{"id":77853,"date":"2018-03-09T23:23:25","date_gmt":"2018-03-09T23:23:25","guid":{"rendered":"https:\/\/www.deberes.net\/tesis\/sin-categoria\/terapia-genica-de-la-deficiencia-de-piruvato-quinasa\/"},"modified":"2018-03-09T23:23:25","modified_gmt":"2018-03-09T23:23:25","slug":"terapia-genica-de-la-deficiencia-de-piruvato-quinasa","status":"publish","type":"post","link":"https:\/\/www.deberes.net\/tesis\/sin-categoria\/terapia-genica-de-la-deficiencia-de-piruvato-quinasa\/","title":{"rendered":"Terapia g\u00e9nica de la deficiencia de piruvato quinasa"},"content":{"rendered":"<h2>Tesis doctoral de <strong> N\u00e9stor Wencelao Meza <\/strong><\/h2>\n<p>La deficiencia en piruvato kinasa eritrocitaria humana (hrpk), un desorden autos\u00f3mico recesivo producido por mutaciones en el gen pklr, es una de las causas m\u00e1s comunes de anemia hemol\u00edtica cr\u00f3nica no esferoc\u00edtica. La expresi\u00f3n transg\u00e9nica de la rpk en precursores eritropoy\u00e9ticos deficientes puede resultar una terapia alternativa en aquellos pacientes con s\u00edntomas severos. En este trabajo hemos construido vectores retrovirales que expresan el cdna de la hrpk y la egfp en un \u00fanico rna mensajero, separados por un ires eucri\u00f3tico (sf11rpxeg). L\u00edneas celulares murinas 3t3 y mel (c\u00e9lulas de eritroleucemia murina ) y humanas hela y 293t fueron transducidas con sobrenadantes retrovirales infectivos. an\u00e1lisis de western-blot y de facs demostraron la expresi\u00f3n estable de egfp y hrpk, respectivamente, con expresi\u00f3n estable del transg\u00e9n durante m\u00e1s de 20 semanas en cultivo. la expresi\u00f3n de los trasngenes no vari\u00f3 significativamente durante el tiempo del cultivo. Adem\u00e1s, la inducci\u00f3n a eritrodiferenciaci\u00f3n de c\u00e9lulas mel no modific\u00f3 la expresi\u00f3n de hrpk ni de egfp. Posteriormente, progenitores hematopoy\u00e9ticos lin-sca-1+ fueron transducidos in vitro con sf11rpkxeg y transplantado s en receptores normales letalmente irradiados. tres meses post-transplante, una elevada expresi\u00f3n de hrpk y egfp fue observada en leucocitos, plaquetas y eritrocitos maduros. Estos resultados indican que los vectores retrovirales pueden ser una sistema efectivo para abordar la correcci\u00f3n fenot\u00edpica de los defectos eritropoy\u00e9ticos asociados a la deficiencia de piruvato kinasa eritrocitaria humana mediante de protocolos de terapia g\u00e9nica.<\/p>\n<p>&nbsp;<\/p>\n<h3>Datos acad\u00e9micos de la tesis doctoral \u00ab<strong>Terapia g\u00e9nica de la deficiencia de piruvato quinasa<\/strong>\u00ab<\/h3>\n<ul>\n<li><strong>T\u00edtulo de la tesis:<\/strong>\u00a0 Terapia g\u00e9nica de la deficiencia de piruvato quinasa <\/li>\n<li><strong>Autor:<\/strong>\u00a0 N\u00e9stor Wencelao Meza <\/li>\n<li><strong>Universidad:<\/strong>\u00a0 Complutense de Madrid<\/li>\n<li><strong>Fecha de lectura de la tesis:<\/strong>\u00a0 16\/12\/2005<\/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> Bautista Santa Cruz Jos\u00e9 Manuel<\/li>\n<\/ul>\n<\/li>\n<li><strong>Tribunal<\/strong>\n<ul>\n<li>Presidente del tribunal: amando Garrido pertierra <\/li>\n<li>florinda Gilsanz rodriguez (vocal)<\/li>\n<li>Jorge Barquinero manez (vocal)<\/li>\n<li>Juan  Antonio Bueren roncero (vocal)<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p>&nbsp;<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Tesis doctoral de N\u00e9stor Wencelao Meza La deficiencia en piruvato kinasa eritrocitaria humana (hrpk), un desorden autos\u00f3mico recesivo producido por [&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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