{"id":20783,"date":"2024-06-24T16:29:24","date_gmt":"2024-06-24T14:29:24","guid":{"rendered":"https:\/\/ela-international.preprod.melisagency.fr\/?post_type=news_research&#038;p=20783"},"modified":"2024-07-11T10:49:30","modified_gmt":"2024-07-11T08:49:30","slug":"gene-therapy-to-treat-megalencephalic-leukodystrophy-with-subcortical-cysts-mlc","status":"publish","type":"news_research","link":"https:\/\/elainternational.eu\/en\/actualites_recherche\/gene-therapy-to-treat-megalencephalic-leukodystrophy-with-subcortical-cysts-mlc\/","title":{"rendered":"Gene therapy to treat Megalencephalic Leukodystrophy with subcortical cysts (MLC)"},"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>Gene therapy to treat Megalencephalic Leukodystrophy with subcortical cysts (MLC)<\/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>Assumpci\u00f3 Bosch &#8211; Universitat Aut\u00f2noma de Barcelona, Barcelona, Spain &#8211; ELA2022-004I2<\/em><br \/>\r\n<\/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>Megalencephalic leukoencephalopathy with subcortical cysts (MLC) is a rare genetic disorder characterized by an abnormal head, loss of motor functions, epilepsy and mild mental decline. The disease is caused by mutations in two genes named MLC1 and GLIALCAM. There are no therapies for MLC patients, only palliative treatment. We recently demonstrated the efficiency of gene therapy in correcting MLC-typical myelin vacuolation. Now we aim to demonstrate that gene therapy is also able to correct the motor impairment present in the MLC knock-out mouse models. Thus, we propose to administer the therapeutic AAV vectors to the affected cells in the brain of these mice, and to look for longitudinal, non-invasive tests to assess for <em>in vivo<\/em> indicators of disease correction in the animal models that could finally be translated to clinical trials for MLC patients.<\/p>\n<p style=\"font-weight: 400;\">Project financed by ELA up to: 44 414 \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":[114,118],"class_list":["post-20783","news_research","type-news_research","status-publish","hentry","news_research_categories-project","news_research_tags-leukodystrophy","news_research_tags-megalencephalic-leukoencephalopathy-with-subcortical-cysts-mlc"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Gene therapy to treat Megalencephalic Leukodystrophy with subcortical cysts (MLC) - 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\/gene-therapy-to-treat-megalencephalic-leukodystrophy-with-subcortical-cysts-mlc\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Gene therapy to treat Megalencephalic Leukodystrophy with subcortical cysts (MLC) - ELA International\" \/>\n<meta property=\"og:url\" content=\"https:\/\/elainternational.eu\/en\/actualites_recherche\/gene-therapy-to-treat-megalencephalic-leukodystrophy-with-subcortical-cysts-mlc\/\" \/>\n<meta property=\"og:site_name\" content=\"ELA International\" \/>\n<meta property=\"article:modified_time\" content=\"2024-07-11T08:49:30+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\\\/gene-therapy-to-treat-megalencephalic-leukodystrophy-with-subcortical-cysts-mlc\\\/\",\"url\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/actualites_recherche\\\/gene-therapy-to-treat-megalencephalic-leukodystrophy-with-subcortical-cysts-mlc\\\/\",\"name\":\"Gene therapy to treat Megalencephalic Leukodystrophy with subcortical cysts (MLC) - 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