Multifunctional nanocomplexes for gene transfer and gene therapy
Identifieur interne : 001691 ( Main/Exploration ); précédent : 001690; suivant : 001692Multifunctional nanocomplexes for gene transfer and gene therapy
Auteurs : Stephen L. Hart [Royaume-Uni]Source :
- Cell Biology and Toxicology [ 0742-2091 ] ; 2010-02-01.
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Abstract
Abstract: DNA formulated into aggregates with polycationic reagents are referred to by a variety of terms including non-viral vectors, synthetic vectors, lipoplexes, polyplexes and more recently nanoparticles. The capacity for delivery of multiple genes, genomic-sized constructs and siRNA delivery, with a diversity of possible formulations, as well as the possibilities of improved efficiency of in vivo gene deliveries, means that nanoparticles, or nanocomplexes to reflect self-assembling systems, will be investigated with increasing vigour in the coming years. This review briefly outlines the applications and challenges for nanoparticle technologies in the field of gene therapy then focuses on the development of a specific kind of formulation, receptor-targeted nanocomplex (RTN), that we have found to be particularly useful in our gene therapy research. An overriding guiding concept that has emerged in the development of synthetic nanodelivery systems is the idea to develop formulations and structures that mimic viruses, whilst retaining the safety elements of synthetic, non-viral systems. RTNs have been optimised and developed for airway epithelial transfection, leading towards gene therapy for cystic fibrosis and for vascular transfection in vein grafts used in bypass surgery. The modular design of the RTN platform further allows for the testing of specific hypotheses relating to the structure and functional role of components in the formation of stable particles and in the transfection pathway, leading to their ultimate disassembly in the nucleus.
Url:
DOI: 10.1007/s10565-009-9141-y
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Hart</name><affiliation wicri:level="3"><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Wolfson Centre for Gene Therapy of Childhood Disease, UCL Institute of Child Health, London</wicri:regionArea><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region></placeName></affiliation><affiliation wicri:level="3"><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Genex Biosystems Ltd., London</wicri:regionArea><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region></placeName></affiliation><affiliation wicri:level="3"><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Molecular Immunology Unit, UCL Institute of Child Health, 30 Guilford Street, WC1N 1EH, London</wicri:regionArea><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region></placeName></affiliation><affiliation wicri:level="1"><country wicri:rule="url">Royaume-Uni</country></affiliation></author></analytic><monogr></monogr><series><title level="j">Cell Biology and Toxicology</title><title level="j" type="sub">An International Journal Devoted to Research at the Cellular Level</title><title level="j" type="abbrev">Cell Biol Toxicol</title><idno type="ISSN">0742-2091</idno><idno type="eISSN">1573-6822</idno><imprint><publisher>Springer Netherlands</publisher><pubPlace>Dordrecht</pubPlace><date type="published" when="2010-02-01">2010-02-01</date><biblScope unit="volume">26</biblScope><biblScope unit="issue">1</biblScope><biblScope unit="page" from="69">69</biblScope><biblScope unit="page" to="81">81</biblScope></imprint><idno type="ISSN">0742-2091</idno></series></biblStruct></sourceDesc><seriesStmt><idno type="ISSN">0742-2091</idno></seriesStmt></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Gene therapy</term><term>Nanoparticle</term><term>Non-viral</term><term>Receptor-mediated</term><term>Targeted</term><term>Transfection</term></keywords></textClass><langUsage><language ident="en">en</language></langUsage></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Abstract: DNA formulated into aggregates with polycationic reagents are referred to by a variety of terms including non-viral vectors, synthetic vectors, lipoplexes, polyplexes and more recently nanoparticles. The capacity for delivery of multiple genes, genomic-sized constructs and siRNA delivery, with a diversity of possible formulations, as well as the possibilities of improved efficiency of in vivo gene deliveries, means that nanoparticles, or nanocomplexes to reflect self-assembling systems, will be investigated with increasing vigour in the coming years. This review briefly outlines the applications and challenges for nanoparticle technologies in the field of gene therapy then focuses on the development of a specific kind of formulation, receptor-targeted nanocomplex (RTN), that we have found to be particularly useful in our gene therapy research. An overriding guiding concept that has emerged in the development of synthetic nanodelivery systems is the idea to develop formulations and structures that mimic viruses, whilst retaining the safety elements of synthetic, non-viral systems. RTNs have been optimised and developed for airway epithelial transfection, leading towards gene therapy for cystic fibrosis and for vascular transfection in vein grafts used in bypass surgery. The modular design of the RTN platform further allows for the testing of specific hypotheses relating to the structure and functional role of components in the formation of stable particles and in the transfection pathway, leading to their ultimate disassembly in the nucleus.</div></front></TEI><affiliations><list><country><li>Royaume-Uni</li></country><region><li>Angleterre</li><li>Grand Londres</li></region><settlement><li>Londres</li></settlement></list><tree><country name="Royaume-Uni"><region name="Angleterre"><name sortKey="Hart, Stephen L" sort="Hart, Stephen L" uniqKey="Hart S" first="Stephen L." last="Hart">Stephen L. Hart</name></region><name sortKey="Hart, Stephen L" sort="Hart, Stephen L" uniqKey="Hart S" first="Stephen L." last="Hart">Stephen L. Hart</name><name sortKey="Hart, Stephen L" sort="Hart, Stephen L" uniqKey="Hart S" first="Stephen L." last="Hart">Stephen L. Hart</name><name sortKey="Hart, Stephen L" sort="Hart, Stephen L" uniqKey="Hart S" first="Stephen L." last="Hart">Stephen L. Hart</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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