{"id":9344,"date":"2022-06-14T20:42:45","date_gmt":"2022-06-14T20:42:45","guid":{"rendered":"https:\/\/dupmecp2.eu\/reversible-phenotype-in-humanized-mouse-model\/"},"modified":"2023-01-13T11:44:59","modified_gmt":"2023-01-13T11:44:59","slug":"fenotipo-reversible-en-modelo-de-raton-humanizado","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/es\/fenotipo-reversible-en-modelo-de-raton-humanizado\/","title":{"rendered":"Fenotipo reversible en un modelo de rat\u00f3n humanizado"},"content":{"rendered":"<p><strong>Shao et al. del grupo del Prof. Zoghbi (Baylor College of Medicine, Texas, EE.UU.) publicaron un art\u00edculo en Science Translational medicine titulado \"Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome\". <\/strong><\/p>\n\n\n\n<p><strong>Cada especie tiene sus propios genes. Los genes codifican prote\u00ednas que son exclusivas de cada especie. Es posible que las prote\u00ednas humanas y de rat\u00f3n sean ligeramente diferentes. El modelo utilizado en el trabajo presentado, denominado \"modelo humanizado\", permite evaluar mejor la actividad de los oligonucle\u00f3tidos antisentido (ASO) espec\u00edficamente sobre la prote\u00edna humana producida por un rat\u00f3n. Por lo tanto, est\u00e1 m\u00e1s cerca del futuro uso de ASO en humanos.<\/strong><\/p>\n\n\n\n<p><strong>Este art\u00edculo presenta los resultados obtenidos tras la inyecci\u00f3n intracerebroventricular de oligonucle\u00f3tidos antisentido en este modelo de rat\u00f3n de duplicaci\u00f3n g\u00e9nica \"MECP2 humanizada\" en el que el rat\u00f3n porta dos alelos MECP2 humanos y ning\u00fan alelo end\u00f3geno de rat\u00f3n.<\/strong><\/p>\n\n\n\n<p><strong>La administraci\u00f3n redujo eficazmente la expresi\u00f3n de MeCP2 en todo el cerebro de estos ratones, alivi\u00f3 varios d\u00e9ficits de comportamiento y restaur\u00f3 la expresi\u00f3n de algunos genes regulados por MeCP2 de forma dependiente de la dosis y sin toxicidad alguna.<\/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>RESUMEN DE LA PUBLICACI\u00d3N<\/strong><\/p>\n\n\n\n<p>Muchos trastornos de la discapacidad intelectual se deben a variaciones en el n\u00famero de copias y, hasta la fecha, no se han probado opciones de tratamiento para esta clase de enfermedades. El s\u00edndrome de duplicaci\u00f3n MECP2 (MDS) es uno de los reordenamientos gen\u00f3micos m\u00e1s comunes en varones y resulta de duplicaciones que abarcan el locus del gen de la prote\u00edna de uni\u00f3n metil-CpG 2 (MECP2). Anteriormente demostramos que la terapia con oligonucle\u00f3tidos antisentido (ASO) puede reducir la cantidad de prote\u00edna MeCP2 en un modelo de rat\u00f3n de SMD y revertir las caracter\u00edsticas de la enfermedad.<\/p>\n\n\n\n<p>Este modelo de rat\u00f3n con SMD, sin embargo, portaba un alelo humano transg\u00e9nico y un alelo de rat\u00f3n, estando este \u00faltimo protegido de la orientaci\u00f3n MECP2-ASO espec\u00edfica humana.<\/p>\n\n\n\n<p>Debido a que MeCP2 es una prote\u00edna sensible a la dosis, el ASO debe ser titulado de tal manera que la cantidad de MeCP2 no se reduzca demasiado, lo que causar\u00eda el s\u00edndrome de Rett. Por lo tanto, generamos un modelo de SMD \"MECP2 humanizado\" que porta dos alelos MECP2 humanos y ning\u00fan alelo end\u00f3geno de rat\u00f3n. La inyecci\u00f3n intracerebroventricular del MECP2-ASO regul\u00f3 eficazmente a la baja la expresi\u00f3n de MeCP2 en todo el cerebro de estos ratones. Adem\u00e1s, el MECP2-ASO mitig\u00f3 varios d\u00e9ficits de comportamiento y restaur\u00f3 la expresi\u00f3n de genes seleccionados regulados por MeCP2 de una manera dependiente de la dosis sin ninguna toxicidad. La administraci\u00f3n de MECP2-ASO en el sistema nervioso central es, por tanto, bien tolerada y beneficiosa en este modelo de rat\u00f3n y proporciona un enfoque traducible que podr\u00eda ser factible para el tratamiento de los SMD.<\/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\"><a class=\"wp-block-button__link has-white-color has-text-color 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 et al. from Prof. Zoghbi&rsquo;s group (Baylor College of Medicine, Texas, USA) published an article in Science Translational medicine entitled \u00ab\u00a0Antisense oligonucleotide therapy in a humanized mouse model of [&hellip;]<\/p>","protected":false},"author":1,"featured_media":11723,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[88],"tags":[],"class_list":["post-9344","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-publications-en"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v25.8 - 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