{"id":1719,"date":"2021-03-01T16:46:56","date_gmt":"2021-03-01T14:46:56","guid":{"rendered":"https:\/\/www.institut-myologie.org\/imotion\/?p=1719"},"modified":"2021-03-30T22:08:54","modified_gmt":"2021-03-30T20:08:54","slug":"dmd-demarrage-a-i-motion-de-lessai-de-therapie-genique-microdystrophine-du-laboratoire-genethon-en-collaboration-avec-sarepta-therapeutics","status":"publish","type":"post","link":"https:\/\/www.institut-myologie.org\/imotion\/2021\/03\/01\/dmd-demarrage-a-i-motion-de-lessai-de-therapie-genique-microdystrophine-du-laboratoire-genethon-en-collaboration-avec-sarepta-therapeutics\/","title":{"rendered":"DMD : D\u00e9marrage \u00e0 I-Motion de l\u2019essai de th\u00e9rapie g\u00e9nique microdystrophine du laboratoire G\u00e9n\u00e9thon en collaboration avec Sarepta Therapeutics"},"content":{"rendered":"<p>La th\u00e9rapie g\u00e9nique utilis\u00e9e dans cet essai associe un vecteur viral de type AAV et une version raccourcie du g\u00e8ne de la dystrophine, la microdystrophine, con\u00e7ue par des chercheurs et experts de G\u00e9n\u00e9thon &#8211; laboratoire financ\u00e9 par l\u2019AFM-T\u00e9l\u00e9thon &#8211; en collaboration avec l\u2019\u00e9quipe du Pr George Dickson (Universit\u00e9 de Londres) et l\u2019\u00e9quipe nantaise de Caroline Le Guiner (laboratoire de th\u00e9rapie g\u00e9nique, CHU\/Inserm).<span class=\"Apple-converted-space\">\u00a0<\/span><\/p>\n<p>Suite aux r\u00e9sultats positifs observ\u00e9s dans les \u00e9tudes pr\u00e9cliniques en termes d\u2019efficacit\u00e9 et de s\u00e9curit\u00e9 (<a href=\"https:\/\/www.nature.com\/articles\/ncomms16105\"><em>Nature Communications<\/em><\/a>, juillet 2017), G\u00e9n\u00e9thon et Sarepta Therapeutics ont annonc\u00e9 une collaboration pour finaliser le d\u00e9veloppement pr\u00e9clinique puis co-d\u00e9velopper le programme clinique.<span class=\"Apple-converted-space\">\u00a0<\/span><\/p>\n<p>Dans le cadre de cet essai international, coordonn\u00e9 par le Pr Francesco Muntoni (H\u00f4pital du Great Ormond Street Hospital, Londres), de jeunes gar\u00e7ons atteints de <a href=\"https:\/\/www.afm-telethon.fr\/myopathie-duchenne-microdystrophine-140639\" target=\"_blank\" rel=\"noopener\">myopathie de Duchenne<\/a>\u00a0 seront inclus en France, au Royaume-Uni, aux Etats-Unis et en Isra\u00ebl.<span class=\"Apple-converted-space\">\u00a0<\/span><\/p>\n<p>En France, l&rsquo;essai se d\u00e9roulera dans plusieurs centres investigateurs. Outre I-Motion pour Paris (Dr Silvana De Lucia), il y aura l\u2019H\u00f4pital de Hautepierre \u00e0 Strasbourg (Pr Vincent Laugel) et les h\u00f4pitaux de Brest, Bordeaux, Lyon, Marseille et Lille.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>La th\u00e9rapie g\u00e9nique utilis\u00e9e dans cet essai associe un vecteur viral de type AAV et une version raccourcie du g\u00e8ne de la dystrophine, la microdystrophine, con\u00e7ue par des chercheurs et experts de G\u00e9n\u00e9thon &#8211; laboratoire financ\u00e9 par l\u2019AFM-T\u00e9l\u00e9thon &#8211; en collaboration avec l\u2019\u00e9quipe du Pr George Dickson (Universit\u00e9 de Londres) et l\u2019\u00e9quipe nantaise de Caroline &hellip; <a href=\"https:\/\/www.institut-myologie.org\/imotion\/2021\/03\/01\/dmd-demarrage-a-i-motion-de-lessai-de-therapie-genique-microdystrophine-du-laboratoire-genethon-en-collaboration-avec-sarepta-therapeutics\/\">Continued<\/a><\/p>\n","protected":false},"author":3,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[6],"tags":[],"class_list":["post-1719","post","type-post","status-publish","format-standard","hentry","category-actualites-recherche"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.1.1 - 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