{"id":4743,"date":"2025-10-07T15:37:43","date_gmt":"2025-10-07T12:37:43","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4743"},"modified":"2025-11-03T20:18:29","modified_gmt":"2025-11-03T17:18:29","slug":"sonothera-presents-new-data-at-esgct-2025-demonstrating-expression-of-full-length-human-dystrophin-using-targeted-ultrasound-mediated-delivery-to-treat-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/sonothera-presents-new-data-at-esgct-2025-demonstrating-expression-of-full-length-human-dystrophin-using-targeted-ultrasound-mediated-delivery-to-treat-duchenne-muscular-dystrophy\/","title":{"rendered":"SonoThera, Duchenne Kas Distrofisini Tedavi Etmek \u0130\u00e7in Hedefli, Ultrason Arac\u0131l\u0131 \u0130letim Kullanarak Tam Uzunlukta \u0130nsan Distrofini Ekspresyonunu G\u00f6steren Yeni Verileri ESGCT 2025'te Sunuyor"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u0130nsan hastal\u0131klar\u0131n\u0131n k\u00f6kenini tedavi etmek i\u00e7in yeni nesil genetik ila\u00e7lar geli\u015ftiren bir biyoteknoloji \u015firketi olan SonoThera, bug\u00fcn 7-10 Ekim tarihleri aras\u0131nda \u0130spanya&#039;n\u0131n Sevilla kentinde d\u00fczenlenecek 32. Avrupa Gen ve H\u00fccre Terapisi Derne\u011fi kongresinde yeni veriler sunaca\u011f\u0131n\u0131 duyurdu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Poster sunumu, SonoThera&#039;nin tescilli RIPPLETM ultrason arac\u0131l\u0131 iletim (UMD) teknolojisi kullan\u0131larak iskelet, kalp ve diyafram kaslar\u0131na verilen tam uzunluktaki insan distrofin y\u00fck\u00fc ile Duchenne Musk\u00fcler Distrofisi&#039;nin (DMD) tedavisine odaklanmaktad\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Ultrason Arac\u0131l\u0131 \u0130letim<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Yeni veriler, RIPPLETM&#039;in boyut k\u0131s\u0131tlamas\u0131 olmaks\u0131z\u0131n \u00e7e\u015fitli y\u00fck formatlar\u0131n\u0131n geni\u015f ve son derece hedefli biyolojik da\u011f\u0131t\u0131m\u0131n\u0131 m\u00fcmk\u00fcn k\u0131lma, yeniden dozlanabilen, g\u00fcvenli, iyi tolere edilen ve uygun maliyetli genetik ila\u00e7lar\u0131n \u00fcretilmesini sa\u011flama yetene\u011fini g\u00f6stermektedir. <a href=\"https:\/\/dmdwarrior.com\/tr\/sonothera-ultrasound-mediated-delivery-method-can-deliver-full-length-dystrophin-to-cells\/\" target=\"_blank\" rel=\"noreferrer noopener\">Ultrason Arac\u0131l\u0131 \u0130letim Y\u00f6ntemi Nedir?<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Tam Uzunlukta \u0130nsan Distrofini<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Yeni veriler, UMD teknolojisinin genetik ila\u00e7lar sunma yetene\u011fini, fare ve insan d\u0131\u015f\u0131 primat (NHP) modellerinde iskelet, kalp ve diyafram kas dokusunda tam uzunlukta insan distrofin protein ekspresyonuna izin verme yetene\u011fini g\u00f6stermektedir.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Sonu\u00e7lar, NHP&#039;de endojen seviyelerin 50%&#039;sini a\u015fan y\u00fcksek seviyelerde dayan\u0131kl\u0131 insan distrofin protein ekspresyonunun elde edildi\u011fini g\u00f6stermektedir.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Poster sunumunda, SonoThera&#039;nin ultrason arac\u0131l\u0131 iletim (UMD) kullanan tescilli, viral olmayan, invaziv olmayan yakla\u015f\u0131m\u0131 vurgulanmaktad\u0131r.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">SonoThera CEO&#039;su Dr. Kenneth Greenberg, &quot;Yeni verilerimiz, genetik ila\u00e7lar\u0131n ultrason arac\u0131l\u0131\u011f\u0131yla iletilmesinin potansiyelini ve Duchenne Musk\u00fcler Distrofi gibi karma\u015f\u0131k hastal\u0131klar\u0131n tedavisindeki benzersiz yeteneklerini g\u00f6stermeye devam ediyor,&quot; dedi. &quot;\u00d6zellikle, hedeflenen, tam uzunlukta insan distrofininin iskelet, kalp ve diyafram kaslar\u0131na kal\u0131c\u0131, g\u00fcvenli, tekrarlanabilir ve invaziv olmayan bir \u015fekilde iletilebilmesi, UMD&#039;nin mevcut gen tedavisi uygulama se\u00e7eneklerinin t\u00fcm zorluklar\u0131n\u0131n \u00fcstesinden nas\u0131l gelebilece\u011fini g\u00f6steriyor. Bu sonu\u00e7lar\u0131 bu y\u0131lki ESGCT kongresinde payla\u015fmay\u0131 d\u00f6rt g\u00f6zle bekliyoruz.&quot;<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/tr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne \u0130\u00e7in Klinik Ara\u015ft\u0131rmalar (T\u00fcm Ara\u015ft\u0131rmalar\u0131n Listesi)<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>SonoThera, a biotechnology company dedicated to treating the root cause of human diseases by developing the next generation of genetic medicines, today announced it will present new data at the 32nd congress of the European Society of Gene and Cell Therapy being held October 7-10 in Seville, Spain. The poster presentation focuses on treating Duchenne [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4744,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,432,285,286],"class_list":["post-4743","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-full-length-dystrophin","tag-ripple","tag-sonothera","tag-ultrasound-mediated-delivery"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4743","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=4743"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4743\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4744"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4743"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4743"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4743"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}