{"id":5594,"date":"2025-02-03T10:04:17","date_gmt":"2025-02-03T09:04:17","guid":{"rendered":"https:\/\/cnatm-1.betapage.de\/?p=5594"},"modified":"2025-02-03T10:04:59","modified_gmt":"2025-02-03T09:04:59","slug":"gen-editierung-bei-duchenne-muskeldystrophie","status":"publish","type":"post","link":"https:\/\/cnatm.de\/en\/gen-editierung-bei-duchenne-muskeldystrophie\/","title":{"rendered":"Gene editing in Duchenne muscular dystrophy"},"content":{"rendered":"<div data-elementor-type=\"wp-post\" data-elementor-id=\"5594\" class=\"elementor elementor-5594\" data-elementor-settings=\"{&quot;ha_cmc_init_switcher&quot;:&quot;no&quot;}\" data-elementor-post-type=\"post\">\n\t\t\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-6363f8b elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"6363f8b\" data-element_type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-807a88d\" data-id=\"807a88d\" data-element_type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-0b60e27 elementor-widget elementor-widget-heading\" data-id=\"0b60e27\" data-element_type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<h2 class=\"elementor-heading-title elementor-size-default\">Gene editing in Duchenne muscular dystrophy<\/h2>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-39021d3 elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"39021d3\" data-element_type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-3c861fc\" data-id=\"3c861fc\" data-element_type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-553a889 elementor-widget elementor-widget-spacer\" data-id=\"553a889\" data-element_type=\"widget\" data-widget_type=\"spacer.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-spacer\">\n\t\t\t<div class=\"elementor-spacer-inner\"><\/div>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-39a4d74 elementor-widget elementor-widget-text-editor\" data-id=\"39a4d74\" data-element_type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>WP9 is dedicated to cardiovascular diseases, in this case in the context of skeletal muscle disease. Specific guide RNAs for CRISPR\/Cas-based gene therapy of DMD will be developed and formulated into nanoparticles. Initially, the therapy will be studied experimentally in organoid models before final validation in large animal models. Since the dystrophin gene responsible for DMD is located on the X chromosome, WP9 also aims to activate the intact dystrophin allele on the second (inactive) X chromosome by manipulating epigenetic factors.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-72d739a elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"72d739a\" data-element_type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-top-column elementor-element elementor-element-89f255c\" data-id=\"89f255c\" data-element_type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-89a1439 elementor-widget elementor-widget-text-editor\" data-id=\"89a1439\" data-element_type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p><strong>Head: Alessandra Moretti<br \/><\/strong><\/p><p><strong>PIs: <\/strong>Alessandra Moretti, Christian Kupatt, Daniel Andergassen<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-top-column elementor-element elementor-element-4e4729f\" data-id=\"4e4729f\" data-element_type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap\">\n\t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<\/div>","protected":false},"excerpt":{"rendered":"<p>Gen-Editierung bei Duchenne-Muskeldystrophie WP9 widmet sich Herz-Kreislauferkrankungen, in diesem Fall im Rahmen einer Skelettmuskelerkrankung. Es werden spezifische guide-RNAs f\u00fcr eine CRISPR\/Cas-basierte Gentherapie der DMD entwickelt und in Nanopartikel formuliert. Zun\u00e4chst soll die Therapie in Organoidmodellen experimentell untersucht werden, ehe abschlie\u00dfend die Validierung im Gro\u00dftiermodell erfolgt. Da das f\u00fcr DMD verantwortliche Dystrophin-Gen auf dem X-Chromosom liegt, [&hellip;]<\/p>","protected":false},"author":11,"featured_media":5595,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[75],"tags":[],"class_list":["post-5594","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-mrna-wirkstoffe-und-andere-prinzipien"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v26.5 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Gen-Editierung bei Duchenne-Muskeldystrophie - CNATM<\/title>\n<meta name=\"description\" content=\"WP9 widmet sich Herz-Kreislauferkrankungen, in diesem Fall im Rahmen einer Skelettmuskelerkrankung.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/cnatm.de\/en\/gen-editierung-bei-duchenne-muskeldystrophie\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Gen-Editierung bei Duchenne-Muskeldystrophie - 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