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Advances in Lipid Nanoparticles for siRNA Delivery Tam, Yuen Yi C.; Chen, Sam; Cullis, Pieter

Abstract

Technological advances in both siRNA (small interfering RNA) and whole genome sequencing have demonstrated great potential in translating genetic information into siRNA-based drugs to halt the synthesis of most disease-causing proteins. Despite its powerful promises as a drug, siRNA requires a sophisticated delivery vehicle because of its rapid degradation in the circulation, inefficient accumulation in target tissues and inability to cross cell membranes to access the cytoplasm where it functions. Lipid nanoparticle (LNP) containing ionizable amino lipids is the leading delivery technology for siRNA, with five products in clinical trials and more in the pipeline. Here, we focus on the technological advances behind these potent systems for siRNA-mediated gene silencing.

Item Metadata

Title
Advances in Lipid Nanoparticles for siRNA Delivery
Creator
Tam, Yuen Yi C.; Chen, Sam; Cullis, Pieter
Publisher
Multidisciplinary Digital Publishing Institute
Date Issued
2013-09-18
Description
Technological advances in both siRNA (small interfering RNA) and whole genome sequencing have demonstrated great potential in translating genetic information into siRNA-based drugs to halt the synthesis of most disease-causing proteins. Despite its powerful promises as a drug, siRNA requires a sophisticated delivery vehicle because of its rapid degradation in the circulation, inefficient accumulation in target tissues and inability to cross cell membranes to access the cytoplasm where it functions. Lipid nanoparticle (LNP) containing ionizable amino lipids is the leading delivery technology for siRNA, with five products in clinical trials and more in the pipeline. Here, we focus on the technological advances behind these potent systems for siRNA-mediated gene silencing.
Subject
Lipid nanoparticle; SiRNA; Ionizable amino lipid; PKa; PEG lipid; Targeting
Genre
Article
Type
Text
Language
eng
Date Available
2019-04-16
Provider
Vancouver : University of British Columbia Library
Rights
CC BY 3.0
DOI
10.14288/1.0378235
URI
http://hdl.handle.net/2429/69706
Affiliation
Medicine, Faculty of; Biochemistry and Molecular Biology, Department of
Citation
Pharmaceutics 5 (3): 498-507 (2013)
Publisher DOI
10.3390/pharmaceutics5030498
Peer Review Status
Reviewed
Scholarly Level
Faculty
Rights URI
https://creativecommons.org/licenses/by-nc-sa/3.0/
Aggregated Source Repository
DSpace

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CC BY 3.0