{"id":3173,"date":"2025-03-10T17:19:24","date_gmt":"2025-03-10T14:19:24","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3173"},"modified":"2025-05-07T10:15:23","modified_gmt":"2025-05-07T07:15:23","slug":"sonothera-presents-new-data-of-genetic-medicine-using-targeted-ultrasound-mediated-delivery-umd-in-treating-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/sonothera-presents-new-data-of-genetic-medicine-using-targeted-ultrasound-mediated-delivery-umd-in-treating-duchenne-muscular-dystrophy\/","title":{"rendered":"SonoThera, Duchenne Musk\u00fcler Distrofi Tedavisinde Hedefli, Ultrason Arac\u0131l\u0131 \u0130letim (UMD) Kullanan Genetik T\u0131p Hakk\u0131nda Yeni Veriler Sunacak"},"content":{"rendered":"<p class=\"wp-block-paragraph\">SonoThera, Duchenne Musk\u00fcler Distrofi tedavisinde hedefli Ultrason Arac\u0131l\u0131 \u0130letim (UMD) kullanan genetik t\u0131bb\u0131n potansiyelini g\u00f6steren yeni verileri 2025 MDA Konferans\u0131nda sunacak.<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Yeni klinik \u00f6ncesi veriler, SonoThera&#039;nin yeni, viral olmayan UMD teknolojisinin genetik ila\u00e7lar sunma yetene\u011fini g\u00f6stererek, iskelet, kalp ve diyafram kaslar\u0131nda tam uzunlukta distrofin protein ekspresyonuna olanak sa\u011flad\u0131\u011f\u0131n\u0131 g\u00f6stermektedir.<\/li>\n\n\n\n<li>SonoThera&#039;nin invaziv olmayan yakla\u015f\u0131m\u0131, g\u00fcvenli ve tekrarlanabilir bir \u015fekilde boyut k\u0131s\u0131tlamas\u0131 olmaks\u0131z\u0131n \u00e7e\u015fitli genetik ila\u00e7lar\u0131n geni\u015f, son derece hedefli biyolojik da\u011f\u0131t\u0131m\u0131n\u0131 sa\u011flamak i\u00e7in geli\u015ftirilmektedir.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Yeni nesil genetik ila\u00e7lar geli\u015ftirerek insan hastal\u0131klar\u0131n\u0131n temel nedenini tedavi etmeye adanm\u0131\u015f bir biyoteknoloji \u015firketi olan SonoThera, bug\u00fcn 16-19 Mart tarihleri aras\u0131nda Dallas, Teksas&#039;ta d\u00fczenlenecek olan y\u0131ll\u0131k Musk\u00fcler Distrofi Derne\u011fi (MDA) Klinik ve Bilimsel Konferans\u0131&#039;nda yeni veriler sunaca\u011f\u0131n\u0131 duyurdu.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Ultrason Arac\u0131l\u0131 \u0130letim (UMD)<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">SonoThera, di\u011fer gen tedavisi yakla\u015f\u0131mlar\u0131n\u0131n s\u0131n\u0131rlamalar\u0131n\u0131 a\u015fmak \u00fczere tasarlanan d\u00fcnyan\u0131n ilk ultrason arac\u0131l\u0131 viral olmayan gen iletim platformunu geli\u015ftiriyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Klinik \u00f6ncesi veriler, ultrason arac\u0131l\u0131 iletim (UMD) y\u00f6nteminin, fare modellerinde iskelet, kalp ve diyafram kaslar\u0131nda tam uzunlukta distrofin ekspresyonuna izin vererek, yeniden dozlanabilir, kal\u0131c\u0131 bir \u015fekilde viral olmayan genetik yap\u0131lar\u0131 ifade etme yetene\u011fini vurgulamaktad\u0131r. SonoThera, yeniden dozlanabilir bir \u015fekilde boyut k\u0131s\u0131tlamas\u0131 olmaks\u0131z\u0131n \u00e7e\u015fitli genetik ila\u00e7lar\u0131n geni\u015f, olduk\u00e7a hedefli biyolojik da\u011f\u0131l\u0131m\u0131n\u0131 sa\u011flayan, g\u00fcvenli, iyi tolere edilen ve uygun maliyetli olacak \u015fekilde tasarlanm\u0131\u015f, ultrason arac\u0131l\u0131 iletim kullanan tescilli, viral olmayan, invaziv olmayan bir yakla\u015f\u0131m geli\u015ftirmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cDMD tedavisinde tam uzunlukta distrofin verememe, viral vekt\u00f6r imm\u00fcnogenitesi, motor fonksiyonu iyile\u015ftirememe, yeniden dozlama yetersizli\u011fi, g\u00fcvenlik sorunlar\u0131 ve tabii ki tedavinin y\u00fcksek maliyeti gibi \u00f6nemli zorluklara odakland\u0131k,\u201d dedi SonoThera CEO&#039;su Dr. Kenneth Greenberg. \u201cUMD&#039;nin bu sorunlar\u0131 \u00e7\u00f6zebilece\u011fine ve bunu viral olmayan, invaziv olmayan bir \u015fekilde yapabilece\u011fine inan\u0131yoruz. Yakla\u015fan MDA konferans\u0131nda en son verilerimizi payla\u015fmay\u0131 d\u00f6rt g\u00f6zle bekliyoruz.\u201d<\/p>","protected":false},"excerpt":{"rendered":"<p>SonoThera will present new data at the 2025 MDA Conference demonstrating the potential of genetic medicine using targeted, Ultrasound-Mediated Delivery (UMD) in treating Duchenne Muscular Dystrophy. SonoThera, a biotechnology company dedicated to treating the root cause of human diseases through developing the next generation of genetic medicines, today announced it will present new data at [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3174,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,285,286],"class_list":["post-3173","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-full-length-dystrophin","tag-sonothera","tag-ultrasound-mediated-delivery"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3173","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=3173"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3173\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3174"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3173"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3173"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3173"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}