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Published on: April 16, 2019
Significant and prolonged antisense effect of a multifunctional envelope-type nano device encapsulating antisense
Yoshio Nakamura1, Kentaro Kogure, Yuma Yamada
1Graduate School of Pharmaceutical Sciences, Hokkaido University, Kita-12, Nishi-6, Sapporo 060-0812, Japan.
The Journal of Pharmacy and Pharmacology
|April 7, 2006
Summary
Researchers developed a novel multifunctional envelope-type nano device (MEND) for efficient non-viral gene delivery. Protamine-based MENDs demonstrated superior antisense effects, outperforming commercial alternatives for plasmid DNA and oligodeoxynucleotide delivery.
Area of Science:
- Nanotechnology
- Gene Delivery
- Molecular Biology
Background:
- Non-viral gene delivery systems are crucial for therapeutic applications.
- Developing efficient and safe delivery vehicles remains a challenge.
- Octaarginine (R8) serves as a promising internalizing ligand for cellular uptake.
Purpose of the Study:
- To develop and evaluate a multifunctional envelope-type nano device (MEND) for plasmid DNA (pDNA) and oligodeoxynucleotide (ODN) delivery.
- To assess the antisense efficacy of R8-MENDs using different polycationic condensing agents.
- To investigate the role of protamine as both a condensing and decondensing agent in R8-MENDs.
Main Methods:
- Preparation of three types of R8-MENDs co-encapsulating pDNA and ODN using stearyl octaarginine (STRR8), poly-L-lysine (PLL), or protamine.
- In vitro analysis of antisense effects of ODN-encapsulated R8-MENDs by measuring luciferase activity.
- Comparison of R8-MEND efficacy with LipofectAmine2000.
Main Results:
- The protamine-based ODN-MEND achieved a 90% antisense effect within 16 hours and maintained over 75% for 48 hours, significantly outperforming LipofectAmine2000.
- R8-MENDs formulated with PLL and STR-R8 showed no significant antisense effect.
- The observed antisense effect correlated with siRNA-encoding R8-MEND silencing effects, suggesting efficient cytosolic and nuclear delivery.
Conclusions:
- Protamine is an effective condensing agent for R8-MENDs, yielding high antisense activity.
- R8-MENDs facilitate DNA delivery to both cytosol and nucleus, with protamine acting as an efficient decondensing agent in both compartments.
- The developed ODN-MEND using protamine represents a highly effective non-viral gene delivery system for antisense therapy.
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