{"id":21098,"date":"2024-08-31T12:11:30","date_gmt":"2024-08-31T16:11:30","guid":{"rendered":"https:\/\/arsacs.com\/?p=21098"},"modified":"2026-03-16T18:16:38","modified_gmt":"2026-03-16T22:16:38","slug":"developing-conditional-mouse-models-and-new-approaches-to-treating-arsacs-dr-stefan-strack-3","status":"publish","type":"post","link":"https:\/\/arsacs.com\/fr\/developing-conditional-mouse-models-and-new-approaches-to-treating-arsacs-dr-stefan-strack-3\/","title":{"rendered":"\u201c D\u00e9veloppement de mod\u00e8les murins conditionnels et de nouvelles approches th\u00e9rapeutiques pour l'ARSACS \u201d \u2013 Dr Stefan Strack"},"content":{"rendered":"<p>Mouse models are invaluable for studying disease mechanisms and ultimately treatment. The <em>Sacs<\/em> La souris \u00ab knock-out \u00bb (KO) constitue un mod\u00e8le fid\u00e8le de l'ARSACS, pr\u00e9sentant une ataxie, une faiblesse musculaire, une d\u00e9g\u00e9n\u00e9rescence c\u00e9r\u00e9belleuse et, comme nous l'avons r\u00e9cemment d\u00e9montr\u00e9, des troubles de l'apprentissage et de la m\u00e9moire. Dans le cadre de cette proposition, nous cherchons \u00e0 restaurer par voie pharmacologique les ph\u00e9notypes moteurs et cognitifs chez la <em>Sacs<\/em> KO model of ARSACS with a drug that raises levels of a second messenger (cAMP) in the brain. This drug is currently in preclinical development for other neurological disorders and could therefore be rapidly repurposed for ARSACS treatment. Indeed, our pilot results show that this drug improves spatial memory of <em>Sacs<\/em> KO mice. Second, we propose to develop gene therapy for ARSACS. To this end, we will develop a novel, high-capacity viral vector to express the huge Sacsin protein in the brain of <em>Sacs<\/em> KO mice.\u00a0 Third, we will begin to develop and characterize a conditional <em>Sacs<\/em> KO model to pinpoint where and when <em>Sacs<\/em> loss-of-function causes the spectrum of ARSACS symptoms. While our drug studies are directly translational, both Aims 2 and 3 will generate invaluable reagents that will be shared with the wider ARSACS research community in an effort to expedite therapies.\u00a0<\/p>\n<p><strong>\u00a0<\/strong><\/p>\n<p>Subvention : $100 000<\/p>\n<p style=\"text-align: left;\">Dur\u00e9e : un an<\/p>\n<p>Contact :<\/p>\n<p><img decoding=\"async\" class=\"alignleft\" src=\"https:\/\/arsacs.com\/wp-content\/uploads\/2023\/09\/Strack.jpg\" \/><\/p>\n<p>Dr Stefan Strack, D\u00e9partement des neurosciences et de pharmacologie<\/p>\n<p>Facult\u00e9 de m\u00e9decine Carver de l'Universit\u00e9 de l'Iowa<\/p>\n<p>2-452 BSB, 51 Newton Rd., Iowa City, IA 52242<br \/><span class=\"gI\"><span class=\"qu\" tabindex=\"-1\" role=\"gridcell\" translate=\"no\"><span class=\"go\">stefan-strack@uiowa.edu<\/span><\/span><\/span><\/p>\n<p style=\"text-align: center;\">\u00a0<\/p>\n\n\n\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>Mouse models are invaluable for studying disease mechanisms and ultimately treatment. The Sacs knock-out (KO) mouse is a faithful model of ARSACS, displaying ataxia, muscle weakness, cerebellar degeneration, and, as we have recently shown, learning and memory deficits. With this proposal, we seek to pharmacologically rescue motor- and cognitive phenotypes in the Sacs KO model [&#8230;]<\/p>\n","protected":false},"author":3,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[90],"tags":[],"class_list":["post-21098","post","type-post","status-publish","format-standard","hentry","category-research-in-2024"],"_links":{"self":[{"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/posts\/21098","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/comments?post=21098"}],"version-history":[{"count":4,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/posts\/21098\/revisions"}],"predecessor-version":[{"id":25006,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/posts\/21098\/revisions\/25006"}],"wp:attachment":[{"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/media?parent=21098"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/categories?post=21098"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/tags?post=21098"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}