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Issue 7, 2011
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Safe and efficient in vitro and in vivo gene delivery: tripodal cationic lipids with programmed biodegradability

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Abstract

The therapeutic use of nucleic acids has long been heralded as a panacea of medicinal opportunity, a vision enhanced by the introduction of RNA interference technology. The Achilles heel of such an approach is the in vivo delivery of the desired nucleic acid into cells, a practice that lacks selectivity, safety and/or efficiency. Herein we report the safe and efficacious in vitro and in vivo delivery of nucleic acids using tripodal biodegradable cationic lipids. Toxicity reduction and transfection potency of these novel amphiphiles were addressed by designing the compounds to undergo complete intracellular degradation thereby enhancing cargo release while minimising toxicity and potential tissue accumulation. Compounds demonstrated high-efficiency in transfecting DNA into cells both in vitro and in vivo with no signs of toxicity, thus potentially offering a safer alternative to viral transfection for gene therapy application.

Graphical abstract: Safe and efficient in vitro and in vivo gene delivery: tripodal cationic lipids with programmed biodegradability

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Publication details

The article was received on 27 Sep 2010, accepted on 08 Nov 2010 and first published on 20 Dec 2010


Article type: Paper
DOI: 10.1039/C0JM03241G
Citation: J. Mater. Chem., 2011,21, 2154-2158
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    Safe and efficient in vitro and in vivo gene delivery: tripodal cationic lipids with programmed biodegradability

    A. Unciti-Broceta, L. Moggio, K. Dhaliwal, L. Pidgeon, K. Finlayson, C. Haslett and M. Bradley, J. Mater. Chem., 2011, 21, 2154
    DOI: 10.1039/C0JM03241G

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