{"id":7365,"date":"2022-06-14T20:42:45","date_gmt":"2022-06-14T20:42:45","guid":{"rendered":"http:\/\/carola.wwwnl1-sr12.supercp.com\/2022\/06\/14\/reversibler-phaenotyp-bei-humanisierten-maeusen\/"},"modified":"2023-01-13T11:46:50","modified_gmt":"2023-01-13T11:46:50","slug":"reversibler-phaenotyp-bei-humanisierten-maeusen","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/es\/reversibler-phaenotyp-bei-humanisierten-maeusen\/","title":{"rendered":"Fenotipo reversible en humanos"},"content":{"rendered":"<p><strong>Shao et al. del grupo del Prof. Zoghbi (Baylor College of Medicine, Texas, EE.UU.) publican en la revista Science Translational medicine un art\u00edculo con el t\u00edtulo \"Antisense-Oligonucleotid-Therapie auf einem humanisierten Mausmodell des MECP2-Duplikation Syndroms\". <\/strong><\/p>\n\n\n\n<p><strong>Cada especie tiene genes que la caracterizan. Gene kodieren f\u00fcr Proteine, die f\u00fcr jede Spezies spezifisch sind. Por lo tanto, es posible distinguir entre prote\u00ednas humanas y animales. Das in der vorliegenden Studie verwendete Modell, das als \" humanisiertes Modell \" bezeichnet wird, erm\u00f6glicht es, die Aktivit\u00e4t von Antisense-Oligonukleotiden (ASOs) spezifisch auf humanes Protein, das von einer Maus produziert wird, besser zu bewerten. Por eso es tan importante el uso futuro de los ASO en humanos.<\/strong><\/p>\n\n\n\n<p><strong>En este art\u00edculo se describen los resultados obtenidos mediante la inyecci\u00f3n intracerebroventricular de oligonucle\u00f3tidos antisentido en este modelo de \"duplicaci\u00f3n gen\u00e9tica humanizada de MECP2\", en el que la Maus tiene dos alelos MECP2 humanos y ning\u00fan alelo Maus end\u00f3geno.<\/strong><\/p>\n\n\n\n<p><strong>La reducci\u00f3n quir\u00fargica de la expresi\u00f3n de MeCP2 en el conjunto de la enfermedad, atenu\u00f3 varios d\u00e9ficits de salud e hizo que la expresi\u00f3n del gen regulador de MeCP2 fuera m\u00e1s baja en dosis y sin ninguna toxicidad a\u00f1adida.<\/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>ZUSAMMENFASSUNG DES ARTIKELS (von Englisch \u00fcbersetzt):<\/strong><\/p>\n\n\n\n<p>Algunos trastornos que conducen a una enfermedad grave se deben a variaciones en la capacidad visual y, por lo general, no se han establecido opciones terap\u00e9uticas para este tipo de enfermedades. El s\u00edndrome de duplicaci\u00f3n MECP2 es uno de los trastornos gen\u00e9ticos m\u00e1s frecuentes en los hombres y resulta de duplicaciones que afectan al genoma de la prote\u00edna 2 de uni\u00f3n a metil-CpG (MECP2). Hasta ahora hemos descubierto que el tratamiento con ASO (Antisense-Oligonukleotiden) puede reducir el tama\u00f1o de las prote\u00ednas MECP2 en un modelo de rat\u00f3n de la duplicaci\u00f3n del gen MECP2 y modificar los s\u00edntomas caracter\u00edsticos del s\u00edndrome.<\/p>\n\n\n\n<p>Este modelo de rat\u00f3n con s\u00edndrome de mutag\u00e9nesis de MECP2 fue, sin embargo, el resultado de la combinaci\u00f3n de dos alelos humanos transg\u00e9nicos y un rat\u00f3n, aunque el \u00faltimo de ellos fue eliminado tras la selecci\u00f3n de la diana humana ASO-MECP2.<\/p>\n\n\n\n<p>Das MeCP2-Protein ist ein Protein mit einer hohen Konzentration. Por lo tanto, ASO deber\u00eda poder afirmar que la cantidad de MeCP2 no est\u00e1 fuertemente reducida, dado que esto causa el s\u00edndrome de Rett. Por lo tanto, hemos desarrollado un \"modelo MECP2 humanizado\" del s\u00edndrome de MECP2enduplikation, en el que el Maus tiene dos alelos MECP2 menschlic y ning\u00fan alelo Maus end\u00f3geno. Mediante la inyecci\u00f3n intracerebroventricular de ASO-MECP2 se pudo reducir la expresi\u00f3n de MeCP2 en el cerebro de esta mujer. Por otra parte, el ASO-MECP2 redujo el deterioro de la salud y aument\u00f3 la expresi\u00f3n de genes reguladores de MeCP2 de forma dosificada y sin ninguna toxicidad. Por lo tanto, la comprobaci\u00f3n de la presencia de ASO-MECP2 en el sistema nervioso central es, en este modelo de rat\u00f3n, muy positiva y veraz, y ofrece una soluci\u00f3n muy interesante para el tratamiento del s\u00edndrome de insuficiencia de MECP2.<\/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\">B\u00fasqueda de publicaciones<\/a><\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>Shao et al. aus der Gruppe von Prof. Zoghbi (Baylor College of Medicine, Texas, USA) publizieren in der Zeitschrift Science Translational medicine einen Artikel mit dem Titel \u00ab\u00a0Antisense-Oligonucleotid-Therapie auf einem [&hellip;]<\/p>","protected":false},"author":1,"featured_media":11724,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[58],"tags":[],"class_list":["post-7365","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-publikationen"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v25.8 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Reversibler Ph\u00e4notyp bei humanisierten M\u00e4usen - DupMECP2<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/dupmecp2.eu\/es\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de\" \/>\n<meta property=\"og:locale\" content=\"es_ES\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Reversibler Ph\u00e4notyp bei humanisierten M\u00e4usen - DupMECP2\" \/>\n<meta property=\"og:description\" content=\"Shao et al. aus der Gruppe von Prof. Zoghbi (Baylor College of Medicine, Texas, USA) publizieren in der Zeitschrift Science Translational medicine einen Artikel mit dem Titel \u00ab\u00a0Antisense-Oligonucleotid-Therapie auf einem [&hellip;]\" \/>\n<meta property=\"og:url\" content=\"https:\/\/dupmecp2.eu\/es\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de\" \/>\n<meta property=\"og:site_name\" content=\"DupMECP2\" \/>\n<meta property=\"article:published_time\" content=\"2022-06-14T20:42:45+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2023-01-13T11:46:50+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2022\/06\/Publication-Zoghbi-Souris-Humanisees.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"1500\" \/>\n\t<meta property=\"og:image:height\" content=\"1050\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"Caroline\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Escrito por\" \/>\n\t<meta name=\"twitter:data1\" content=\"Caroline\" \/>\n\t<meta name=\"twitter:label2\" content=\"Tiempo de lectura\" \/>\n\t<meta name=\"twitter:data2\" content=\"2 minutos\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de#article\",\"isPartOf\":{\"@id\":\"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de\"},\"author\":{\"name\":\"Caroline\",\"@id\":\"https:\/\/dupmecp2.eu\/#\/schema\/person\/b41e8cbba153cd974ee340386af279ee\"},\"headline\":\"Reversibler Ph\u00e4notyp bei humanisierten M\u00e4usen\",\"datePublished\":\"2022-06-14T20:42:45+00:00\",\"dateModified\":\"2023-01-13T11:46:50+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de\"},\"wordCount\":475,\"publisher\":{\"@id\":\"https:\/\/dupmecp2.eu\/#organization\"},\"image\":{\"@id\":\"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de#primaryimage\"},\"thumbnailUrl\":\"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2022\/06\/Publication-Zoghbi-Souris-Humanisees.jpg\",\"articleSection\":[\"Publikationen\"],\"inLanguage\":\"es\"},{\"@type\":\"WebPage\",\"@id\":\"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de\",\"url\":\"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de\",\"name\":\"Reversibler Ph\u00e4notyp bei humanisierten M\u00e4usen - DupMECP2\",\"isPartOf\":{\"@id\":\"https:\/\/dupmecp2.eu\/#website\"},\"primaryImageOfPage\":{\"@id\":\"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de#primaryimage\"},\"image\":{\"@id\":\"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de#primaryimage\"},\"thumbnailUrl\":\"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2022\/06\/Publication-Zoghbi-Souris-Humanisees.jpg\",\"datePublished\":\"2022-06-14T20:42:45+00:00\",\"dateModified\":\"2023-01-13T11:46:50+00:00\",\"breadcrumb\":{\"@id\":\"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de#breadcrumb\"},\"inLanguage\":\"es\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de\"]}]},{\"@type\":\"ImageObject\",\"inLanguage\":\"es\",\"@id\":\"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de#primaryimage\",\"url\":\"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2022\/06\/Publication-Zoghbi-Souris-Humanisees.jpg\",\"contentUrl\":\"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2022\/06\/Publication-Zoghbi-Souris-Humanisees.jpg\",\"width\":1500,\"height\":1050},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Accueil\",\"item\":\"https:\/\/dupmecp2.eu\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"Publikationen\",\"item\":\"https:\/\/dupmecp2.eu\/category\/publikationen\/?lang=de\"},{\"@type\":\"ListItem\",\"position\":3,\"name\":\"Reversibler Ph\u00e4notyp bei humanisierten M\u00e4usen\"}]},{\"@type\":\"WebSite\",\"@id\":\"https:\/\/dupmecp2.eu\/#website\",\"url\":\"https:\/\/dupmecp2.eu\/\",\"name\":\"DupMECP2\",\"description\":\"EU\",\"publisher\":{\"@id\":\"https:\/\/dupmecp2.eu\/#organization\"},\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\/\/dupmecp2.eu\/?s={search_term_string}\"},\"query-input\":{\"@type\":\"PropertyValueSpecification\",\"valueRequired\":true,\"valueName\":\"search_term_string\"}}],\"inLanguage\":\"es\"},{\"@type\":\"Organization\",\"@id\":\"https:\/\/dupmecp2.eu\/#organization\",\"name\":\"DupMECP2\",\"url\":\"https:\/\/dupmecp2.eu\/\",\"logo\":{\"@type\":\"ImageObject\",\"inLanguage\":\"es\",\"@id\":\"https:\/\/dupmecp2.eu\/#\/schema\/logo\/image\/\",\"url\":\"\",\"contentUrl\":\"\",\"caption\":\"DupMECP2\"},\"image\":{\"@id\":\"https:\/\/dupmecp2.eu\/#\/schema\/logo\/image\/\"}},{\"@type\":\"Person\",\"@id\":\"https:\/\/dupmecp2.eu\/#\/schema\/person\/b41e8cbba153cd974ee340386af279ee\",\"name\":\"Caroline\",\"sameAs\":[\"https:\/\/dupmecp2.eu\"],\"url\":\"https:\/\/dupmecp2.eu\/es\/author\/417a2385dc37aa45\/\"}]}<\/script>\n<!-- \/ Yoast SEO plugin. -->","yoast_head_json":{"title":"Ph\u00e4notyp reversibler bei humanisierten M\u00e4usen - DupMECP2","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/dupmecp2.eu\/es\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de","og_locale":"es_ES","og_type":"article","og_title":"Reversibler Ph\u00e4notyp bei humanisierten M\u00e4usen - DupMECP2","og_description":"Shao et al. aus der Gruppe von Prof. Zoghbi (Baylor College of Medicine, Texas, USA) publizieren in der Zeitschrift Science Translational medicine einen Artikel mit dem Titel \u00ab\u00a0Antisense-Oligonucleotid-Therapie auf einem [&hellip;]","og_url":"https:\/\/dupmecp2.eu\/es\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de","og_site_name":"DupMECP2","article_published_time":"2022-06-14T20:42:45+00:00","article_modified_time":"2023-01-13T11:46:50+00:00","og_image":[{"width":1500,"height":1050,"url":"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2022\/06\/Publication-Zoghbi-Souris-Humanisees.jpg","type":"image\/jpeg"}],"author":"Caroline","twitter_card":"summary_large_image","twitter_misc":{"Escrito por":"Caroline","Tiempo de lectura":"2 minutos"},"schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"Article","@id":"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de#article","isPartOf":{"@id":"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de"},"author":{"name":"Caroline","@id":"https:\/\/dupmecp2.eu\/#\/schema\/person\/b41e8cbba153cd974ee340386af279ee"},"headline":"Reversibler Ph\u00e4notyp bei humanisierten M\u00e4usen","datePublished":"2022-06-14T20:42:45+00:00","dateModified":"2023-01-13T11:46:50+00:00","mainEntityOfPage":{"@id":"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de"},"wordCount":475,"publisher":{"@id":"https:\/\/dupmecp2.eu\/#organization"},"image":{"@id":"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de#primaryimage"},"thumbnailUrl":"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2022\/06\/Publication-Zoghbi-Souris-Humanisees.jpg","articleSection":["Publikationen"],"inLanguage":"es"},{"@type":"WebPage","@id":"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de","url":"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de","name":"Ph\u00e4notyp reversibler bei humanisierten M\u00e4usen - DupMECP2","isPartOf":{"@id":"https:\/\/dupmecp2.eu\/#website"},"primaryImageOfPage":{"@id":"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de#primaryimage"},"image":{"@id":"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de#primaryimage"},"thumbnailUrl":"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2022\/06\/Publication-Zoghbi-Souris-Humanisees.jpg","datePublished":"2022-06-14T20:42:45+00:00","dateModified":"2023-01-13T11:46:50+00:00","breadcrumb":{"@id":"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de#breadcrumb"},"inLanguage":"es","potentialAction":[{"@type":"ReadAction","target":["https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de"]}]},{"@type":"ImageObject","inLanguage":"es","@id":"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de#primaryimage","url":"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2022\/06\/Publication-Zoghbi-Souris-Humanisees.jpg","contentUrl":"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2022\/06\/Publication-Zoghbi-Souris-Humanisees.jpg","width":1500,"height":1050},{"@type":"BreadcrumbList","@id":"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Accueil","item":"https:\/\/dupmecp2.eu\/"},{"@type":"ListItem","position":2,"name":"Publikationen","item":"https:\/\/dupmecp2.eu\/category\/publikationen\/?lang=de"},{"@type":"ListItem","position":3,"name":"Reversibler Ph\u00e4notyp bei humanisierten M\u00e4usen"}]},{"@type":"WebSite","@id":"https:\/\/dupmecp2.eu\/#website","url":"https:\/\/dupmecp2.eu\/","name":"DupMECP2","description":"UE","publisher":{"@id":"https:\/\/dupmecp2.eu\/#organization"},"potentialAction":[{"@type":"SearchAction","target":{"@type":"EntryPoint","urlTemplate":"https:\/\/dupmecp2.eu\/?s={search_term_string}"},"query-input":{"@type":"PropertyValueSpecification","valueRequired":true,"valueName":"search_term_string"}}],"inLanguage":"es"},{"@type":"Organization","@id":"https:\/\/dupmecp2.eu\/#organization","name":"DupMECP2","url":"https:\/\/dupmecp2.eu\/","logo":{"@type":"ImageObject","inLanguage":"es","@id":"https:\/\/dupmecp2.eu\/#\/schema\/logo\/image\/","url":"","contentUrl":"","caption":"DupMECP2"},"image":{"@id":"https:\/\/dupmecp2.eu\/#\/schema\/logo\/image\/"}},{"@type":"Person","@id":"https:\/\/dupmecp2.eu\/#\/schema\/person\/b41e8cbba153cd974ee340386af279ee","name":"Caroline","sameAs":["https:\/\/dupmecp2.eu"],"url":"https:\/\/dupmecp2.eu\/es\/author\/417a2385dc37aa45\/"}]}},"_links":{"self":[{"href":"https:\/\/dupmecp2.eu\/es\/wp-json\/wp\/v2\/posts\/7365","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dupmecp2.eu\/es\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dupmecp2.eu\/es\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dupmecp2.eu\/es\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dupmecp2.eu\/es\/wp-json\/wp\/v2\/comments?post=7365"}],"version-history":[{"count":0,"href":"https:\/\/dupmecp2.eu\/es\/wp-json\/wp\/v2\/posts\/7365\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dupmecp2.eu\/es\/wp-json\/wp\/v2\/media\/11724"}],"wp:attachment":[{"href":"https:\/\/dupmecp2.eu\/es\/wp-json\/wp\/v2\/media?parent=7365"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dupmecp2.eu\/es\/wp-json\/wp\/v2\/categories?post=7365"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dupmecp2.eu\/es\/wp-json\/wp\/v2\/tags?post=7365"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}