{"id":26941,"date":"2025-09-24T14:39:32","date_gmt":"2025-09-24T18:39:32","guid":{"rendered":"https:\/\/arsacs.com\/?p=26941"},"modified":"2026-09-10T16:45:20","modified_gmt":"2026-09-10T20:45:20","slug":"developing-conditional-mouse-models-and-new-approaches-to-treating-arsacs-dr-strack","status":"publish","type":"post","link":"https:\/\/arsacs.com\/fr\/developing-conditional-mouse-models-and-new-approaches-to-treating-arsacs-dr-strack\/","title":{"rendered":"Developing Conditional Mouse Models and New Approaches to Treating ARSACS – Dr. Strack"},"content":{"rendered":"

Autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS) is one of the most common autosomal-recessive ataxias. Initially identified in the northern Quebec region of Charlevoix-Sagenay, but now diagnosed around the world, ARSACS is caused by loss-of-function mutations in the SACS<\/em> gene. Its gene product, Sascin, is a gigantic multi-domain protein thought to help properly assemble equally large protein complexes. Unlike many models of human diseases, the Sacs<\/em> KO mouse is a faithful model of ARSACS, displaying ataxia, muscle weakness, PC loss, and even dementia as we have recently reported (PMID: 38735882). A better understanding of pathogenic mechanisms in the Sacs<\/em> KO mouse likely has therapeutic implications not only for ARSACS, but for cerebellar disorders in general.<\/p>\n

We have generated a large capacity adenoviral vector to express full-length Sacsin in the brain of Sacs KO mice as poof-of-concept for gene therapy. In parallel, we have made a conditional knock-out (cKO) model of ARSACS in order to identify cell types and brain regions responsible for motor and cognitive deficits in ARSACS.<\/p>\n

Subvention : $100 000
\nThis is a jointly founded project with Action for ARSACS Foundation USA (AFA)<\/strong>
\nDur\u00e9e : un an<\/p>\n

\"\"Dr Stefan Strack<\/strong>
Professor and Vice Chair
Department of Neuroscience and Pharmacology
Facult\u00e9 de m\u00e9decine Carver de l'Universit\u00e9 de l'Iowa
2-452 Bowen Science Building
51 Newton Road
Iowa City, IA 52242, USA
Contact : stefan-strack@uiowa.edu<\/a><\/p>","protected":false},"excerpt":{"rendered":"

This project focuses on developing conditional mouse models and new approaches to treating ARSACS.<\/p>","protected":false},"author":9,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[105],"tags":[],"class_list":["post-26941","post","type-post","status-publish","format-standard","hentry","category-research-in-2025"],"_links":{"self":[{"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/posts\/26941","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\/9"}],"replies":[{"embeddable":true,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/comments?post=26941"}],"version-history":[{"count":7,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/posts\/26941\/revisions"}],"predecessor-version":[{"id":26986,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/posts\/26941\/revisions\/26986"}],"wp:attachment":[{"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/media?parent=26941"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/categories?post=26941"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/tags?post=26941"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}