{"id":13941,"date":"2018-03-09T09:00:26","date_gmt":"2018-03-09T09:00:26","guid":{"rendered":"https:\/\/www.deberes.net\/tesis\/sin-categoria\/terapa%c2%ada-genica-experimental-desarrollo-de-vectores-no-virales-para-la-transferencia-del-gen-de-la-fibrosis-qua%c2%adstica\/"},"modified":"2018-03-09T09:00:26","modified_gmt":"2018-03-09T09:00:26","slug":"terapa%c2%ada-genica-experimental-desarrollo-de-vectores-no-virales-para-la-transferencia-del-gen-de-la-fibrosis-qua%c2%adstica","status":"publish","type":"post","link":"https:\/\/www.deberes.net\/tesis\/quimica\/terapa%c2%ada-genica-experimental-desarrollo-de-vectores-no-virales-para-la-transferencia-del-gen-de-la-fibrosis-qua%c2%adstica\/","title":{"rendered":"Terap\u00edag\u00e9nica experimental: desarrollo de vectores no virales para la transferencia del gen de la fibrosis qu\u00edstica"},"content":{"rendered":"<h2>Tesis doctoral de <strong> Marta Benet Gim\u00e9nez <\/strong><\/h2>\n<p>La fibrosis qu\u00edstica es una alteraci\u00f3n hereditaria de car\u00e1cter recesivo que afecta a 1 de cada 2000 individuos cauc\u00e1sicos y se origina por mutaciones en el gen regulador de la conductancia de transmembrana responsable de la fibrosis qu\u00edstica cftr, que codifica  un canal de cloro regulado por ampc. El objetivo principal de este trabajo ha sido transferir el gen de la fibrosis qu\u00edstica humano a c\u00e9lulas cuyo nivel de expresi\u00f3n es bajo o nulo, con el fin de inducir la expresi\u00f3n del gen cftr humano e instaurar la funcionalidad de la prote\u00edna cftr. Para este trabajo, hemos utilizado los lipoplejos (complejos l\u00edpido cationico: adn) y poliplejos (complejos pol\u00edmero cati\u00f3nico: adn) como vectores no virales de transferncia g\u00e9ncia, y el gen humano de la fibrosis qu\u00edstica, cftr, como gen terap\u00e9utico. Haciendo uan breve s\u00edntesis del trabajo presentado en este manuscrito pod\u00edamos decir, que son la utilizaci\u00f3n de los vectores no virales, como los lipoplejos y poliplejos:  i) se ha consegudio transferir y expresar el gen cftr en c\u00e9lulas que normalmente no lo expresan y los niveles de expresi\u00f3n alcanzados han sido superiores al de las c\u00e9lulas que lo expresan de forma normal.  ii) se ha instaurado una prote\u00edna nueva para la c\u00e9lula y funcionalmente activa, en c\u00e9lulas cuya actividad funcional de la prote\u00edna previa a la transferncia, era pr\u00e1cticamente nula.  iii) en estudios in vivo se ha conseguido transferir el gen cftr a las v\u00edas a\u00e9reas, alcanzando niveles de expresi\u00f3n relativamente elevados en este territorio, sin llegar a la circulaci\u00f3n sistem\u00e1tica.  iv) en los estudios de tratamiento m\u00faltiple, se ha consegudio la persistencia de expresi\u00f3n del gen en el tiempo durante per\u00edodos relativamente prolongados.<\/p>\n<p>&nbsp;<\/p>\n<h3>Datos acad\u00e9micos de la tesis doctoral \u00ab<strong>Terap\u00edag\u00e9nica experimental: desarrollo de vectores no virales para la transferencia del gen de la fibrosis qu\u00edstica<\/strong>\u00ab<\/h3>\n<ul>\n<li><strong>T\u00edtulo de la tesis:<\/strong>\u00a0 Terap\u00edag\u00e9nica experimental: desarrollo de vectores no virales para la transferencia del gen de la fibrosis qu\u00edstica <\/li>\n<li><strong>Autor:<\/strong>\u00a0 Marta Benet Gim\u00e9nez <\/li>\n<li><strong>Universidad:<\/strong>\u00a0 Universitat de val\u00e9ncia (estudi general)<\/li>\n<li><strong>Fecha de lectura de la tesis:<\/strong>\u00a0 22\/11\/2001<\/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>Salvador Francisco Ali\u00f1o Pellicer<\/li>\n<\/ul>\n<\/li>\n<li><strong>Tribunal<\/strong>\n<ul>\n<li>Presidente del tribunal: Santiago Grisolia <\/li>\n<li>Fernando Revert ros (vocal)<\/li>\n<li>Carlos Ciudad g\u00f3mez (vocal)<\/li>\n<li>guillermo Saez tormo (vocal)<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p>&nbsp;<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Tesis doctoral de Marta Benet Gim\u00e9nez La fibrosis qu\u00edstica es una alteraci\u00f3n hereditaria de car\u00e1cter recesivo que afecta a 1 [&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 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