Affiliation:
1. CBMA (Center of Molecular and Environmental Biology), Department of Biology, University of Minho, Campus of Gualtar, 4710-057 Braga, Portugal
2. CFUM (Center of Physics), Department of Physics, University of Minho, Campus of Gualtar, 4710-057 Braga, Portugal
Abstract
The possibility of using the RNA interference (RNAi) mechanisms in gene therapy was one
of the scientific breakthroughs of the last century. Despite the extraordinary therapeutic potential of
this approach, the need for an efficient gene carrier is hampering the translation of the RNAi technology
to the clinical setting. Although a diversity of nanocarriers has been described, liposomes continue
to be one of the most attractive siRNA vehicles due to their relatively low toxicity, facilitated siRNA
complexation, high transfection efficiency and enhanced pharmacokinetic properties.
</P><P>
This review focuses on RNAi as a therapeutic approach, the challenges to its application, namely the
nucleic acids’ delivery process, and current strategies to improve therapeutic efficacy. Additionally,
lipid-based nanocarriers are described, and lessons learned from the relation between biophysical
properties and biological performance of the dioctadecyldimethylammonium:monoolein (DODAX:
MO) system are explored.
</P><P>
Liposomes show great potential as siRNA delivery systems, being safe nanocarriers to protect nucleic
acids in circulation, extend their half-life time, target specific cells and reduce off-target effects. Nevertheless,
several issues related to delivery must be overcome before RNAi therapies reach their full
potential, namely target-cell specificity and endosomal escape. Understanding the relationship between
biophysical properties and biological performance is an essential step in the gene therapy field.
Funder
FCT Scholarship
Fundação para a Ciência e a Tecnologia (FCT)
Publisher
Bentham Science Publishers Ltd.
Subject
Clinical Biochemistry,Drug Discovery,Pharmacology,Molecular Medicine
Cited by
16 articles.
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