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Delivery of RNAi therapeutics: work in progress
1Institute of Pharmaceutical Science, King's College London, Franklin-Wilkins Building, Waterloo Campus, 150 Stamford Street, London SE1 9NH , UK and GlobalAcorn Limited , London , UK a.miller07@btinternet.com andrew.miller@globalacorn.com.
Expert Review of Medical Devices
|November 8, 2013
Summary
Lipid-based nanoparticles (LNPs) are crucial for delivering RNA interference (RNAi) therapeutics to target cells in vivo. Research is advancing LNP systems for improved RNAi therapy efficacy and clinical viability.
Area of Science:
- Biotechnology
- Nanomedicine
- Molecular Biology
Background:
- RNA interference (RNAi) therapeutics hold significant promise for future medical treatments.
- Effective delivery of RNAi effectors to target cells in vivo remains a critical challenge, often requiring specialized delivery systems or vectors.
Purpose of the Study:
- This review focuses on lipid-based nanoparticle (LNP) delivery systems for RNAi effectors.
- It examines LNPs that have demonstrated effective in vivo delivery of functional RNAi payloads to target cells.
Main Methods:
- The review synthesizes current research and development in LNP delivery systems for RNAi.
- It analyzes studies investigating LNP-mediated delivery in vivo and the biophysical and chemical principles governing their behavior.
Main Results:
- LNPs are emerging as preferred synthetic delivery systems in preclinical studies and clinical trials for RNAi effectors.
- Studies are identifying key biophysical parameters and physical organic chemistry rules for understanding LNP delivery in vivo.
Conclusions:
- LNP delivery systems are rapidly growing in utility for RNAi therapeutics.
- The identified parameters and rules provide a foundation for developing next-generation LNPs with enhanced clinical viability.
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