{"id":21205,"date":"2024-08-31T14:43:13","date_gmt":"2024-08-31T18:43:13","guid":{"rendered":"https:\/\/arsacs.com\/?p=21205"},"modified":"2025-09-24T15:46:07","modified_gmt":"2025-09-24T19:46:07","slug":"therapeutic-approaches-for-arsacs-drs-benoit-gentil-et-heather-durham","status":"publish","type":"post","link":"https:\/\/arsacs.com\/fr\/therapeutic-approaches-for-arsacs-drs-benoit-gentil-et-heather-durham\/","title":{"rendered":"\u201cTherapeutic Approaches for ARSACS\u201d- Drs Benoit Gentil et Heather Durham"},"content":{"rendered":"\n<p><\/p>\n\n\n<p>ARSACS is characterised by the lack of sacsin expression, and the formation of intermediate filaments bundles in neurons and fibroblasts derived from skin biopsies. Our strategy over the last years has been aimed at developing a replacement therapy, by peptide and\/or gene vectors. The challenge of gene replacement therapies in ARSACS resides in the size of the sacsin coding sequence which is not compatible with current vectors used for gene therapies. Using our expertise in sacsin domains functions, we have designed a minigene to be used for the resorption of molecular biomarker of the disease. Our laboratory is moving forward the development of replacement therapies. We have generated a vector for gene therapy and are moving forward our project to determine its efficiency in modifying the course of ARSACS pathology in mice models.\u00a0Our project at the preclinical stage also received support from NeuroSphere, the McGill platform dedicated to innovation and partnership in neuroscience research, through the ACCELERATE partnership between Neurosphere and the ARSACS Foundation ($200,000). This convergence of support from the Foundation and NeuroSphere will advance investigations into the development and feasibility of gene therapy for ARSACS.<\/p>\n<p>Financement : 100 000$<\/p>\n<p>Dur\u00e9e: 2i\u00e8me ann\u00e9e d&#8217;un projet de 2 ans<\/p>\n<p>Coordonn\u00e9es:<\/p>\n<p style=\"text-align: left;\"><img loading=\"lazy\" decoding=\"async\" class=\"size-medium wp-image-11359 alignleft\" src=\"https:\/\/arsacs.com\/wp-content\/uploads\/2021\/02\/Durham_Benoit_Nov_2017-2-300x201.jpg\" alt=\"\" width=\"300\" height=\"201\" \/><\/p>\n<p style=\"text-align: left;\">Dr. Heather Durham &amp; Dr. Benoit Gentil<\/p>\n<p style=\"text-align: left;\">Department of Kinesiology and Physical Education, McGill University<br \/>475 Pine Avenue West, room 210, Montreal, Quebec\u00a0 H3A 2B4<\/p>\n<p style=\"text-align: left;\"><a href=\"mailto:heather.durham@mcgill.ca\">heather.durham@mcgill.ca<\/a> ; <a href=\"mailto:benoit.gentil@mcgill.ca\">benoit.gentil@mcgill.ca<\/a><\/p>\n\n\n<p><\/p>\n\n\n\n<p><\/p>\n\n\n\n<p><\/p>\n\n\n\n<p><\/p>\n\n\n\n<p><\/p>\n\n\n\n<p class=\"has-text-align-left\"><\/p>\n\n\n\n<p><\/p>\n","protected":false},"excerpt":{"rendered":"<p>ARSACS is characterised by the lack of sacsin expression, and the formation of intermediate filaments bundles in neurons and fibroblasts derived from skin biopsies. Our strategy over the last years has been aimed at developing a replacement therapy, by peptide and\/or gene vectors. The challenge of gene replacement therapies in ARSACS resides in the size [&hellip;]<\/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-21205","post","type-post","status-publish","format-standard","hentry","category-recherche-2024"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>\u201cTherapeutic Approaches for ARSACS\u201d- Drs Benoit Gentil et Heather Durham - 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\/therapeutic-approaches-for-arsacs-drs-benoit-gentil-et-heather-durham\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"\u201cTherapeutic Approaches for ARSACS\u201d- Drs Benoit Gentil et Heather Durham - ARSACS\" \/>\n<meta property=\"og:description\" content=\"ARSACS is characterised by the lack of sacsin expression, and the formation of intermediate filaments bundles in neurons and fibroblasts derived from skin biopsies. Our strategy over the last years has been aimed at developing a replacement therapy, by peptide and\/or gene vectors. 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