{"id":3350,"date":"2026-08-28T10:28:23","date_gmt":"2026-08-28T08:28:23","guid":{"rendered":"https:\/\/moirefoundation.nl\/?page_id=3350"},"modified":"2026-08-28T10:47:24","modified_gmt":"2026-08-28T08:47:24","slug":"gene-therapy","status":"publish","type":"page","link":"https:\/\/moirefoundation.nl\/en\/gene-therapy\/","title":{"rendered":"Gene therapy"},"content":{"rendered":"\t\t<div data-elementor-type=\"wp-page\" data-elementor-id=\"3350\" class=\"elementor elementor-3350\">\n\t\t\t\t<div class=\"elementor-element elementor-element-93263f3 e-flex e-con-boxed e-con e-parent\" data-id=\"93263f3\" data-element_type=\"container\" data-e-type=\"container\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t\t\t<div class=\"e-con-inner\">\n\t\t<div class=\"elementor-element elementor-element-6c32ff7 e-flex e-con-boxed e-con e-child\" data-id=\"6c32ff7\" data-element_type=\"container\" data-e-type=\"container\">\n\t\t\t\t\t<div class=\"e-con-inner\">\n\t\t\t\t<div class=\"elementor-element elementor-element-888fcee page-title elementor-widget elementor-widget-heading\" data-id=\"888fcee\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<h1 class=\"elementor-heading-title elementor-size-default\">Gene therapy<\/h1>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t<div class=\"elementor-element elementor-element-c96eca9 main-container e-flex e-con-boxed e-con e-parent\" data-id=\"c96eca9\" data-element_type=\"container\" data-e-type=\"container\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t\t\t<div class=\"e-con-inner\">\n\t\t<div class=\"elementor-element elementor-element-5db1738 e-con-full e-flex e-con e-child\" data-id=\"5db1738\" data-element_type=\"container\" data-e-type=\"container\">\n\t\t\t\t<div class=\"elementor-element elementor-element-f3a63c2 elementor-widget elementor-widget-heading\" data-id=\"f3a63c2\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<h4 class=\"elementor-heading-title elementor-size-default\">Towards a gene therapy for UGDH syndrome<\/h4>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-728a3ca elementor-widget elementor-widget-text-editor\" data-id=\"728a3ca\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p align=\"justify\">UGDH syndrome is caused by a change in the UGDH gene. As a result, insufficient UGDH enzyme may be produced, or the enzyme may not function properly, disrupting important biological processes and leading to the symptoms of the syndrome.<\/p><p align=\"justify\">Because the cause of UGDH syndrome is directly linked to a single specific gene, this genetic change provides a clear target for the development of a treatment. In recent years, gene therapy has advanced rapidly, creating new possibilities for treating rare genetic disorders. These advances have made it possible to introduce genetic information into cells, enabling them to produce a functional protein. This makes gene therapy a promising approach to investigate for treating UGDH syndrome.<\/p><p align=\"justify\">To this end, the Moir\u00e9 Foundation has chosen an approach using an AAV vector (adeno-associated virus). AAV vectors are modified viruses that do not cause disease and can be used as a delivery vehicle to transport genetic information into cells in the body. This allows a functional copy of the UGDH gene to be delivered to cells, enabling them to produce sufficient UGDH enzyme that functions normally.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-9c58e8c elementor-widget elementor-widget-image\" data-id=\"9c58e8c\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t<figure class=\"wp-caption\">\n\t\t\t\t\t\t\t\t\t\t<img fetchpriority=\"high\" decoding=\"async\" width=\"1024\" height=\"729\" src=\"https:\/\/moirefoundation.nl\/wp-content\/uploads\/2026\/08\/gene-therapy-1024x729.png\" class=\"attachment-large size-large wp-image-3352\" alt=\"\" srcset=\"https:\/\/moirefoundation.nl\/wp-content\/uploads\/2026\/08\/gene-therapy-1024x729.png 1024w, https:\/\/moirefoundation.nl\/wp-content\/uploads\/2026\/08\/gene-therapy-300x214.png 300w, https:\/\/moirefoundation.nl\/wp-content\/uploads\/2026\/08\/gene-therapy-768x547.png 768w, https:\/\/moirefoundation.nl\/wp-content\/uploads\/2026\/08\/gene-therapy-1536x1093.png 1536w, https:\/\/moirefoundation.nl\/wp-content\/uploads\/2026\/08\/gene-therapy.png 1551w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/>\t\t\t\t\t\t\t\t\t\t\t<figcaption class=\"widget-image-caption wp-caption-text\">From genetic cause to gene therapy: an AAV vector delivers a functional copy of the UGDH gene to cells, with the aim of enabling the production of functional UGDH enzyme.<\/figcaption>\n\t\t\t\t\t\t\t\t\t\t<\/figure>\n\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-6312dea elementor-widget elementor-widget-heading\" data-id=\"6312dea\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<h4 class=\"elementor-heading-title elementor-size-default\">A step-by-step research program<\/h4>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-b5fa383 elementor-widget elementor-widget-text-editor\" data-id=\"b5fa383\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p align=\"justify\">The development of a gene therapy for UGDH syndrome is a careful and step-by-step process. Before a treatment can be administered to patients, several stages of research need to be completed. Each stage provides important information about the efficacy, safety and further development of the therapy approach.<\/p><p align=\"justify\">Together with an international research team, the Moir\u00e9 Foundation has established a step-by-step research program for the development of a gene therapy for UGDH syndrome. The program consists of four consecutive phases, from the initial laboratory studies to a potential first application in patients.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-133332a elementor-widget elementor-widget-image\" data-id=\"133332a\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t<figure class=\"wp-caption\">\n\t\t\t\t\t\t\t\t\t\t<img decoding=\"async\" width=\"1024\" height=\"359\" src=\"https:\/\/moirefoundation.nl\/wp-content\/uploads\/2026\/08\/gene-therapy-phases-1024x359.png\" class=\"attachment-large size-large wp-image-3353\" alt=\"\" srcset=\"https:\/\/moirefoundation.nl\/wp-content\/uploads\/2026\/08\/gene-therapy-phases-1024x359.png 1024w, https:\/\/moirefoundation.nl\/wp-content\/uploads\/2026\/08\/gene-therapy-phases-300x105.png 300w, https:\/\/moirefoundation.nl\/wp-content\/uploads\/2026\/08\/gene-therapy-phases-768x270.png 768w, https:\/\/moirefoundation.nl\/wp-content\/uploads\/2026\/08\/gene-therapy-phases-1536x539.png 1536w, https:\/\/moirefoundation.nl\/wp-content\/uploads\/2026\/08\/gene-therapy-phases.png 1749w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/>\t\t\t\t\t\t\t\t\t\t\t<figcaption class=\"widget-image-caption wp-caption-text\">The four consecutive phases form the research pathway outlined by the Moir\u00e9 Foundation for the development of a gene therapy for UGDH syndrome.<\/figcaption>\n\t\t\t\t\t\t\t\t\t\t<\/figure>\n\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-92645bd elementor-widget elementor-widget-text-editor\" data-id=\"92645bd\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p align=\"justify\">The Moir\u00e9 Foundation plays an initiating and coordinating role by bringing together scientific expertise, research partners and funding. Through this collaboration, the foundation aims to contribute to the development of a potential treatment for patients with UGDH syndrome.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t","protected":false},"excerpt":{"rendered":"<p>Gene therapy Towards a gene therapy for UGDH syndrome UGDH syndrome is caused by a change in the UGDH gene. As a result, insufficient UGDH enzyme may be produced, or the enzyme may not function properly, disrupting important biological processes and leading to the symptoms of the syndrome. Because the cause of UGDH syndrome is [&hellip;]<\/p>\n","protected":false},"author":3,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"inline_featured_image":false,"site-sidebar-layout":"no-sidebar","site-content-layout":"page-builder","ast-site-content-layout":"full-width-container","site-content-style":"unboxed","site-sidebar-style":"unboxed","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"disabled","ast-breadcrumbs-content":"","ast-featured-img":"disabled","footer-sml-layout":"","ast-disable-related-posts":"","theme-transparent-header-meta":"enabled","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"set","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"ast-content-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"_locale":"en_US","_original_post":"urn:uuid:767135d0-37cb-4131-97b3-19542585d7a8","footnotes":""},"class_list":["post-3350","page","type-page","status-publish","hentry","en-US"],"rttpg_featured_image_url":null,"rttpg_author":{"display_name":"Jannes","author_link":"https:\/\/moirefoundation.nl\/author\/jannes\/"},"rttpg_comment":0,"rttpg_category":null,"rttpg_excerpt":"Gene therapy Towards a gene therapy for UGDH syndrome UGDH syndrome is caused by a change in the UGDH gene. As a result, insufficient UGDH enzyme may be produced, or the enzyme may not function properly, disrupting important biological processes and leading to the symptoms of the syndrome. Because the cause of UGDH syndrome is&hellip;","_links":{"self":[{"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/pages\/3350","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/comments?post=3350"}],"version-history":[{"count":4,"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/pages\/3350\/revisions"}],"predecessor-version":[{"id":3356,"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/pages\/3350\/revisions\/3356"}],"wp:attachment":[{"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/media?parent=3350"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}