{"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\/en\/reversibler-phaenotyp-bei-humanisierten-maeusen\/","title":{"rendered":"Reversibler Ph\u00e4notyp bei humanisierten M\u00e4usen"},"content":{"rendered":"<p><strong>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 \"Antisense-Oligonucleotid-Therapie auf einem humanisierten Mausmodell des MECP2-Duplikation Syndroms\". <\/strong><\/p>\n\n\n\n<p><strong>Jede Species hat Gene, die ihr charakteristisch sind. Gene kodieren f\u00fcr Proteine, die f\u00fcr jede Spezies spezifisch sind. It is therefore possible to distinguish between human and animal proteins. 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. This is why the future use of ASOs in humans is so important.<\/strong><\/p>\n\n\n\n<p><strong>In diesem Artikel werden die Ergebnisse vorgestellt, die nach intrazerebroventrikul\u00e4rer Injektion von Antisense-Oligonukleotiden in diesem \"humanisierten MECP2 gen-duplikation\"-Mausmodell beobachtet wurden, bei dem die Maus zwei menschliche MECP2-Allele und kein endogenes Maus-Allel tr\u00e4gt.<\/strong><\/p>\n\n\n\n<p><strong>Die Verabreichung wirksam reduzierte die MeCP2-Expression im gesamten Gehirn dieser M\u00e4use, milderte mehrere Verhaltensdefizite und stellte die Expression bestimmter durch MeCP2 regulierter Gene dosisabh\u00e4ngig und ohne jegliche Toxizit\u00e4t wieder her.<\/strong><\/p>\n\n\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\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>Viele St\u00f6rungen, die zu geistiger Behinderung f\u00fchren, sind auf Kopienzahlvariationen zur\u00fcckzuf\u00fchren, und bislang wurden f\u00fcr diese Art von Krankheiten noch keine Therapieoptionen getestet. Das MECP2-Duplikationssyndrom ist eine der h\u00e4ufigsten genomischen Neuordnungen bei M\u00e4nnern und resultiert aus Duplikationen, die den Genlocus des Methyl-CpG-Bindungsproteins 2 (MECP2) abdecken. Wir haben zuvor gezeigt, dass die Therapie mit Antisense-Oligonukleotiden (ASO) die Menge des MECP2-Proteins in einem Mausmodell it MECP2 gen-duplikation reduzieren und die charakteristischen Symptome des Syndroms umkehren kann.<\/p>\n\n\n\n<p>Dieses Mausmodell mit MECP2-Genduplikation Syndrome war jedoch Tr\u00e4ger eines transgenen menschlichen Allels und eines Mausallels, wobei letzteres vor dem Targeting durch das humanspezifische ASO-MECP2 gesch\u00fctzt war.<\/p>\n\n\n\n<p>Das MeCP2-Protein ist ein Protein mit einer hohen Konzentration. ASO should therefore be able to claim that the amount of MeCP2 is not starkly reduced, given that this causes the Rett syndrome. We therefore developed a \"humanised MECP2 model\" of MECP2enduplikation syndrome, in which the Maus has two menschlic MECP2 alleles and no endogenous Maus alleles. Durch die intrazerebroventrikul\u00e4re Injektion von ASO-MECP2 konnte die MeCP2-Expression im gesamten Gehirn dieser M\u00e4use wirksam reduziert werden. Dar\u00fcber hinaus milderte ASO-MECP2 mehrere Verhaltensdefizite und stellte die Expression bestimmter durch MeCP2 regulierter Gene dosisabh\u00e4ngig und ohne jegliche Toxizit\u00e4t wieder her. Die Verabreichung von ASO-MECP2 in das zentrale Nervensystem ist daher in diesem Mausmodell gut vertr\u00e4glich und vorteilhaft und stellt einen \u00fcbertragbaren Ansatz dar, der f\u00fcr die Behandlung von MECP2-Genduplikation Syndrome in Frage kommen k\u00f6nnte.<\/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\">Publikation ansehen<\/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\/en\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de\" \/>\n<meta property=\"og:locale\" content=\"en_GB\" \/>\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\/en\/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=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"Caroline\" \/>\n\t<meta name=\"twitter:label2\" content=\"Estimated reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"2 minutes\" \/>\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\":\"en-GB\"},{\"@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\":\"en-GB\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de\"]}]},{\"@type\":\"ImageObject\",\"inLanguage\":\"en-GB\",\"@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\":\"en-GB\"},{\"@type\":\"Organization\",\"@id\":\"https:\/\/dupmecp2.eu\/#organization\",\"name\":\"DupMECP2\",\"url\":\"https:\/\/dupmecp2.eu\/\",\"logo\":{\"@type\":\"ImageObject\",\"inLanguage\":\"en-GB\",\"@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\/en\/author\/417a2385dc37aa45\/\"}]}<\/script>\n<!-- \/ Yoast SEO plugin. -->","yoast_head_json":{"title":"Reversibler Ph\u00e4notyp 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\/en\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de","og_locale":"en_GB","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\/en\/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":{"Written by":"Caroline","Estimated reading time":"2 minutes"},"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":"en-GB"},{"@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":"en-GB","potentialAction":[{"@type":"ReadAction","target":["https:\/\/dupmecp2.eu\/reversibler-phaenotyp-bei-humanisierten-maeusen\/?lang=de"]}]},{"@type":"ImageObject","inLanguage":"en-GB","@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":"en-GB"},{"@type":"Organization","@id":"https:\/\/dupmecp2.eu\/#organization","name":"DupMECP2","url":"https:\/\/dupmecp2.eu\/","logo":{"@type":"ImageObject","inLanguage":"en-GB","@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\/en\/author\/417a2385dc37aa45\/"}]}},"_links":{"self":[{"href":"https:\/\/dupmecp2.eu\/en\/wp-json\/wp\/v2\/posts\/7365","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dupmecp2.eu\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dupmecp2.eu\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dupmecp2.eu\/en\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dupmecp2.eu\/en\/wp-json\/wp\/v2\/comments?post=7365"}],"version-history":[{"count":0,"href":"https:\/\/dupmecp2.eu\/en\/wp-json\/wp\/v2\/posts\/7365\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dupmecp2.eu\/en\/wp-json\/wp\/v2\/media\/11724"}],"wp:attachment":[{"href":"https:\/\/dupmecp2.eu\/en\/wp-json\/wp\/v2\/media?parent=7365"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dupmecp2.eu\/en\/wp-json\/wp\/v2\/categories?post=7365"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dupmecp2.eu\/en\/wp-json\/wp\/v2\/tags?post=7365"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}