{"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\/en\/fenotipo-reversible-en-un-modelo-humanizado-en-ratones\/","title":{"rendered":"Fenotipo reversible 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 \" Terapia con oligonucle\u00f3tidos antisentido en un modelo humanizado del s\u00edndrome de duplicaci\u00f3n MECP2 en ratones\". <\/strong><\/p>\n\n<p><strong>Each species has its own genes, which code for proteins that are exclusive to each species. Es posible que las prote\u00ednas humanas y de rat\u00f3n sean ligeramente diferentes. The model used in the work presented, known as the \"humanised model\", allows better evaluation of the activity of antisense oligonucleotides (ASOs) specifically on the human protein produced by a rat. 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 \" MECP2 humanizado \" 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>SUMMARY OF THE PUBLICATION<\/strong><\/p>\n\n<p>Many forms of intellectual disability are due to variations in the number of copies, and to date there have been no proven treatment options for this class of disorders. 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>Because MeCP2 is a dose-sensitive protein, the ASO must be titrated in such a way that the MeCP2 cantidad is not reduced too much, which would cause Rett syndrome. Por lo tanto, generamos un modelo de MDS \" MECP2 humanizado \" que porta dos alelos MECP2 humanos y ning\u00fan alelo end\u00f3geno de rat\u00f3n. Intracerebroventricular injection of MECP2-ASO effectively regulated the low level of MECP2 expression in the cerebrum of these rats. Furthermore, MECP2-ASO mitigated several behavioural deficits and restored the expression of selected genes regulated by MECP2 in a dose-dependent manner without any toxicity. 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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