Effects of a Novel Archaeal Tetraether-Based Colipid on the In Vivo Gene Transfer Activity of Two Cationic Amphiphiles

Gene therapy for treating inherited diseases like cystic fibrosis might be achieved using multimodular nonviral lipid-based systems. To date, most optimizations have concerned cationic lipids rather than colipids. In this study, an original archaeal tetraether derivative was used as a colipid in com...

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Veröffentlicht in:	Molecular pharmaceutics 2014-09, Vol.11 (9), p.2973-2988
Hauptverfasser:	Le Gall, Tony, Barbeau, Julie, Barrier, Sylvain, Berchel, Mathieu, Lemiègre, Loïc, Jeftić, Jelena, Meriadec, Cristelle, Artzner, Franck, Gill, Deborah R, Hyde, Stephen C, Férec, Claude, Lehn, Pierre, Jaffrès, Paul-Alain, Benvegnu, Thierry, Montier, Tristan
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Archaea - chemistry Cations - chemistry Chemical Sciences DNA - administration & dosage DNA - chemistry Ethers - chemistry Female Gene Transfer Techniques Lipids - chemistry Liposomes - chemistry Mice Organic chemistry Surface-Active Agents - chemistry Transfection - methods
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container_title	Molecular pharmaceutics
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creator	Le Gall, Tony Barbeau, Julie Barrier, Sylvain Berchel, Mathieu Lemiègre, Loïc Jeftić, Jelena Meriadec, Cristelle Artzner, Franck Gill, Deborah R Hyde, Stephen C Férec, Claude Lehn, Pierre Jaffrès, Paul-Alain Benvegnu, Thierry Montier, Tristan
description	Gene therapy for treating inherited diseases like cystic fibrosis might be achieved using multimodular nonviral lipid-based systems. To date, most optimizations have concerned cationic lipids rather than colipids. In this study, an original archaeal tetraether derivative was used as a colipid in combination with one or the other of two monocationic amphiphiles. The liposomes obtained, termed archaeosomes, were characterized regarding lipid self-assembling properties, macroscopic/microscopic structures, DNA condensation/neutralization/relaxation abilities, and colloidal stability in the presence of serum. In addition, gene transfer experiments were conducted in mice with lipid/DNA complexes being administered via systemic or local delivery routes. Altogether, the results showed that the tetraether colipid can provide complexes with different in vivo transfection abilities depending on the lipid combination, the lipid/colipid molar ratio, and the administration route. This original colipid appears thus as an innovative modular platform endowed with properties possibly beneficial for fine-tuning of in vivo lipofection and other biomedical applications.
doi_str_mv	10.1021/mp4006276
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Pharmaceutics</addtitle><description>Gene therapy for treating inherited diseases like cystic fibrosis might be achieved using multimodular nonviral lipid-based systems. To date, most optimizations have concerned cationic lipids rather than colipids. In this study, an original archaeal tetraether derivative was used as a colipid in combination with one or the other of two monocationic amphiphiles. The liposomes obtained, termed archaeosomes, were characterized regarding lipid self-assembling properties, macroscopic/microscopic structures, DNA condensation/neutralization/relaxation abilities, and colloidal stability in the presence of serum. In addition, gene transfer experiments were conducted in mice with lipid/DNA complexes being administered via systemic or local delivery routes. 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Pharmaceutics</addtitle><date>2014-09-02</date><risdate>2014</risdate><volume>11</volume><issue>9</issue><spage>2973</spage><epage>2988</epage><pages>2973-2988</pages><issn>1543-8384</issn><eissn>1543-8392</eissn><abstract>Gene therapy for treating inherited diseases like cystic fibrosis might be achieved using multimodular nonviral lipid-based systems. To date, most optimizations have concerned cationic lipids rather than colipids. In this study, an original archaeal tetraether derivative was used as a colipid in combination with one or the other of two monocationic amphiphiles. The liposomes obtained, termed archaeosomes, were characterized regarding lipid self-assembling properties, macroscopic/microscopic structures, DNA condensation/neutralization/relaxation abilities, and colloidal stability in the presence of serum. In addition, gene transfer experiments were conducted in mice with lipid/DNA complexes being administered via systemic or local delivery routes. 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subjects	Animals Archaea - chemistry Cations - chemistry Chemical Sciences DNA - administration & dosage DNA - chemistry Ethers - chemistry Female Gene Transfer Techniques Lipids - chemistry Liposomes - chemistry Mice Organic chemistry Surface-Active Agents - chemistry Transfection - methods
title	Effects of a Novel Archaeal Tetraether-Based Colipid on the In Vivo Gene Transfer Activity of Two Cationic Amphiphiles
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