{"id":122217,"date":"2021-05-02T21:22:21","date_gmt":"2021-05-03T04:22:21","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2021\/05\/a-milestone-in-muscular-dystrophy-therapy"},"modified":"2021-05-02T21:22:21","modified_gmt":"2021-05-03T04:22:21","slug":"a-milestone-in-muscular-dystrophy-therapy","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2021\/05\/a-milestone-in-muscular-dystrophy-therapy","title":{"rendered":"A milestone in muscular dystrophy therapy"},"content":{"rendered":"<p><a class=\"aligncenter blog-photo\" href=\"https:\/\/lifeboat.com\/blog.images\/a-milestone-in-muscular-dystrophy-therapy.jpg\"><\/a><\/p>\n<p>Muscle stem cells enable our muscle to build up and regenerate over a lifetime through exercise. But if certain muscle genes are mutated, the opposite occurs. In patients suffering from muscular dystrophy, the skeletal muscle already starts to weaken in childhood. Suddenly, these children are no longer able to run, play the piano or climb the stairs, and often they are dependent on a wheelchair by the age of 15. Currently, no therapy for this condition exists.<\/p>\n<p>\u201cNow, we are able to access these patients\u2019 gene mutations using CRISPR-Cas9 technology,\u201d explains Professor Simone Spuler, head of the Myology Lab at the Experimental and Clinical Research Center (ECRC), a joint institution of the Max Delbr\u00fcck Center for Molecular Medicine in the Helmholtz Association and Charit\u00e9 \u2014 Universit\u00e4tsmedizin Berlin. \u201cWe care for more than 2000 patients at the Charit\u00e9 outpatient clinic for muscle disorders, and quickly recognized the potential of the new technology.\u201d The researchers immediately started working with some of the affected families, and have now presented their results in the journal <i>JCI Insight<\/i>. In the families studied, the parents were healthy and had no idea they possessed a mutated gene. The children all inherited a copy of the disease mutation from both parents.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Muscle stem cells enable our muscle to build up and regenerate over a lifetime through exercise. But if certain muscle genes are mutated, the opposite occurs. In patients suffering from muscular dystrophy, the skeletal muscle already starts to weaken in childhood. Suddenly, these children are no longer able to run, play the piano or climb [\u2026]<\/p>\n","protected":false},"author":621,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11,412,1495],"tags":[],"class_list":["post-122217","post","type-post","status-publish","format-standard","hentry","category-biotech-medical","category-genetics","category-health"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/122217","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/621"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=122217"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/122217\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=122217"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=122217"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=122217"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}