{"id":20795,"date":"2024-06-25T08:57:32","date_gmt":"2024-06-25T06:57:32","guid":{"rendered":"https:\/\/ela-international.preprod.melisagency.fr\/?post_type=news_research&#038;p=20795"},"modified":"2024-07-11T14:18:36","modified_gmt":"2024-07-11T12:18:36","slug":"characterization-of-lysosomal-dysfunction-and-targeted-intervention-in-x-ald","status":"publish","type":"news_research","link":"https:\/\/elainternational.eu\/en\/actualites_recherche\/characterization-of-lysosomal-dysfunction-and-targeted-intervention-in-x-ald\/","title":{"rendered":"Characterization of lysosomal dysfunction and targeted intervention in X-ALD"},"content":{"rendered":"\n\n<section class=\"contenus-titre-texte\">\n    <div class=\"contenus-titre-texte-container container-content\">\n        <div class=\"contenus-titre-texte-header mobile-flex-display\">\n                        <h3><strong>Characterization of lysosomal dysfunction and targeted intervention in X-ALD<\/strong><\/h3>\n                                <\/div>\n            <\/div>\n<\/section>\n\n\n<section class=\"infos-ribbon\">\n    <div class=\"infos-ribbon-container container-content mobile-flex-display\">\n        <p><em>Josh Bonkowsky  &#8211; University of Utah School of Medicine, Salt Lake City, USA &#8211; ELA2020-004I3<\/em><\/p>\n            <\/div>\n<\/section>\n\n\n\n\n\n<section class=\"contenus-titre-texte\">\n    <div class=\"contenus-titre-texte-container container-content\">\n        <div class=\"contenus-titre-texte-header mobile-flex-display\">\n                        <h5><strong>Description of the project<\/strong><\/h5>\n                                <\/div>\n                    <div class=\"contenu-texte\">\n                <p>X-linked adrenoleukodystrophy (ALD) is a severe disease, ranging from the fatal cerebral inflammatory demyelinating form affecting boys (cALD) to the peripheral dying back axonopathy that affects adults (AMN). There are no effective therapies for AMN, and the cause of cALD is poorly understood.<\/p>\n<p>The goal is to develop, validate, and use for treatment discovery, a new model of ALD. To accomplish this, the group will use the small genetic vertebrate model zebrafish, which has low costs and the ability to perform high-throughput screens that are unfeasible in other vertebrate systems. Zebrafish have the same genes as humans, including <em>ABCD1<\/em>, the gene responsible for ALD. The data shows that mutations in zebrafish <em>ABCD1<\/em> cause ALD in zebrafish. The team is using the ALD zebrafish model to validate a new potential treatment for ALD, to understand this drug\u2019s mechanism, and to understand the cause of cALD.<\/p>\n<p style=\"font-weight: 400;\">Project financed by ELA up to: 98 841 \u20ac<\/p>\n            <\/div>\n            <\/div>\n<\/section>","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":0,"parent":0,"template":"","news_research_categories":[127],"news_research_tags":[110],"class_list":["post-20795","news_research","type-news_research","status-publish","hentry","news_research_categories-project","news_research_tags-cerebral-adrenoleukodystrophy-cald"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Characterization of lysosomal dysfunction and targeted intervention in X-ALD - ELA International<\/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:\/\/elainternational.eu\/en\/actualites_recherche\/characterization-of-lysosomal-dysfunction-and-targeted-intervention-in-x-ald\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Characterization of lysosomal dysfunction and targeted intervention in X-ALD - 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