{"id":2621,"date":"2024-12-05T01:57:59","date_gmt":"2024-12-05T00:57:59","guid":{"rendered":"https:\/\/rvh-synergie.org\/actualites\/evaluation-de-la-survie-a-long-terme-et-de-lefficacite-cardiaque-dune-therapie-genique-pour-la-dystrophie-musculaire-de-duchenne\/"},"modified":"2024-12-05T01:58:15","modified_gmt":"2024-12-05T00:58:15","slug":"evaluation-de-la-survie-a-long-terme-et-de-lefficacite-cardiaque-dune-therapie-genique-pour-la-dystrophie-musculaire-de-duchenne","status":"publish","type":"post","link":"https:\/\/rvh-synergie.org\/actualites\/evaluation-de-la-survie-a-long-terme-et-de-lefficacite-cardiaque-dune-therapie-genique-pour-la-dystrophie-musculaire-de-duchenne\/","title":{"rendered":"\u00c9valuation de la survie \u00e0 long terme et de l&rsquo;efficacit\u00e9 cardiaque d&rsquo;une th\u00e9rapie g\u00e9nique pour la dystrophie musculaire de Duchenne"},"content":{"rendered":"<div>\n<div class=\"article-gallery lightGallery\">\n<div data-thumb=\"https:\/\/scx1.b-cdn.net\/csz\/news\/tmb\/2024\/long-term-survival-and.jpg\" data-src=\"https:\/\/scx2.b-cdn.net\/gfx\/news\/hires\/2024\/long-term-survival-and.jpg\" data-sub-html=\"Fibrotic remodeling in skeletal muscle and heart of DMD&lt;sup&gt;MDX&lt;\/sup&gt; rats and impact of delandistrogene moxeparvovec treatment.\">\n<figure class=\"article-img\">\n<\/figure><\/div>\n<\/div>\n<p>Une \u00e9tude r\u00e9cente publi\u00e9e dans la revue <i>Th\u00e9rapie g\u00e9nique humaine<\/i> \u00e9valu\u00e9 la survie \u00e0 long terme et l&rsquo;efficacit\u00e9 cardiaque de la th\u00e9rapie g\u00e9nique delandistrog\u00e8ne moxeparvovec dans un mod\u00e8le de rat atteint de dystrophie musculaire de Duchenne (DMD).<\/p>\n<p>Delandistrogene moxeparvovec utilise un vecteur viral ad\u00e9no-associ\u00e9 (AAV) pour d\u00e9livrer un transg\u00e8ne de micro-dystrophine aux muscles squelettiques et cardiaques.<\/p>\n<p>Rachel Potter et les co-auteurs de Sarepta Therapeutics ont inject\u00e9 du delandistrog\u00e8ne moxeparvovec \u00e0 des rats DMD, puis ont \u00e9valu\u00e9 la marche et divers param\u00e8tres de la maladie cardiaque 12, 24 et 52 semaines apr\u00e8s le traitement.<\/p>\n<p>Le traitement par th\u00e9rapie g\u00e9nique a prolong\u00e9 la survie m\u00e9diane des rats DMD \u00e0 > 25 mois, par rapport \u00e0 la dur\u00e9e de vie m\u00e9diane de 13 mois pour le groupe t\u00e9moin de rats DMD. Par rapport aux rats t\u00e9moins, le traitement par delandistrog\u00e8ne moxeparvovec a entra\u00een\u00e9 des am\u00e9liorations statistiquement significatives des param\u00e8tres cardiaques, se rapprochant des valeurs de type sauvage, avec des avantages suppl\u00e9mentaires en mati\u00e8re de mobilit\u00e9.<\/p>\n<p>Les enqu\u00eateurs ont rapport\u00e9 que \u00ab l&rsquo;expression du transg\u00e8ne \u00e9tait maintenue jusqu&rsquo;\u00e0 plus de 25 mois et que l&rsquo;expression de la microdystrophine \u00e9tait largement distribu\u00e9e dans les muscles squelettiques et cardiaques \u00bb.<\/p>\n<p>\u00ab\u00a0Il s&rsquo;agit de donn\u00e9es encourageantes selon lesquelles la th\u00e9rapie g\u00e9nique cliniquement approuv\u00e9e delandistrogene moxeparvovec produit des am\u00e9liorations fonctionnelles du muscle cardiaque pour lutter contre la cardiomyopathie induite par la DMD\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>Une \u00e9tude r\u00e9cente publi\u00e9e dans la revue Th\u00e9rapie g\u00e9nique humaine \u00e9valu\u00e9 la survie \u00e0 long<\/p>\n","protected":false},"author":1,"featured_media":2622,"comment_status":"closed","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[86],"tags":[1638,2902,284,2900,2899,1064,1137,619,2901,8,1324,1637,1063],"class_list":["post-2621","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-les-maladies","tag-cardiaque","tag-duchenne","tag-dune","tag-dystrophie","tag-evaluation","tag-genique","tag-lefficacite","tag-long","tag-musculaire","tag-pour","tag-survie","tag-terme","tag-therapie","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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