{"id":26866,"date":"2026-09-11T08:01:48","date_gmt":"2026-09-11T12:01:48","guid":{"rendered":"https:\/\/arsacs.com\/?p=26866"},"modified":"2026-09-11T08:06:26","modified_gmt":"2026-09-11T12:06:26","slug":"advancing-a-nanoparticle-based-gene-replacement-therapy-for-arsacs-drs-gentil-durham","status":"publish","type":"post","link":"https:\/\/arsacs.com\/fr\/advancing-a-nanoparticle-based-gene-replacement-therapy-for-arsacs-drs-gentil-durham\/","title":{"rendered":"D\u00e9veloppement d'une th\u00e9rapie de remplacement g\u00e9nique \u00e0 base de nanoparticules pour le syndrome d'ARSACS \u2013 Drs Gentil et Durham"},"content":{"rendered":"

This proposal aims to advance a gene replacement therapy for ARSACS based on a proprietary functional sacsin minigene (minisacsinV2) developed by the investigators. The central hypothesis is that delivery of minisacsinV2 using QurCan’s C-TERP nanoparticle platform, either as DNA or mRNA, will restore sacsin function and delay or reverse disease progression when administered to symptomatic ARSACS mice. Previous work from the group demonstrated that AAV9-mediated minisacsinV2 delivery delayed disease onset in presymptomatic mice but failed in symptomatic animals due to insufficient transgene expression. The current proposal seeks to overcome this limitation using a non-viral nanoparticle delivery system capable of crossing the blood-brain barrier and targeting neurons. replacement remains one of the most direct and biologically sound therapeutic strategies.<\/p>\n

Subvention : $100 000
\nCe projet est cofinanc\u00e9 par le Fonds fiduciaire Richardson, qui apporte une contribution de $50 000.<\/strong>
\nDur\u00e9e : un an<\/p>\n

\"Dr<\/p>\n

Dr. Benoit Gentil & Dr. Heather Durham<\/strong>
D\u00e9partement de kin\u00e9siologie et d'\u00e9ducation physique, Universit\u00e9 McGill
475 Pine Avenue West, Room 210
Montreal, Quebec H3A 2B4
Contact : heather.durham@mcgill.ca<\/a> ; benoit.gentil@mcgill.ca<\/a><\/strong><\/p>","protected":false},"excerpt":{"rendered":"

Ce projet vise \u00e0 faire progresser une th\u00e9rapie de remplacement g\u00e9nique \u00e0 base de nanoparticules, con\u00e7ue pour r\u00e9tablir la fonction de la saccine dans le syndrome d'ARSACS.<\/p>","protected":false},"author":9,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[34],"tags":[],"class_list":["post-26866","post","type-post","status-publish","format-standard","hentry","category-current"],"_links":{"self":[{"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/posts\/26866","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=26866"}],"version-history":[{"count":7,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/posts\/26866\/revisions"}],"predecessor-version":[{"id":26991,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/posts\/26866\/revisions\/26991"}],"wp:attachment":[{"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/media?parent=26866"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/categories?post=26866"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/tags?post=26866"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}