{"id":2723,"date":"2025-02-01T09:45:45","date_gmt":"2025-02-01T06:45:45","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=2723"},"modified":"2025-02-01T14:34:43","modified_gmt":"2025-02-01T11:34:43","slug":"a-novel-mini-crispr-could-be-more-successful-at-editing-the-dystrophin-gene","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/a-novel-mini-crispr-could-be-more-successful-at-editing-the-dystrophin-gene\/","title":{"rendered":"Un nouveau \u00ab mini-CRISPR \u00bb pourrait \u00eatre plus efficace pour modifier le g\u00e8ne de la dystrophine"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Plusieurs groupes de recherche ont tent\u00e9 de cr\u00e9er des syst\u00e8mes CRISPR compacts pouvant s\u2019int\u00e9grer dans un seul vecteur AAV tout en conservant des capacit\u00e9s d\u2019\u00e9dition. Le groupe de Mammoth Biosciences est l\u2019un d\u2019entre eux et il est possible qu\u2019il ait r\u00e9ussi. Ses chercheurs ont commenc\u00e9 par trier les informations de s\u00e9quence g\u00e9n\u00e9tique de plusieurs types de CRISPR pr\u00e9sents dans les micro-organismes.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Lucas Harrington, directeur scientifique et cofondateur de la soci\u00e9t\u00e9 et ancien \u00e9tudiant de Doudna, affirme qu&#039;apr\u00e8s avoir soumis 176 candidats \u00e0 une \u00ab avalanche de tests \u00bb, ils ont d\u00e9couvert \u00ab l&#039;aiguille dans la botte de foin \u00bb. Le meilleur choix a \u00e9t\u00e9 baptis\u00e9 NanoCas ; avec 425 acides amin\u00e9s, il est environ un tiers plus long que Cas9. Il a \u00e9t\u00e9 con\u00e7u par Harrington et ses associ\u00e9s pour couper efficacement l&#039;ADN des mammif\u00e8res.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Les principaux points \u00e0 retenir de l\u2019\u00e9tude NanoCas sont les suivants\u00a0:<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>L&#039;efficacit\u00e9 de l&#039;\u00e9dition correspond \u00e0 celle des syst\u00e8mes CRISPR de premi\u00e8re g\u00e9n\u00e9ration : en ciblant le g\u00e8ne PCSK9 dans le foie de souris in vivo, NanoCas a montr\u00e9 une efficacit\u00e9 d&#039;\u00e9dition saturante d&#039;environ 60%, comparable \u00e0 celle de SaCas9, qui est environ trois fois plus grande. Les deux syst\u00e8mes CRISPR ont r\u00e9duit la prot\u00e9ine s\u00e9rique PCSK9 \u00e0 des niveaux ind\u00e9tectables.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u00c9dition AAV unique robuste sur plusieurs tissus musculaires : NanoCas a d\u00e9montr\u00e9 une \u00e9dition 10% \u00e0 40% du g\u00e8ne de la dystrophine sur le quadriceps, le mollet et le muscle cardiaque dans un mod\u00e8le murin humanis\u00e9 de dystrophie musculaire de Duchenne (DMD), lorsqu&#039;il est d\u00e9livr\u00e9 via un seul vecteur AAV.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Premi\u00e8re d\u00e9monstration de l&#039;\u00e9dition musculaire d&#039;un seul AAV chez des primates non humains : NanoCas a obtenu des efficacit\u00e9s d&#039;\u00e9dition in vivo allant jusqu&#039;\u00e0 30% en ciblant la dystrophine dans le muscle squelettique des macaques cynomolgus. NanoCas a \u00e9galement montr\u00e9 une \u00e9dition 15% sur le c\u0153ur, par rapport \u00e0 10% avec SaCas9. L&#039;analyse du tissu h\u00e9patique a montr\u00e9 une \u00e9dition hors cible minimale.<\/li>\n<\/ul>","protected":false},"excerpt":{"rendered":"<p>Several research groups have been attempting to create compact CRISPR systems that can fit into a single AAV vector while maintaining editing capabilities. The group at Mammoth Biosciences is one of these; they might have found success. Its researchers started by sorting through genetic sequence information from several CRISPR types that are found in microorganisms. [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":2724,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[66,226,72,227,225,228],"class_list":["post-2723","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-duchenne-muscular-dystrophy","tag-dystrophin-gene","tag-gene-editing","tag-mammoth-biosciences","tag-mini-crispr","tag-nanocas"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/2723","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/comments?post=2723"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/2723\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/2724"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=2723"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=2723"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=2723"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}