood-brain barrier (BBB) and blood tumor barrier (BTB) impede effective gene delivery to brain tumors.
Purpose of the Study:
- To develop a dual-targeting gene delivery system to overcome BBB and BTB for glioma treatment.
- To enhance the efficacy of RNA interference (RNAi) in brain tumors.
- To create a safe and efficient nanomedicine platform for brain tumor therapy.
Main Methods:
- Synthesized a gene delivery system targeting the transferrin (Tf) receptor.
- Utilized U87 cells expressing luciferase to simulate glioma.
- Conducted in vitro and in vivo RNAi experiments to evaluate gene silencing activity.
Main Results:
- The dual-targeting system successfully targeted the transferrin receptor on BBB and glioma cells.
- Gene silencing activity was 2.17-fold higher with the targeting ligand modification.
- The system demonstrated high efficiency, low toxicity, stability, and high transfection efficiency.
Conclusions:
- The developed dual-targeting gene delivery system effectively overcomes BBB and BTB for glioma.
- This nanomedicine approach significantly enhances RNAi efficacy in brain tumors.
- The system presents a promising strategy for RNAi therapeutics and nanomedicine in treating brain tumors.
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02:15Experimental RNAi
01:23RNA Interference
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...