{"id":15078,"date":"2022-06-14T20:42:45","date_gmt":"2022-06-14T20:42:45","guid":{"rendered":"https:\/\/dupmecp2.eu\/fenotipo-reversible-en-un-modelo-humanizado-en-ratones\/"},"modified":"2023-02-17T20:01:00","modified_gmt":"2023-02-17T20:01:00","slug":"fenotipo-reversible-en-un-modelo-humanizado-en-ratones","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/de\/fenotipo-reversible-en-un-modelo-humanizado-en-ratones\/","title":{"rendered":"Reversible Fenotipo en un modelo humanizado en ratones"},"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 \u00a0\u00bb Terapia con oligonucle\u00f3tidos antisentido en un modelo humanizado del s\u00edndrome de duplicaci\u00f3n MECP2 en ratones\u201d. <\/strong><\/p>\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 \u00ab\u00a0modelo humanizado\u00a0\u00bb 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<p><strong>Este art\u00edculo presenta los resultados obtenidos tras la inyecci\u00f3n intracerebroventricular de oligonucle\u00f3tidos antisentido en un modelo de duplicaci\u00f3n g\u00e9nica \u00ab\u00a0MECP2 humanizado\u00a0\u00bb en el que el rat\u00f3n porta dos alelos MECP2 humanos y ning\u00fan alelo end\u00f3geno.<\/strong><\/p>\n\n<p><strong>La administraci\u00f3n redujo eficazmente la expresi\u00f3n del MECP2 en todo el cerebro de estos ratones, alivi\u00f3 varios d\u00e9ficits conductuales y restaur\u00f3 la expresi\u00f3n de algunos genes regulados por el MeCP2 de forma dosis-dependiente y sin toxicidad alguna.<\/strong><\/p>\n\n<p>Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome, Shao et al.<em>Sci. Transl. Med<\/em>. <strong>13<\/strong>, 7785 (2021)<\/p>\n\n<p><\/p>\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n<p><\/p>\n\n<p><strong>RESUMEN DE LA PUBLICACI\u00d3N<\/strong><\/p>\n\n<p>Muchos trastornos de 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 en ratones con MDS y revertir las caracter\u00edsticas de la enfermedad.<\/p>\n\n<p>Este modelo en ratones con MDS, sin embargo, presentaba un alelo humano transg\u00e9nico y un alelo de rat\u00f3n, estando este \u00faltimo protegido de la terapia MECP2-ASO espec\u00edfica para humanos.<\/p>\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 MDS \u00ab\u00a0MECP2 humanizado\u00a0\u00bb 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 la baja de la expresi\u00f3n de MECP2 en 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 el 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 MDS.<\/p>\n\n<div style=\"height:100px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\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\">Ver la publicaci\u00f3n<\/a><\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>Shao et al. del grupo del Prof. Zoghbi (Baylor College of Medicine, Texas, EE.UU.) publicaron un art\u00edculo en Science Translational medicine titulado \u00a0\u00bb Terapia con oligonucle\u00f3tidos antisentido en un modelo [&hellip;]<\/p>","protected":false},"author":1,"featured_media":13608,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[113],"tags":[],"class_list":["post-15078","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-publicacion"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v25.8 - 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