{"id":21167,"date":"2024-08-31T14:40:59","date_gmt":"2024-08-31T18:40:59","guid":{"rendered":"https:\/\/arsacs.com\/?p=21167"},"modified":"2025-09-24T16:06:09","modified_gmt":"2025-09-24T20:06:09","slug":"developing-conditional-mouse-models-and-new-approaches-to-treating-arsacs-dr-stefan-strack","status":"publish","type":"post","link":"https:\/\/arsacs.com\/fr\/developing-conditional-mouse-models-and-new-approaches-to-treating-arsacs-dr-stefan-strack\/","title":{"rendered":"“Developing conditional mouse models and new approaches to treating ARSACS\u201d – Dr Stefan Strack"},"content":{"rendered":"\n


<\/p>\n\n\n

Mouse models are invaluable for studying disease mechanisms and ultimately treatment. The Sacs<\/em> 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<\/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 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 Sacs<\/em> KO mice.\u00a0 Third, we will begin to develop and characterize a conditional Sacs<\/em> KO model to pinpoint where and when 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

Financement: 100 000$<\/p>\n

Dur\u00e9e:\u00a0 un an<\/p>\n

Coordonn\u00e9es:<\/p>\n

\"Dr.<\/p>\n

Dr. Stefan Strack, Dept. Neuroscience & Pharmacology<\/span><\/p>\n

University of Iowa Carver College of Medicine
2-452 BSB, 51 Newton Rd.,Iowa City, IA 52242 USA<\/p>\n

email: Stefan-strack@uiowa.edu<\/p>","protected":false},"excerpt":{"rendered":"

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 […]<\/p>\n","protected":false},"author":3,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_et_pb_use_builder":"","_et_pb_old_content":"","_et_gb_content_width":"","inline_featured_image":false,"footnotes":""},"categories":[91],"tags":[],"class_list":["post-21167","post","type-post","status-publish","format-standard","hentry","category-recherche-2024"],"yoast_head":"\n"Developing conditional mouse models and new approaches to treating ARSACS\u201d - Dr Stefan Strack - ARSACS<\/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:\/\/arsacs.com\/fr\/developing-conditional-mouse-models-and-new-approaches-to-treating-arsacs-dr-stefan-strack\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\""Developing conditional mouse models and new approaches to treating ARSACS\u201d - Dr Stefan Strack - ARSACS\" \/>\n<meta property=\"og:description\" content=\"Mouse models are invaluable for studying disease mechanisms and ultimately treatment. 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