{"id":2264,"date":"2024-11-20T05:57:19","date_gmt":"2024-11-20T04:57:19","guid":{"rendered":"https:\/\/rvh-synergie.org\/actualites\/une-nouvelle-strategie-pour-ameliorer-la-therapie-genique-contre-la-drepanocytose\/"},"modified":"2024-11-20T05:57:22","modified_gmt":"2024-11-20T04:57:22","slug":"une-nouvelle-strategie-pour-ameliorer-la-therapie-genique-contre-la-drepanocytose","status":"publish","type":"post","link":"https:\/\/rvh-synergie.org\/actualites\/une-nouvelle-strategie-pour-ameliorer-la-therapie-genique-contre-la-drepanocytose\/","title":{"rendered":"Une nouvelle strat\u00e9gie pour am\u00e9liorer la th\u00e9rapie g\u00e9nique contre la dr\u00e9panocytose"},"content":{"rendered":"<div>\n<div class=\"article-gallery lightGallery\">\n<div data-thumb=\"https:\/\/scx1.b-cdn.net\/csz\/news\/tmb\/2024\/a-new-strategy-to-enha.jpg\" data-src=\"https:\/\/scx2.b-cdn.net\/gfx\/news\/hires\/2024\/a-new-strategy-to-enha.jpg\" data-sub-html=\"Cyclosporin H Improves the Transduction of CD34+ Cells with an Anti-Sickling Globin Vector, a Possible Therapeutic Approach for Sickle Cell Disease. Credit: Mary Ann Liebert, Inc.\">\n<figure class=\"article-img\">\n<\/figure><\/div>\n<\/div>\n<p>Les chercheurs ont d\u00e9crit dans la revue une nouvelle approche prometteuse pour utiliser la th\u00e9rapie g\u00e9nique pour traiter la dr\u00e9panocytose. <i>Th\u00e9rapie g\u00e9nique humaine<\/i>. Pour am\u00e9liorer l&rsquo;efficacit\u00e9 de la th\u00e9rapie g\u00e9nique lors de l&rsquo;utilisation du transfert de g\u00e8ne de la b\u00eata-globine anti-falciforme, ils ont ajout\u00e9 de la cyclosporine (CsH) pour augmenter la transduction en inhibant les facteurs de restriction lentiviraux.<\/p>\n<p>Anne Galy, Ph.D., de l&rsquo;Inserm, et ses co-auteurs, ont appliqu\u00e9 une nouvelle strat\u00e9gie de th\u00e9rapie g\u00e9nique bas\u00e9e sur les lentivirus au CD34<sup>+<\/sup> cellules prog\u00e9nitrices et souches h\u00e9matopo\u00ef\u00e9tiques (HSPC) obtenues \u00e0 partir de sang de cordon. Ils ont d\u2019abord d\u00e9montr\u00e9 que l\u2019ajout de CsH am\u00e9liorait les niveaux d\u2019expression du transg\u00e8ne dans CD34<sup>+<\/sup> cellules, qui sont rest\u00e9es viables et fonctionnelles apr\u00e8s avoir \u00e9t\u00e9 trait\u00e9es.<\/p>\n<p>Ensuite, ils ont transduit le CD34<sup>+<\/sup> cellules obtenues \u00e0 partir du sang de cordon de nouveau-n\u00e9s dr\u00e9panocytaires. Les cellules ont \u00e9t\u00e9 transduites avec un vecteur lentiviral d\u00e9livrant le g\u00e8ne codant pour la prot\u00e9ine b\u00eata-globine HbAS3 anti-falciforme. Les enqu\u00eateurs ont montr\u00e9 que la transduction lentivirale \u00e9tait am\u00e9lior\u00e9e par la CsH et conduisait \u00e0 des niveaux th\u00e9rapeutiques d&rsquo;expression de l&rsquo;HbAs3.<\/p>\n<p>\u00ab\u00a0En plus de sa capacit\u00e9 \u00e0 am\u00e9liorer l&rsquo;efficacit\u00e9 de la transduction, il a d\u00e9j\u00e0 \u00e9t\u00e9 signal\u00e9 que la CsH pr\u00e9servait la viabilit\u00e9 cellulaire\u00a0\u00bb, ont d\u00e9clar\u00e9 les enqu\u00eateurs. \u00ab\u00a0Nos donn\u00e9es ont confirm\u00e9 les \u00e9tudes publi\u00e9es montrant que les HSPC trait\u00e9es avec CsH semblent conserver un potentiel de diff\u00e9renciation h\u00e9matopo\u00ef\u00e9tique normal.\u00a0\u00bb<\/p>\n<p>\u00ab\u00a0Il s&rsquo;agit d&rsquo;une avanc\u00e9e passionnante \u00e0 la fois pour la th\u00e9rapie g\u00e9nique ex vivo en g\u00e9n\u00e9ral et pour l&rsquo;am\u00e9lioration du traitement des patients atteints de dr\u00e9panocytose en particulier\u00a0\u00bb, d\u00e9clare le r\u00e9dacteur en chef de <i>Th\u00e9rapie g\u00e9nique humaine<\/i> Thomas Gallagher, Ph.D., de la facult\u00e9 de m\u00e9decine Chan de l&rsquo;Universit\u00e9 du Massachusetts.<\/p>\n<p>                                        <!-- print only --><\/p><\/div>\n","protected":false},"excerpt":{"rendered":"<p>Les chercheurs ont d\u00e9crit dans la revue une nouvelle approche prometteuse pour utiliser la th\u00e9rapie<\/p>\n","protected":false},"author":1,"featured_media":2265,"comment_status":"closed","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[86],"tags":[1021,90,881,1064,244,8,650,1063,47],"class_list":["post-2264","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-les-maladies","tag-ameliorer","tag-contre","tag-drepanocytose","tag-genique","tag-nouvelle","tag-pour","tag-strategie","tag-therapie","tag-une","generate-columns","tablet-grid-50","mobile-grid-100","grid-parent","grid-50","resize-featured-image"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v23.4 - 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