{"id":20816,"date":"2024-06-25T09:49:39","date_gmt":"2024-06-25T07:49:39","guid":{"rendered":"https:\/\/ela-international.preprod.melisagency.fr\/?post_type=news_research&#038;p=20816"},"modified":"2024-07-11T15:22:14","modified_gmt":"2024-07-11T13:22:14","slug":"aav%e2%80%90mediated-gene-delivery-for-the-motor-and-sensory-phenotype-of-adrenomyeloneuropathy","status":"publish","type":"news_research","link":"https:\/\/elainternational.eu\/en\/actualites_recherche\/aav%e2%80%90mediated-gene-delivery-for-the-motor-and-sensory-phenotype-of-adrenomyeloneuropathy\/","title":{"rendered":"AAV\u2010mediated Gene Delivery for the Motor and Sensory Phenotype of Adrenomyeloneuropathy"},"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>AAV\u2010mediated Gene Delivery for the Motor and Sensory Phenotype of Adrenomyeloneuropathy<\/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>Florian Eichler &#8211; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston MA USA &#8211; ELA2019\u2010012I2<\/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>Adrenomyeloneuropathy is a debilitating lifelong disorder that currently has not treatments available. As an inherited disorder, gene correction is clearly necessary to alter the trajectory of disease burden. We have a developed an approach to deliver a healthy copy of the faulty gene directly into brain and spinal cord.<\/p>\n<p>We have tested this in mice with the disease and have made key insights into a cell type critical to this process: neurons in the brain and alongside the spinal cord. With this knowledge we are now improving gene delivery using new viral vectors.<\/p>\n<p>Importantly we have also developed industry partnerships that can help with manufacturing and setting up future clinical trials. Beyond creating a much-needed treatment, we are also through our studies gaining a better understanding of the disease biology of adrenomyeloneuropathy.<\/p>\n<p style=\"font-weight: 400;\">Project financed by ELA up to:\u00a092 300 \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":[107,111,114],"class_list":["post-20816","news_research","type-news_research","status-publish","hentry","news_research_categories-project","news_research_tags-adrenoleukodystrophy","news_research_tags-adrenomyeloneuropathy-amn","news_research_tags-leukodystrophy"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>AAV\u2010mediated Gene Delivery for the Motor and Sensory Phenotype of Adrenomyeloneuropathy - 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\/aav%e2%80%90mediated-gene-delivery-for-the-motor-and-sensory-phenotype-of-adrenomyeloneuropathy\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"AAV\u2010mediated Gene Delivery for the Motor and Sensory Phenotype of Adrenomyeloneuropathy - 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