{"id":5894,"date":"2017-04-05T11:37:23","date_gmt":"2017-04-05T09:37:23","guid":{"rendered":"https:\/\/www.institut-biotherapies.fr\/?p=5894\/"},"modified":"2017-04-05T11:38:35","modified_gmt":"2017-04-05T09:38:35","slug":"5894","status":"publish","type":"post","link":"https:\/\/www.institut-biotherapies.fr\/en\/2017\/04\/05\/5894\/","title":{"rendered":"New step towards the treatment of myotubular myopathy: gene therapy restores strength and prolongs lives in affected dogs"},"content":{"rendered":"<p>A team of researchers in France, led by Dr. Ana Buj-Bello (Genethon\/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, achieved a new step towards the treatment of myotubular myopathy by gene therapy. The researchers demonstrated the efficacy of administration of a therapeutic vector by a single intravenous injection and identified the dose that restores long-term muscular strength in a large animal model of the disease.<\/p>\n<p>This work, published today in <em>Molecular Therapy<\/em>, has been achieved thanks to donations from the French Telethon and the support of the Myotubular Trust.<\/p>\n<p>In the present study, Genethon\u2019s team developed and manufactured an adeno-associated virus (AAV) vector able to deliver a normal copy of the MTM1 gene in the entire musculature. The AAV product was administrated by a simple intravenous injection in ten week-old dogs manifesting the first symptoms of the disease \u2013 instead of the locoregional route of administration used in previous studies (<em>Science Translational Medicine, January 2014<\/em>).<\/p>\n<p>The treatment restored whole-body muscle strength and function, and prolonged the life of affected dogs. Treated dogs were indistinguishable from normal animals 9 months after product injection.<\/p>\n<p>&nbsp;<\/p>\n<p><iframe loading=\"lazy\" src=\"https:\/\/www.youtube.com\/embed\/VmG5Tc2X9KI\" width=\"560\" height=\"315\" frameborder=\"0\" allowfullscreen=\"allowfullscreen\"><\/iframe><\/p>\n","protected":false},"excerpt":{"rendered":"<p>A team of researchers in France, led by Dr. Ana Buj-Bello (Genethon\/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, achieved a new step towards the treatment of myotubular myopathy by gene therapy. The researchers demonstrated the efficacy of administration of a therapeutic vector by a single intravenous &hellip; <a href=\"https:\/\/www.institut-biotherapies.fr\/en\/2017\/04\/05\/5894\/\">[Read more]<\/a><\/p>\n","protected":false},"author":3,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[3],"tags":[],"class_list":["post-5894","post","type-post","status-publish","format-standard","hentry","category-news"],"_links":{"self":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/posts\/5894","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/comments?post=5894"}],"version-history":[{"count":2,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/posts\/5894\/revisions"}],"predecessor-version":[{"id":5896,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/posts\/5894\/revisions\/5896"}],"wp:attachment":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/media?parent=5894"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/categories?post=5894"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/tags?post=5894"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}