{"id":1722,"date":"2021-03-01T17:14:32","date_gmt":"2021-03-01T15:14:32","guid":{"rendered":"https:\/\/www.institut-myologie.org\/imotion\/?p=1722&lang=en"},"modified":"2021-03-04T12:38:15","modified_gmt":"2021-03-04T10:38:15","slug":"dmd-start-motion-gene-therapy-trial-using-microdystrophin-designed-genethon-collaboration-sarepta-therapeutics","status":"publish","type":"post","link":"https:\/\/www.institut-myologie.org\/imotion\/2021\/03\/01\/dmd-start-motion-gene-therapy-trial-using-microdystrophin-designed-genethon-collaboration-sarepta-therapeutics\/?lang=en","title":{"rendered":"DMD: start at I-Motion of a gene therapy trial using a microdystrophin designed by Genethon in collaboration with Sarepta Therapeutics"},"content":{"rendered":"

The gene therapy used in this trial combines an AAV-type viral vector and a shortened version of the dystrophin gene, the microdystrophin, designed by researchers and experts from Genethon – a laboratory funded by AFM-Telethon – in collaboration with the team of Prof. George Dickson (University of London) and the team of Caroline Le Guiner in Nantes (gene therapy laboratory, CHU \/ Inserm).\u00a0<\/span><\/p>\n

On the basis of the positive results observed in preclinical studies in terms of efficacy and safety (Nature Communications, July 2017), G\u00e9n\u00e9thon and Sarepta Therapeutics have announced a collaboration to finalize the preclinical development then co-develop the clinical program.\u00a0<\/span><\/p>\n

As part of this international trial coordinated by Prof. Francesco Muntoni (Great Ormond Street Hospital, London), young boys with Duchenne muscular dystrophy will be included in France,\u00a0 <\/span>United Kingdom, United States and Israel.\u00a0<\/span><\/p>\n

In France, the trial will take place in several research centres. In addition to I-Motion for Paris (Dr Silvana De Lucia), there will be the Hautepierre Hospital in Strasbourg (Prof. Vincent Laugel) and the hospitals in Brest, Bordeaux, Lyon, Marseille and Lille.\u00a0<\/span><\/p>\n","protected":false},"excerpt":{"rendered":"

The gene therapy used in this trial combines an AAV-type viral vector and a shortened version of the dystrophin gene, the microdystrophin, designed by researchers and experts from Genethon – a laboratory funded by AFM-Telethon – in collaboration with the team of Prof. George Dickson (University of London) and the team of Caroline Le Guiner … 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":[15],"tags":[],"class_list":["post-1722","post","type-post","status-publish","format-standard","hentry","category-clinical-news"],"acf":[],"yoast_head":"\nDMD: start at I-Motion of a gene therapy trial using a microdystrophin designed by Genethon in collaboration with Sarepta Therapeutics - I-Motion<\/title>\n<meta name=\"description\" content=\"The gene therapy used in this trial combines an AAV-type viral vector and a shortened version of the dystrophin gene, the microdystrophin.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.institut-myologie.org\/imotion\/2021\/03\/01\/dmd-start-motion-gene-therapy-trial-using-microdystrophin-designed-genethon-collaboration-sarepta-therapeutics\/?lang=en\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"DMD: start at I-Motion of a gene therapy trial using a microdystrophin designed by Genethon in collaboration with Sarepta Therapeutics - 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