{"id":23095,"date":"2025-04-22T08:38:39","date_gmt":"2025-04-22T06:38:39","guid":{"rendered":"https:\/\/elainternational.eu\/?post_type=news_research&#038;p=23095"},"modified":"2025-04-22T09:48:50","modified_gmt":"2025-04-22T07:48:50","slug":"exploring-new-molecular-mechanisms-and-potential-therapeutic-strategies-for-mlc-disease-using-patientderived-cells","status":"publish","type":"news_research","link":"https:\/\/elainternational.eu\/en\/actualites_recherche\/exploring-new-molecular-mechanisms-and-potential-therapeutic-strategies-for-mlc-disease-using-patientderived-cells\/","title":{"rendered":"Exploring new molecular mechanisms and potential therapeutic strategies for MLC disease using patientderived cells"},"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                        <h2><strong>Exploring new molecular mechanisms and potential therapeutic strategies for MLC disease using patientderived cells<\/strong><\/h2>\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>Elena Ambrosini, Istituto Superiore di Sanit\u00e0 &#8211; Fiorella Piemonte, Ospedale Pediatrico Bambino Ges\u00f9, OPBG, IRCCS &#8211; Italy<br>ELA 2024-002C4<\/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>Project abstract<\/strong><\/h5>\n                                <\/div>\n                    <div class=\"contenu-texte\">\n                <p>MLC is a very rare, incurable leukodystrophy. Patients develop progressive deterioration of motor functions leading to wheel-chair dependence, cognitive decline and seizures, and their management requires intensive parental, scholastic and social support. Since the pathological process is partially reversible, the comprehension of the molecular defects causing the disease may allow the identification of specific molecules to be targeted to correct the dysregulated processes and slow down MLC progression, thus improving disease symptoms. We here proposed to study MLC dysfunctional mechanisms and test therapeutic compounds in astrocytes differentiated from patient skin cells and in blood samples. Results obtained from these studies will allow the identification of pharmacological compounds able to correct MLC defects in patient-derived cells, providing the foundation for therapeutic strategies potentially translatable 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-23095","news_research","type-news_research","status-publish","hentry","news_research_categories-project","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>Exploring new molecular mechanisms and potential therapeutic strategies for MLC disease using patientderived cells - 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\/exploring-new-molecular-mechanisms-and-potential-therapeutic-strategies-for-mlc-disease-using-patientderived-cells\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Exploring new molecular mechanisms and potential therapeutic strategies for MLC disease using patientderived cells - 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