{"id":23606,"date":"2025-11-21T09:49:05","date_gmt":"2025-11-21T08:49:05","guid":{"rendered":"https:\/\/elainternational.eu\/?post_type=news_research&p=23606"},"modified":"2025-11-21T10:17:45","modified_gmt":"2025-11-21T09:17:45","slug":"mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet","status":"publish","type":"news_research","link":"https:\/\/elainternational.eu\/en\/actualites_recherche\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\/","title":{"rendered":"MLC Advanced Gene therapy: Next-step Evaluation and Translation (MAGNET)"},"content":{"rendered":"\n\n
\n
\n
\n

MLC Advanced Gene therapy: Next-step Evaluation and Translation (MAGNET)<\/h1>\n <\/div>\n <\/div>\n<\/section>\n\n\n
\n
\n

Assumpci\u00f3 Bosch, Universitat Aut\u00f2noma de Barcelona \/ ELA 2025-004C2<\/em><\/p>\n <\/div>\n<\/section>\n\n\n\n\n\n

\n
\n
\n
Project abstract<\/strong><\/h5>\n <\/div>\n
\n

Megalencephalic Leukoencephalopathy with Subcortical Cysts (MLC) is a rare brain disease that mainly affects children. It is caused by changes in a gene called MLC1<\/em>, which is active in a type of brain cell known as an astrocyte. When this gene doesn’t work properly, water builds up in the brain, leading to swelling, cysts, and damage to the brain\u2019s white matter. This causes symptoms like an enlarged head, balance problems, muscle stiffness, seizures, and mild learning difficulties.<\/p>\n

There is currently no cure for MLC. Our team is working on a gene therapy approach that uses a harmless virus (called AAV) to deliver a healthy copy of the MLC1 <\/em>gene directly to the brain. In mouse models of MLC, we have already shown that this treatment restores normal brain function\u2014even when given months after the disease has started. The treated mice showed long-lasting correction of brain abnormalities and improvement in movement.<\/p>\n

To move closer to treating patients, we are now optimizing how the therapy is delivered, which viral vector to use, and how to ensure safety. Importantly, we have created a new regulatory sequence that helps the therapy target only the right brain cells. We will test this new approach in non-human primates to confirm that the treatment works across species and reaches all necessary brain regions. These studies are a key step toward launching clinical trials and bringing the first effective therapy for MLC to patients.<\/p>\n <\/div>\n <\/div>\n<\/section>","protected":false},"excerpt":{"rendered":"","protected":false},"author":6,"featured_media":0,"parent":0,"template":"","news_research_categories":[127],"news_research_tags":[118],"class_list":["post-23606","news_research","type-news_research","status-publish","hentry","news_research_categories-project","news_research_tags-megalencephalic-leukoencephalopathy-with-subcortical-cysts-mlc"],"acf":[],"yoast_head":"\nMLC Advanced Gene therapy: Next-step Evaluation and Translation (MAGNET) - 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\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"MLC Advanced Gene therapy: Next-step Evaluation and Translation (MAGNET) - ELA International\" \/>\n<meta property=\"og:url\" content=\"https:\/\/elainternational.eu\/en\/actualites_recherche\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\/\" \/>\n<meta property=\"og:site_name\" content=\"ELA International\" \/>\n<meta property=\"article:modified_time\" content=\"2025-11-21T09:17:45+00:00\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/actualites_recherche\\\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\\\/\",\"url\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/actualites_recherche\\\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\\\/\",\"name\":\"MLC Advanced Gene therapy: Next-step Evaluation and Translation (MAGNET) - ELA International\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/#website\"},\"datePublished\":\"2025-11-21T08:49:05+00:00\",\"dateModified\":\"2025-11-21T09:17:45+00:00\",\"breadcrumb\":{\"@id\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/actualites_recherche\\\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\\\/#breadcrumb\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\\\/\\\/elainternational.eu\\\/en\\\/actualites_recherche\\\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\\\/\"]}]},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/actualites_recherche\\\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\\\/#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"MLC Advanced Gene therapy: Next-step Evaluation and Translation (MAGNET)\"}]},{\"@type\":\"WebSite\",\"@id\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/#website\",\"url\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/\",\"name\":\"ELA International\",\"description\":\"\",\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/?s={search_term_string}\"},\"query-input\":{\"@type\":\"PropertyValueSpecification\",\"valueRequired\":true,\"valueName\":\"search_term_string\"}}],\"inLanguage\":\"en-US\"}]}<\/script>\n<!-- \/ Yoast SEO plugin. -->","yoast_head_json":{"title":"MLC Advanced Gene therapy: Next-step Evaluation and Translation (MAGNET) - ELA International","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:\/\/elainternational.eu\/en\/actualites_recherche\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\/","og_locale":"en_US","og_type":"article","og_title":"MLC Advanced Gene therapy: Next-step Evaluation and Translation (MAGNET) - ELA International","og_url":"https:\/\/elainternational.eu\/en\/actualites_recherche\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\/","og_site_name":"ELA International","article_modified_time":"2025-11-21T09:17:45+00:00","twitter_card":"summary_large_image","schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"WebPage","@id":"https:\/\/elainternational.eu\/en\/actualites_recherche\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\/","url":"https:\/\/elainternational.eu\/en\/actualites_recherche\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\/","name":"MLC Advanced Gene therapy: Next-step Evaluation and Translation (MAGNET) - ELA International","isPartOf":{"@id":"https:\/\/elainternational.eu\/en\/#website"},"datePublished":"2025-11-21T08:49:05+00:00","dateModified":"2025-11-21T09:17:45+00:00","breadcrumb":{"@id":"https:\/\/elainternational.eu\/en\/actualites_recherche\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\/#breadcrumb"},"inLanguage":"en-US","potentialAction":[{"@type":"ReadAction","target":["https:\/\/elainternational.eu\/en\/actualites_recherche\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\/"]}]},{"@type":"BreadcrumbList","@id":"https:\/\/elainternational.eu\/en\/actualites_recherche\/mlc-advanced-gene-therapy-next-step-evaluation-and-translation-magnet\/#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https:\/\/elainternational.eu\/en\/"},{"@type":"ListItem","position":2,"name":"MLC Advanced Gene therapy: Next-step Evaluation and Translation (MAGNET)"}]},{"@type":"WebSite","@id":"https:\/\/elainternational.eu\/en\/#website","url":"https:\/\/elainternational.eu\/en\/","name":"ELA International","description":"","potentialAction":[{"@type":"SearchAction","target":{"@type":"EntryPoint","urlTemplate":"https:\/\/elainternational.eu\/en\/?s={search_term_string}"},"query-input":{"@type":"PropertyValueSpecification","valueRequired":true,"valueName":"search_term_string"}}],"inLanguage":"en-US"}]}},"_links":{"self":[{"href":"https:\/\/elainternational.eu\/en\/wp-json\/wp\/v2\/news_research\/23606","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/elainternational.eu\/en\/wp-json\/wp\/v2\/news_research"}],"about":[{"href":"https:\/\/elainternational.eu\/en\/wp-json\/wp\/v2\/types\/news_research"}],"author":[{"embeddable":true,"href":"https:\/\/elainternational.eu\/en\/wp-json\/wp\/v2\/users\/6"}],"wp:attachment":[{"href":"https:\/\/elainternational.eu\/en\/wp-json\/wp\/v2\/media?parent=23606"}],"wp:term":[{"taxonomy":"news_research_categories","embeddable":true,"href":"https:\/\/elainternational.eu\/en\/wp-json\/wp\/v2\/news_research_categories?post=23606"},{"taxonomy":"news_research_tags","embeddable":true,"href":"https:\/\/elainternational.eu\/en\/wp-json\/wp\/v2\/news_research_tags?post=23606"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}