An American study conducted in collaboration with Genethon has provided proof-of-concept for enzyme replacement therapy in mice. The treatment carried out in mice involved replacing the missing myotubularin in this disease by a laboratory-modified form of myotubularin that is capable of specifically targeting the inside of skeletal muscle cells.<\/p>\n
Intramuscular injections of this product improved contraction and reduced histological abnormalities in the muscles of a mouse model of X-linked myotubular congenital myopathy (Mtm1\u03b44), thus providing proof-of-concept of the potential effectiveness of this therapeutic approach.<\/p>\n
Genethon participated in this study. The laboratory created and supported by the AFM-Telethon has been working for several years on the development of a gene therapy treatment for congenital myotubular myopathy. Preclinical studies have been completed or are underway at the U.S. universities Wake Forrest and Washington in Seattle.<\/p>\n","protected":false},"excerpt":{"rendered":"
An American study conducted in collaboration with Genethon has provided proof-of-concept for enzyme replacement therapy in mice. The treatment carried out in mice involved replacing the missing myotubularin in this disease by a laboratory-modified form of myotubularin that is capable of specifically targeting the inside of skeletal muscle cells. Intramuscular injections of this product improved … [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-4519","post","type-post","status-publish","format-standard","hentry","category-news"],"_links":{"self":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/posts\/4519","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=4519"}],"version-history":[{"count":0,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/posts\/4519\/revisions"}],"wp:attachment":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/media?parent=4519"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/categories?post=4519"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/tags?post=4519"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}