{"id":15973,"date":"2022-06-14T20:42:45","date_gmt":"2022-06-14T20:42:45","guid":{"rendered":"https:\/\/dupmecp2.eu\/fenotipo-reversibile-in-un-modello-di-topo-umanizzato\/"},"modified":"2023-04-17T16:11:06","modified_gmt":"2023-04-17T16:11:06","slug":"fenotipo-reversible-en-un-modelo-de-superficie-humanizada","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/es\/fenotipo-reversible-en-un-modelo-de-superficie-humanizada\/","title":{"rendered":"Fenotipo reversible en un modelo de techo humanizado"},"content":{"rendered":"<p><strong>Shao y sus colaboradores del grupo de la profesora Zoghbi del Baylor College of Medicine de Texas (EE.UU.) han publicado un art\u00edculo en la revista Science Translational Medicine titulado \"Terapia con oligonucle\u00f3tidos antisensibles en un modelo de topo umanizado con S\u00edndrome de Duplicaci\u00f3n de MECP2\". <\/strong><\/p>\n\n\n\n<p><strong>Cada especie tiene sus propios genes. Los genes codifican las prote\u00ednas que son \u00fanicas y t\u00edpicas de cada especie. E' possibile che le proteine umane o di topo siano leggermente diverse. Il modello utilizzato in questo articolo, chiamato \"modello umanizzato\", permette di valutare meglio l'attivit\u00e0 degli oligonucleoti antisenti, soprattutto in relazione alle proteine umane prodotte dal topo. E' quindi piu' vicino all'uso futuro di oligonucleotidi antisenso nell'uomo.<\/strong><\/p>\n\n\n\n<p><strong>Este art\u00edculo presenta los resultados obtenidos mediante la ingesta intracerebroventricular de oligonucle\u00f3tidos antisensos en este modelo de topo con \"MECP2 umanizado\" en el que el topo porta dos alelos humanos de MECP2 y ning\u00fan alelo end\u00f3geno.<\/strong><\/p>\n\n\n\n<p><strong>La somministraci\u00f3n ha reducido la expresi\u00f3n de MeCP2 en el cerebro superior, aliviando el d\u00e9ficit conductual y aumentando la expresi\u00f3n de algunos genes en grado de regulaci\u00f3n.<\/strong><\/p>\n\n\n\n<p>Terapia con oligonucle\u00f3tidos antisentido en un modelo de rat\u00f3n humanizado del s\u00edndrome de duplicaci\u00f3n MECP2, Shao et al.<em>Sci. Transl. Med<\/em>. <strong>13<\/strong>, 7785 (2021)<\/p>\n\n\n\n<p><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p><\/p>\n\n\n\n<p><strong>REESCRITURA DEL ART\u00cdCULO:<\/strong><\/p>\n\n\n\n<p>Muchas discapacidades intelectuales se deben a una variaci\u00f3n en el n\u00famero de copias del gen, y actualmente no existen opciones de tratamiento probadas para esta clase de enfermedad. El S\u00edndrome de Duplicaci\u00f3n MECP2 (SDM) es una de las reordenaciones gen\u00f3micas m\u00e1s comunes en los masquios y resulta de la duplicaci\u00f3n del gen que codifica la prote\u00edna MECP2. In passato, abbiamo dimostrato che la terapia con oligonucleotidi antisenso (ASO) puo' ridurre la quantita' di proteina MeCP2 nei topi modello e invertire il manifesto dei sintomi.<\/p>\n\n\n\n<p>Questo modello di topo porta un allele umano transgenico e un allele di topo, con quest'ultimo protetto dall'azione dell'oligonucleotide antisenso MECP2 specifico. Dado que la concentraci\u00f3n de la prote\u00edna MeCP2 es un par\u00e1metro muy importante, la cantidad de oligonucle\u00f3tidos antisensores debe dosificarse de forma que la cantidad de MeCP2 no disminuya demasiado, provocando as\u00ed el s\u00edndrome de Rett. Por esta raz\u00f3n, hemos generado un modelo de topo con \"MECP2 umanizzato\" que porta dos alelos humanos de MECP2 y ning\u00fan alelo end\u00f3geno de topo. L'iniezione intracerebroventricolare di oligonucleotide antisenso riduce in maniera efficace la quantita' di proteina MECP2 prodotta nel cervello dei topi. Adem\u00e1s, este tratamiento es capaz de mitigar varios d\u00e9ficits de comportamiento y restaurar la expresi\u00f3n de ciertos genes reguladores de MeCP2 de una manera dependiente de su dosificaci\u00f3n y sin ninguna toxicidad. La administraci\u00f3n de oligonucle\u00f3tidos antisensos a nivel del sistema nervioso central es, por tanto, ben\u00e9fica y beneficiosa en el modelo de topo y ofrece un enfoque que podr\u00eda tener \u00e9xito tambi\u00e9n en la curaci\u00f3n del S\u00edndrome de Duplicaci\u00f3n de MECP2 en el hombre.<\/p>\n\n\n\n<div style=\"height:100px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<div class=\"wp-block-buttons is-layout-flex wp-block-buttons-is-layout-flex\">\n<div class=\"wp-block-button is-style-fill\"><a class=\"wp-block-button__link has-background wp-element-button\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/33658357\/\" style=\"border-radius:14px;background-color:#f7a13f\" target=\"_blank\" rel=\"noreferrer noopener\">A la publicaci\u00f3n<\/a><\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>Shao e i suoi collaboratori del gruppo del Prof.ssa Zoghbi al Baylor College of Medicine in Texas, USA hanno pubblicato un articolo sulla rivista Science Translational Medicine intitolato \u201cTerapia con [&hellip;]<\/p>","protected":false},"author":1,"featured_media":13607,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[139],"tags":[],"class_list":["post-15973","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-pubblicazioni"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v25.8 - 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