{"id":5849,"date":"2023-09-05T13:03:18","date_gmt":"2023-09-05T11:03:18","guid":{"rendered":"https:\/\/www.areasciencepark.it\/squadra-icgeb-contribuisce-a-dimostrare-lefficacia-della-terapia-genica-in-una-malattia-metabolica-del-fegato\/"},"modified":"2024-07-24T16:06:05","modified_gmt":"2024-07-24T14:06:05","slug":"icgeb-team-instrumental-in-proving-efficacy-of-gene-therapy-in-metabolic-liver-disease","status":"publish","type":"post","link":"https:\/\/www.areasciencepark.it\/en\/icgeb-team-instrumental-in-proving-efficacy-of-gene-therapy-in-metabolic-liver-disease\/","title":{"rendered":"ICGEB Team instrumental in proving efficacy of gene therapy in metabolic liver disease"},"content":{"rendered":"<p>ICGEB Group Leader,\u00a0<a href=\"https:\/\/9ljio.r.bh.d.sendibt3.com\/mk\/cl\/f\/sh\/7nVU1aA6Qs4xydippSjSUzjU86qkNeP\/q2SgA50RKH4Y\" target=\"_blank\" rel=\"noreferrer noopener\">Mouse Molecular Genetics Lab<\/a>,\u00a0<strong>Dr. Andr\u00e9s F. Muro<\/strong>\u00a0and Research Associate,\u00a0<strong>Dr. Giulia Bortolussi\u00a0<\/strong>have taken part in the European research project\u00a0<a href=\"https:\/\/9ljio.r.bh.d.sendibt3.com\/mk\/cl\/f\/sh\/7nVU1aA6Qs4xydippSjSUzjU86qkNeP\/nFFPH30V06OX\" target=\"_blank\" rel=\"noreferrer noopener\">CureCN<\/a>, which aims to develop a curative gene therapy for the ultra-rare\u00a0<strong>Crigler-Najjar syndrome\u00a0<\/strong>(CN) \u2013 a life-threatening liver disease which affects one in a million individuals at birth.<\/p>\n<p>The project commenced in 2013 and is led by\u00a0<a href=\"https:\/\/9ljio.r.bh.d.sendibt3.com\/mk\/cl\/f\/sh\/7nVU1aA6Qs4xydippSjSUzjU86qkNeP\/1Edot__SMEvF\" target=\"_blank\" rel=\"noreferrer noopener\">G\u00e9n\u00e9thon<\/a>, France and sponsored by the European Commission programme H2020. The consortium includes 11\u00a0<a href=\"https:\/\/9ljio.r.bh.d.sendibt3.com\/mk\/cl\/f\/sh\/7nVU1aA6Qs4xydippSjSUzjU86qkNeP\/gJ-Q8EuI2JQS\" target=\"_blank\" rel=\"noreferrer noopener\">partners<\/a>\u00a0from six European countries and has involved the external collaboration of ICGEB which was\u00a0<strong>pivotal in generating pre-clinical data<\/strong>\u00a0using its Ugt1a KO mouse model, thus setting the basis for the subsequent clinical translation of the trial.<\/p>\n<p>The results of the trial were published on 17 August in a\u00a0<strong>joint\u00a0<\/strong><a href=\"https:\/\/9ljio.r.bh.d.sendibt3.com\/mk\/cl\/f\/sh\/7nVU1aA6Qs4xydippSjSUzjU86qkNeP\/T8xjrZVQM4iY\" target=\"_blank\" rel=\"noreferrer noopener\"><strong>manuscript<\/strong><\/a><strong>\u00a0in the New England Journal of Medicine<\/strong>, co-authored by Dr. Bortolussi and Dr. Muro. These represent the\u00a0<strong>first clinical demonstration of the efficacy of gene therapy in a metabolic disease of the liver<\/strong>, demonstrating the safety and tolerance for the treatment as well as its efficacy at the highest dose.<\/p>\n<p>The trial demonstrated restored long-term expression of the missing enzyme with a large reduction in plasma bilirubin levels in the three adult patients treated with the highest dose. The current part of the study, launched in January 2023, aims to confirm the observed effect in a larger number of patients including children aged 10 years and over, the age at which the liver matures. Should the results be conclusive, this would enable a\u00a0<strong>product license application<\/strong>\u00a0at the French and European authorities.<\/p>\n<p>Dr. Muro states:\u00a0\u201cWe are very proud of our contribution to the trial. The obtained in-patient results represent a fundamental step forward towards the application of gene therapy non only for Crigler-Najjar patients, but also for other liver genetic diseases. This is the first report of a long-term correction of a disease caused by a non-secreted liver protein.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"<p>ICGEB Group Leader,\u00a0Mouse Molecular Genetics Lab,\u00a0Dr. Andr\u00e9s F. Muro\u00a0and Research Associate,\u00a0Dr. Giulia Bortolussi\u00a0have taken part in the European research project\u00a0CureCN, which aims to develop a curative gene therapy for the ultra-rare\u00a0Crigler-Najjar syndrome\u00a0(CN) \u2013 a life-threatening liver disease which affects one in a million individuals at birth. The project commenced in 2013 and is led by\u00a0G\u00e9n\u00e9thon, [&hellip;]<\/p>\n","protected":false},"author":2,"featured_media":5850,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[313,301],"tags":[],"class_list":["post-5849","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-from-our-campuses","category-press-releases"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v26.9 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>ICGEB Team instrumental in proving efficacy of gene therapy in metabolic liver disease - Area Science Park<\/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:\/\/www.areasciencepark.it\/en\/icgeb-team-instrumental-in-proving-efficacy-of-gene-therapy-in-metabolic-liver-disease\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"ICGEB Team instrumental in proving efficacy of gene therapy in metabolic liver disease - Area Science Park\" \/>\n<meta property=\"og:description\" content=\"ICGEB Group Leader,\u00a0Mouse Molecular Genetics Lab,\u00a0Dr. Andr\u00e9s F. Muro\u00a0and Research Associate,\u00a0Dr. Giulia Bortolussi\u00a0have taken part in the European research project\u00a0CureCN, which aims to develop a curative gene therapy for the ultra-rare\u00a0Crigler-Najjar syndrome\u00a0(CN) \u2013 a life-threatening liver disease which affects one in a million individuals at birth. The project commenced in 2013 and is led by\u00a0G\u00e9n\u00e9thon, [&hellip;]\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.areasciencepark.it\/en\/icgeb-team-instrumental-in-proving-efficacy-of-gene-therapy-in-metabolic-liver-disease\/\" \/>\n<meta property=\"og:site_name\" content=\"Area Science Park\" \/>\n<meta property=\"article:published_time\" content=\"2023-09-05T11:03:18+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2024-07-24T14:06:05+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.areasciencepark.it\/wp-content\/uploads\/2023\/09\/Andres-F.-Muro-and-Giulia-Bortolussi-ICGEB.png\" \/>\n\t<meta property=\"og:image:width\" content=\"1920\" \/>\n\t<meta property=\"og:image:height\" content=\"760\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/png\" \/>\n<meta name=\"author\" content=\"dev@ssnt\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"dev@ssnt\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"2 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\/\/www.areasciencepark.it\/en\/icgeb-team-instrumental-in-proving-efficacy-of-gene-therapy-in-metabolic-liver-disease\/#article\",\"isPartOf\":{\"@id\":\"https:\/\/www.areasciencepark.it\/en\/icgeb-team-instrumental-in-proving-efficacy-of-gene-therapy-in-metabolic-liver-disease\/\"},\"author\":{\"name\":\"dev@ssnt\",\"@id\":\"https:\/\/www.areasciencepark.it\/en\/#\/schema\/person\/34938641d271e62fd211c99ed7ba7ede\"},\"headline\":\"ICGEB Team instrumental in proving efficacy of gene therapy in metabolic liver disease\",\"datePublished\":\"2023-09-05T11:03:18+00:00\",\"dateModified\":\"2024-07-24T14:06:05+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\/\/www.areasciencepark.it\/en\/icgeb-team-instrumental-in-proving-efficacy-of-gene-therapy-in-metabolic-liver-disease\/\"},\"wordCount\":330,\"image\":{\"@id\":\"https:\/\/www.areasciencepark.it\/en\/icgeb-team-instrumental-in-proving-efficacy-of-gene-therapy-in-metabolic-liver-disease\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/www.areasciencepark.it\/wp-content\/uploads\/2023\/09\/Andres-F.-Muro-and-Giulia-Bortolussi-ICGEB.png\",\"articleSection\":[\"From our campuses\",\"Press releases\"],\"inLanguage\":\"en-US\"},{\"@type\":\"WebPage\",\"@id\":\"https:\/\/www.areasciencepark.it\/en\/icgeb-team-instrumental-in-proving-efficacy-of-gene-therapy-in-metabolic-liver-disease\/\",\"url\":\"https:\/\/www.areasciencepark.it\/en\/icgeb-team-instrumental-in-proving-efficacy-of-gene-therapy-in-metabolic-liver-disease\/\",\"name\":\"ICGEB Team instrumental in proving efficacy of gene therapy in metabolic liver disease - 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