{"id":23610,"date":"2025-11-21T10:01:46","date_gmt":"2025-11-21T09:01:46","guid":{"rendered":"https:\/\/elainternational.eu\/?post_type=news_research&#038;p=23610"},"modified":"2025-11-21T10:25:53","modified_gmt":"2025-11-21T09:25:53","slug":"lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease","status":"publish","type":"news_research","link":"https:\/\/elainternational.eu\/en\/actualites_recherche\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\/","title":{"rendered":"Lactosylceramide as a novel target for Substrate Reduction Therapy in Krabbe disease"},"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\">\n            <h1 class=\"heading1\">Lactosylceramide as a novel target for Substrate Reduction Therapy in Krabbe disease<\/h1>\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>Luca Mignani, University of Brescia \/ ELA 2025-021F4<\/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>Abstract project<\/strong><\/h5>\n                                <\/div>\n                    <div class=\"contenu-texte\">\n                <p>Krabbe Disease (KD), also known as Globoid Cell Leukodystrophy, is a rare genetic disorder that affects the brain and nervous system. It is caused by mutations in a gene called <em>GALC<\/em>, which leads to the loss of an important lysosomal enzyme called \u03b2-galactosylceramidase. Without this enzyme, different sphingolipids accumulate in the brain, damaging the cells that produce myelin\u2014the protective layer around nerves\u2014and causing severe neurodegeneration, especially in infants.<\/p>\n<p>Until now, most research has focused on a toxic molecule called psychosine, believed to be the main cause of this damage. However, new findings suggest that another substance called lactosylceramide (LacCer), which also accumulates when the GALC enzyme is missing, might play a significant role in the disease. LacCer is known to trigger inflammation in the brain and is found in high amounts in the brains of patients and animal models of KD\u2014even in cases where psychosine is not elevated. This project will explore whether LacCer contributes to the damage seen in KD, especially how it affects the cells responsible for forming myelin (called oligodendrocytes).<\/p>\n<p>The ultimate goal is to determine if LacCer can be targeted by new treatments to slow or stop the disease. Because current treatments for Krabbe Disease\u2014like stem cell transplants\u2014are only effective before symptoms appear, this research could lead to new therapies that work even after the disease has begun. By uncovering a previously overlooked disease mechanism, this project offers a new direction for treating KD and gives hope to patients and families affected by this devastating disorder.<\/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":[129],"class_list":["post-23610","news_research","type-news_research","status-publish","hentry","news_research_categories-project","news_research_tags-krabbe-disease"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Lactosylceramide as a novel target for Substrate Reduction Therapy in Krabbe disease - 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\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Lactosylceramide as a novel target for Substrate Reduction Therapy in Krabbe disease - ELA International\" \/>\n<meta property=\"og:url\" content=\"https:\/\/elainternational.eu\/en\/actualites_recherche\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\/\" \/>\n<meta property=\"og:site_name\" content=\"ELA International\" \/>\n<meta property=\"article:modified_time\" content=\"2025-11-21T09:25:53+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\\\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\\\/\",\"url\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/actualites_recherche\\\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\\\/\",\"name\":\"Lactosylceramide as a novel target for Substrate Reduction Therapy in Krabbe disease - ELA International\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/#website\"},\"datePublished\":\"2025-11-21T09:01:46+00:00\",\"dateModified\":\"2025-11-21T09:25:53+00:00\",\"breadcrumb\":{\"@id\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/actualites_recherche\\\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\\\/#breadcrumb\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\\\/\\\/elainternational.eu\\\/en\\\/actualites_recherche\\\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\\\/\"]}]},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/actualites_recherche\\\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\\\/#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"Lactosylceramide as a novel target for Substrate Reduction Therapy in Krabbe disease\"}]},{\"@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":"Lactosylceramide as a novel target for Substrate Reduction Therapy in Krabbe disease - 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\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\/","og_locale":"en_US","og_type":"article","og_title":"Lactosylceramide as a novel target for Substrate Reduction Therapy in Krabbe disease - ELA International","og_url":"https:\/\/elainternational.eu\/en\/actualites_recherche\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\/","og_site_name":"ELA International","article_modified_time":"2025-11-21T09:25:53+00:00","twitter_card":"summary_large_image","schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"WebPage","@id":"https:\/\/elainternational.eu\/en\/actualites_recherche\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\/","url":"https:\/\/elainternational.eu\/en\/actualites_recherche\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\/","name":"Lactosylceramide as a novel target for Substrate Reduction Therapy in Krabbe disease - ELA International","isPartOf":{"@id":"https:\/\/elainternational.eu\/en\/#website"},"datePublished":"2025-11-21T09:01:46+00:00","dateModified":"2025-11-21T09:25:53+00:00","breadcrumb":{"@id":"https:\/\/elainternational.eu\/en\/actualites_recherche\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\/#breadcrumb"},"inLanguage":"en-US","potentialAction":[{"@type":"ReadAction","target":["https:\/\/elainternational.eu\/en\/actualites_recherche\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\/"]}]},{"@type":"BreadcrumbList","@id":"https:\/\/elainternational.eu\/en\/actualites_recherche\/lactosylceramide-as-a-novel-target-for-substrate-reduction-therapy-in-krabbe-disease\/#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https:\/\/elainternational.eu\/en\/"},{"@type":"ListItem","position":2,"name":"Lactosylceramide as a novel target for Substrate Reduction Therapy in Krabbe disease"}]},{"@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\/23610","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=23610"}],"wp:term":[{"taxonomy":"news_research_categories","embeddable":true,"href":"https:\/\/elainternational.eu\/en\/wp-json\/wp\/v2\/news_research_categories?post=23610"},{"taxonomy":"news_research_tags","embeddable":true,"href":"https:\/\/elainternational.eu\/en\/wp-json\/wp\/v2\/news_research_tags?post=23610"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}