{"id":15581,"date":"2021-10-29T15:29:30","date_gmt":"2021-10-29T19:29:30","guid":{"rendered":"https:\/\/arsacs.com\/?p=15581"},"modified":"2026-03-16T18:13:30","modified_gmt":"2026-03-16T22:13:30","slug":"therapeutic-approaches-for-arsacs-drs-benoit-gentil-andheather-durham","status":"publish","type":"post","link":"https:\/\/arsacs.com\/fr\/therapeutic-approaches-for-arsacs-drs-benoit-gentil-andheather-durham\/","title":{"rendered":"&#8220;Therapeutic Approaches for ARSACS&#8221;- Drs. Benoit Gentil and Heather Durham"},"content":{"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.<em>\u00a0<\/em>Our strategy over the last years has been aimed at developing a replacement therapy, by peptide and\/or gene vectors and, because of the chaperone function of sacsin, to investigate the potential of alternative therapies already in development in our laboratory to promote protein chaperoning.\u00a0We have defined minimal domains necessary for the replacement therapy and identified that HDAC inhibitors have a strong therapeutic potential. Our laboratory is moving forward the development of a gene therapy and focused on the \u00a0Development of peptide and gene therapies. We have generated a AAV9 carrying a combination of sacsin domains and generated peptides derived from sacsin domains and will obtain proof of principle of efficiency. Our lab is also working on finding other therapeutic strategies and we are assessing\u00a0the therapeutic potential of brain permeant HDAC inhibitors for ARSACS<strong>.<\/strong>\u00a0We will examine the role of different HDACs inhibitors in resorbing intermediate filaments bundles.<\/p>\n<p>Grant: $80,000<\/p>\n<p>Duration: One year<\/p>\n<p>Contacts<br \/><img loading=\"lazy\" decoding=\"async\" class=\"alignleft size-thumbnail wp-image-3643\" src=\"https:\/\/arsacs.com\/wp-content\/uploads\/2021\/02\/Durham_Benoit_Nov_2017-150x150-2.jpg\" alt=\"\" width=\"150\" height=\"150\" \/>Le Dr Benoit Gentil  et le Dr Heather Durham<\/p>\n<p><span style=\"font-size: revert;\">D\u00e9partement de kin\u00e9siologie et d'\u00e9ducation physique, Universit\u00e9 McGill\u00a0<\/span><\/p>\n<p>475 Pine Avenue West,\u00a0 \u00a0room 210,\u00a0 Montreal (Qu\u00e9bec)\u00a0 H2W 1S4<\/p>\n<p>email: heather.durham@mcgill.ca, <span style=\"font-size: revert;\">\u00a0benoit.gentil@mcgill.ca<\/span><\/p>\n\n\n<p class=\"wp-block-paragraph\"><\/p>","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.\u00a0Our strategy over the last years has been aimed at developing a replacement therapy, by peptide and\/or gene vectors and, because of the chaperone function of sacsin, to investigate the potential of [&#8230;]<\/p>\n","protected":false},"author":3,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[83],"tags":[],"class_list":["post-15581","post","type-post","status-publish","format-standard","hentry","category-research-in-2021"],"_links":{"self":[{"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/posts\/15581","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=15581"}],"version-history":[{"count":9,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/posts\/15581\/revisions"}],"predecessor-version":[{"id":24743,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/posts\/15581\/revisions\/24743"}],"wp:attachment":[{"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/media?parent=15581"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/categories?post=15581"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/arsacs.com\/fr\/wp-json\/wp\/v2\/tags?post=15581"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}