Recent advances in siRNA delivery mediated by lipid-based nanoparticles
Sei Yonezawa1, Hiroyuki Koide1, Tomohiro Asai1
1Department of Medical Biochemistry, School of Pharmaceutical Sciences, University of Shizuoka, 52-1 Yada, Suruga-ku, Shizuoka 422-8526, Japan.
Advanced Drug Delivery Reviews
|August 10, 2020
Summary
Lipid-based nanoparticles (LNPs) are advancing small interfering RNA (siRNA) delivery, overcoming challenges like degradation and charge. This technology shows promise for treating various intractable diseases.
Area of Science:
- Biotechnology
- Nanomedicine
- Drug Delivery Systems
Background:
- Small interfering RNA (siRNA) offers therapeutic potential for intractable diseases but faces challenges like degradation and negative charge.
- Advancements in siRNA delivery technology are crucial for overcoming these barriers to clinical application.
- The approval of Patisiran, an siRNA drug utilizing lipid-based nanoparticles (LNPs), marks a significant milestone.
Purpose of the Study:
- To review the design principles of LNPs for enhancing siRNA properties.
- To discuss the improvements in bioavailability and pharmacokinetics achieved through LNP-based siRNA delivery.
- To highlight the potential of LNP-encapsulated siRNA for treating diverse intractable diseases.
Main Methods:
- Review of recent literature on LNP design for siRNA encapsulation.
- Analysis of studies reporting LNP-based siRNA formulations for various diseases.
- Discussion of strategies to improve siRNA stability, cellular uptake, and therapeutic efficacy.
Main Results:
- LNP technology has significantly improved siRNA properties, including stability and charge.
- Successful application of LNP-encapsulated siRNA in preclinical and clinical studies for diseases like cancer and genetic disorders.
- Demonstrated enhancement of siRNA bioavailability and pharmacokinetics through optimized LNP formulations.
Conclusions:
- LNP-based siRNA delivery systems are a promising platform for next-generation therapeutics.
- Continued development in LNP design will accelerate the clinical translation of siRNA therapies.
- LNP-encapsulated siRNA holds potential for treating a wide range of intractable conditions.
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