{"id":114679,"date":"2018-03-11T10:42:46","date_gmt":"2018-03-11T10:42:46","guid":{"rendered":"https:\/\/www.deberes.net\/tesis\/sin-categoria\/design-of-a-non-viral-vector-based-on-solid-lipid-nanoparticles-for-the-treatment-of-fabry-disease\/"},"modified":"2018-03-11T10:42:46","modified_gmt":"2018-03-11T10:42:46","slug":"design-of-a-non-viral-vector-based-on-solid-lipid-nanoparticles-for-the-treatment-of-fabry-disease","status":"publish","type":"post","link":"https:\/\/www.deberes.net\/tesis\/evaluacion-de-farmacos\/design-of-a-non-viral-vector-based-on-solid-lipid-nanoparticles-for-the-treatment-of-fabry-disease\/","title":{"rendered":"Design of a non-viral vector based on solid lipid nanoparticles for the treatment of fabry disease"},"content":{"rendered":"<h2>Tesis doctoral de <strong> Aritz Perez Ruiz De Garibay <\/strong><\/h2>\n<p>El objetivo de esta tesis ha sido el desarrollo de un vector no viral basado en nanopart\u00edculas s\u00f3lidas lip\u00eddicas (sln) para el tratamiento de la enfermedad de fabry mediante terapia g\u00e9nica.Inicialmente, se estudiaron vectores conteniendo el pl\u00e1smido que codifica para la \u00c2\u00bf-galactosidasa a, enzima deficiente en la enfermedad de fabry, y basados en sln y protamina o dextrano y protamina en c\u00e9lulas hep\u00e1ticas humanas (hepg2). Este estudio demostr\u00f3 por primera vez un aumento de hasta 12 veces en la producci\u00f3n de la \u00c2\u00bf-galactosidasa a mediante la utilizaci\u00f3n de vectores no virales en c\u00e9lulas hepg2. Adem\u00e1s, se observ\u00f3 que el porcentaje de c\u00e9lulas transfectadas no era proporcional a la cantidad de prote\u00edna sintetizada, debido al efecto de la protamina en la transcripci\u00f3n, as\u00ed como por la presencia de se\u00f1ales de localizaci\u00f3n nuclear en este compuesto.Estos estudios se trasladaron posteriormente a un modelo celular de la enfermedad de fabry, las c\u00e9lulas imfe1. En estas c\u00e9lulas en las que la producci\u00f3n de \u00c2\u00bf-galactosidasa a est\u00e1 disminuida, se consigui\u00f3 aumentar hasta 4 veces la producci\u00f3n de la misma. Los resultados m\u00e1s altos se obtuvieron con vectores basados en sln que conten\u00edan \u00e1cido hialur\u00f3nico y protamina.Finalmente, se analizaron los posibles mecanismos de entrada de 3 de los vectores no virales. Para ello se utilizaron inhibidores de la endocitosis en c\u00e9lulas hela, en los que estaban presenten gran parte de los mecanismos utilizados por las formulaciones basadas en sln. Tras este estudio se determin\u00f3 que los vectores dna-sln eran capaces de utilizar m\u00faltiples v\u00edas. Sin embargo, las formulaciones que contienen protamina no necesitan las v\u00edas dependientes de dinamina para transfectar las c\u00e9lulas. Adem\u00e1s, la potenciaci\u00f3n de la macropinocitosis resulta en un aumento de la transfecci\u00f3n con vectores que contienen dextrano. A falta de una confirmaci\u00f3n en un modelo animal de la enfermedad, estos resultados avalar\u00edan la capacidad de los vectores no virales basados en sln como futura alternativa en el tratamiento de la enfermedad de fabry.<\/p>\n<p>&nbsp;<\/p>\n<h3>Datos acad\u00e9micos de la tesis doctoral \u00ab<strong>Design of a non-viral vector based on solid lipid nanoparticles for the treatment of fabry disease<\/strong>\u00ab<\/h3>\n<ul>\n<li><strong>T\u00edtulo de la tesis:<\/strong>\u00a0 Design of a non-viral vector based on solid lipid nanoparticles for the treatment of fabry disease <\/li>\n<li><strong>Autor:<\/strong>\u00a0 Aritz Perez Ruiz De Garibay <\/li>\n<li><strong>Universidad:<\/strong>\u00a0 Pa\u00eds vasco\/euskal herriko unibertsitatea<\/li>\n<li><strong>Fecha de lectura de la tesis:<\/strong>\u00a0 23\/09\/2013<\/li>\n<\/ul>\n<p>&nbsp;<\/p>\n<h3>Direcci\u00f3n y tribunal<\/h3>\n<ul>\n<li><strong>Director de la tesis<\/strong>\n<ul>\n<li>M\u00aaangeles Solinis Aspiazu<\/li>\n<\/ul>\n<\/li>\n<li><strong>Tribunal<\/strong>\n<ul>\n<li>Presidente del tribunal: M\u00aa bego\u00f1a Calvo hernaez <\/li>\n<li>renate Fuches &#8212; (vocal)<\/li>\n<li>pilar Prieto peraita (vocal)<\/li>\n<li>Mar\u00eda  Luisa Gonzalez rodriguez (vocal)<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p>&nbsp;<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Tesis doctoral de Aritz Perez Ruiz De Garibay El objetivo de esta tesis ha sido el desarrollo de un vector [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"site-sidebar-layout":"default","site-content-layout":"","ast-site-content-layout":"","site-content-style":"default","site-sidebar-style":"default","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","theme-transparent-header-meta":"","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"default","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-gradient":""},"tablet":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-gradient":""},"mobile":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-gradient":""}},"ast-content-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-gradient":""},"tablet":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-gradient":""},"mobile":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-gradient":""}},"footnotes":""},"categories":[2445,12909,3304],"tags":[227222,94617,32144,177809,227224,227223],"class_list":["post-114679","post","type-post","status-publish","format-standard","hentry","category-evaluacion-de-farmacos","category-pais-vasco-euskal-herriko-unibertsitatea","category-preparacion-de-farmacos","tag-aritz-perez-ruiz-de-garibay","tag-maria-luisa-gonzalez-rodriguez","tag-ma-begona-calvo-hernaez","tag-maangeles-solinis-aspiazu","tag-pilar-prieto-peraita","tag-renate-fuches"],"_links":{"self":[{"href":"https:\/\/www.deberes.net\/tesis\/wp-json\/wp\/v2\/posts\/114679","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.deberes.net\/tesis\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.deberes.net\/tesis\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.deberes.net\/tesis\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.deberes.net\/tesis\/wp-json\/wp\/v2\/comments?post=114679"}],"version-history":[{"count":0,"href":"https:\/\/www.deberes.net\/tesis\/wp-json\/wp\/v2\/posts\/114679\/revisions"}],"wp:attachment":[{"href":"https:\/\/www.deberes.net\/tesis\/wp-json\/wp\/v2\/media?parent=114679"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.deberes.net\/tesis\/wp-json\/wp\/v2\/categories?post=114679"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.deberes.net\/tesis\/wp-json\/wp\/v2\/tags?post=114679"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}