{"id":19101,"date":"2024-07-04T18:21:03","date_gmt":"2024-07-04T16:21:03","guid":{"rendered":"http:\/\/www.fbg.ub.edu\/?page_id=19101"},"modified":"2024-07-04T18:24:56","modified_gmt":"2024-07-04T16:24:56","slug":"variantx-potential-novel-gene-therapy-for-er-stress-related-rare-liver-disorders","status":"publish","type":"page","link":"https:\/\/www.fbg.ub.edu\/es\/que-hacemos\/tecnologias-materiales-y-licencias\/variantx-potential-novel-gene-therapy-for-er-stress-related-rare-liver-disorders\/","title":{"rendered":"VariantX &#8211; Potential novel gene therapy for ER-stress-related rare liver disorders"},"content":{"rendered":"<h3>Introduction<\/h3>\n<p>Endoplasmic Reticulum stress plays a key role in the development of a broad number of diseases of different etiologies with genetic and metabolic origins: diabetes, cardiovascular, cancer, aging, neurodegenerative diseases. The effective resolution or amelioration of ER-stress holds significant potential as a single or combined therapy for rare diseases lacking an efficient treatment. This technology has been validated in human in vitro models and in vivo mouse model.<\/p>\n<h3><strong>The problem<\/strong><\/h3>\n<ul>\n<li>ER-stress is a common hallmark of a broad range of diseases without effective therapeutic solution.<\/li>\n<li>Liver is one of the most affected organs by ER stress.<\/li>\n<li>AATD (Alpha-1 antitrypsin deficiency) disease is a genetic disease with limited therapeutic options.<\/li>\n<li>WD gene replacement requires a boost to tackle the liver ER-stress in patients.<\/li>\n<\/ul>\n<h3><strong>The solution<\/strong><\/h3>\n<ul>\n<li>We have identified the therapeutic potential of variants of the Mitofusin 2 (MFN2) gene to restore the normal ER homeostasis.<\/li>\n<li>We propose VariantX, a novel monogenic gene therapy based on these MFN2 variants with a primary focus on Wilson Disease and AATD Disease. Other potential indications: NAFLD, NASH, Acute kidney injury, renal fibrosis.<\/li>\n<\/ul>\n<h3><strong>Current stage of development<\/strong><\/h3>\n<p>The therapeutic potential of VariantX has been validated in human in vitro models and murine in vivo models of liver disease.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Introduction Endoplasmic Reticulum stress plays a key role in the development of a broad number of diseases of different etiologies&#8230;<\/p>\n","protected":false},"author":2,"featured_media":19098,"parent":1395,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"page-tecnologies.php","meta":{"_acf_changed":false,"inline_featured_image":false,"_monsterinsights_skip_tracking":false,"_monsterinsights_sitenote_active":false,"_monsterinsights_sitenote_note":"","_monsterinsights_sitenote_category":0,"footnotes":""},"class_list":["post-19101","page","type-page","status-publish","has-post-thumbnail","hentry"],"acf":[],"_links":{"self":[{"href":"https:\/\/www.fbg.ub.edu\/es\/wp-json\/wp\/v2\/pages\/19101","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.fbg.ub.edu\/es\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/www.fbg.ub.edu\/es\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/www.fbg.ub.edu\/es\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.fbg.ub.edu\/es\/wp-json\/wp\/v2\/comments?post=19101"}],"version-history":[{"count":3,"href":"https:\/\/www.fbg.ub.edu\/es\/wp-json\/wp\/v2\/pages\/19101\/revisions"}],"predecessor-version":[{"id":19105,"href":"https:\/\/www.fbg.ub.edu\/es\/wp-json\/wp\/v2\/pages\/19101\/revisions\/19105"}],"up":[{"embeddable":true,"href":"https:\/\/www.fbg.ub.edu\/es\/wp-json\/wp\/v2\/pages\/1395"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.fbg.ub.edu\/es\/wp-json\/wp\/v2\/media\/19098"}],"wp:attachment":[{"href":"https:\/\/www.fbg.ub.edu\/es\/wp-json\/wp\/v2\/media?parent=19101"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}