{"id":5315,"date":"2025-12-12T17:44:59","date_gmt":"2025-12-12T14:44:59","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5315"},"modified":"2025-12-12T17:51:07","modified_gmt":"2025-12-12T14:51:07","slug":"ractigen-therapeutics-study-of-rag-18-sarna-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/ractigen-therapeutics-study-of-rag-18-sarna-for-duchenne-muscular-dystrophy\/","title":{"rendered":"Ractigen Therapeutics, Duchenne Musk\u00fcler Distrofisi \u0130\u00e7in \u00c7\u0131\u011f\u0131r A\u00e7\u0131c\u0131 Bir SaRNA Terapisi Olan RAG-18&#039;in IIT \u00c7al\u0131\u015fmas\u0131nda \u0130lk Hastaya Doz Verdi"},"content":{"rendered":"<p class=\"wp-block-paragraph\">K\u00fc\u00e7\u00fck aktive edici RNA (saRNA) terap\u00f6tiklerinin \u00f6nc\u00fc geli\u015ftiricisi Ractigen Therapeutics, bug\u00fcn Pekin&#039;deki \u00c7in T\u0131p Bilimleri Akademisi&#039;ne ba\u011fl\u0131 Pekin Birlik T\u0131p Koleji Hastanesi&#039;nde (PUMCH) RAG-18&#039;in ara\u015ft\u0131rmac\u0131 taraf\u0131ndan ba\u015flat\u0131lan denemesinde (IIT) ilk hastaya ba\u015far\u0131l\u0131 doz uygulamas\u0131n\u0131n yap\u0131ld\u0131\u011f\u0131n\u0131 duyurdu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu d\u00f6n\u00fcm noktas\u0131, Duchenne Musk\u00fcler Distrofisi (DMD) ile m\u00fccadelede d\u00f6n\u00fc\u015ft\u00fcr\u00fcc\u00fc bir an\u0131 i\u015faret ediyor ve RAG-18&#039;i bu alanda potansiyel bir oyun de\u011fi\u015ftirici olarak konumland\u0131r\u0131yor.<\/strong> Yeni bir saRNA mekanizmas\u0131 kullanarak UTRN ekspresyonunu art\u0131ran RAG-18, mevcut tedavilerin s\u0131n\u0131rlamalar\u0131n\u0131n \u00fcstesinden gelmeyi ama\u00e7layan ve bu y\u0131k\u0131c\u0131 durumdan etkilenen t\u00fcm hastalara fayda sa\u011flayabilecek evrensel bir \u00e7\u00f6z\u00fcm sunabilecek devrim niteli\u011finde bir terap\u00f6tik yakla\u015f\u0131m\u0131 temsil etmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu ara\u015ft\u0131rma ekibi, n\u00f6rom\u00fcsk\u00fcler bozukluklar konusunda \u00f6nde gelen bir uzman ve PUMCH N\u00f6roloji B\u00f6l\u00fcm\u00fc M\u00fcd\u00fcr Yard\u0131mc\u0131s\u0131 olan Profes\u00f6r Dai Yi taraf\u0131ndan y\u00f6netilmektedir. \u00c7al\u0131\u015fman\u0131n amac\u0131, RAG-18&#039;in g\u00fcvenli\u011fini ve farmakokinetik profilini de\u011ferlendirirken, DMD hastalar\u0131nda kas fonksiyonunu iyile\u015ftirme potansiyelini ara\u015ft\u0131rmakt\u0131r. <strong>Profes\u00f6r Dai \u015funlar\u0131 s\u00f6yledi: &quot;DMD tedavisinde \u00e7\u0131\u011f\u0131r a\u00e7an bir terap\u00f6tik yakla\u015f\u0131m olan RAG-18 ile bu klinik deneye ba\u015flamaktan heyecan duyuyoruz. Hastalar i\u00e7in yeni, yenilik\u00e7i tedavi se\u00e7eneklerinin \u00f6n\u00fcn\u00fc a\u00e7abilecek ve ya\u015fam kalitelerini \u00f6nemli \u00f6l\u00e7\u00fcde iyile\u015ftirebilecek olumlu klinik sonu\u00e7lar bekliyoruz.&quot;\u201c<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ractigen Therapeutics&#039;nin Kurucusu ve CEO&#039;su Dr. Long-Cheng Li, deneme hakk\u0131ndaki heyecan\u0131n\u0131 \u015fu s\u00f6zlerle dile getirdi: &quot;Bu \u00e7al\u0131\u015fmaya ba\u015flamak, RAG-18 i\u00e7in \u00e7ok \u00f6nemli bir ad\u0131m olup, DMD&#039;nin zorluklar\u0131n\u0131n \u00fcstesinden gelmede benzersiz rol\u00fcn\u00fc vurgulamaktad\u0131r. Bu tedavi, DMD ile ilgili t\u00fcm genetik mutasyonlar\u0131 ele alma potansiyeline sahip olup, RNA aktivasyonu (RNAa) teknolojisini klinik uygulamada devrim niteli\u011finde bir y\u00f6ntem olarak g\u00f6stermekte ve daha \u00f6nce tedavi edilemeyen durumlar i\u00e7in umut sunmaktad\u0131r.&quot;\u201c<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">RAG-18 saRNA nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>RAG-18, RNAa mekanizmas\u0131 yoluyla kas h\u00fccrelerinde UTRN gen ekspresyonunu spesifik olarak hedeflemek ve aktive etmek \u00fczere tasarlanm\u0131\u015f, t\u00fcr\u00fcn\u00fcn ilk \u00f6rne\u011fi bir saRNA aday\u0131d\u0131r.<\/strong> UTRN geni taraf\u0131ndan kodlanan utrofin proteini, yap\u0131sal ve i\u015flevsel olarak distrofine benzer ve yukar\u0131 do\u011fru d\u00fczenlenmesi, DMD kas h\u00fccrelerinde eksik olan distrofinin i\u015flevsel bir ikamesi olarak potansiyel olarak hizmet edebilir ve spesifik mutasyon konumundan ba\u011f\u0131ms\u0131z olarak t\u00fcm DMD hastalar\u0131 i\u00e7in tedavi sa\u011flayabilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Klinik \u00f6ncesi veriler, Ractigen&#039;nin tescilli LiCO\u2122 (lipit konjugeli oligon\u00fckleotid) teknolojisi kullan\u0131larak uygulanan RAG-18&#039;in, UTRN ekspresyonunu etkili bir \u015fekilde art\u0131rd\u0131\u011f\u0131n\u0131, kas hasar\u0131n\u0131 azaltt\u0131\u011f\u0131n\u0131 ve kas fonksiyonunu iyile\u015ftirdi\u011fini, b\u00f6ylece DMD hastalar\u0131n\u0131n tedavisinde \u00f6nemli bir potansiyel g\u00f6sterdi\u011fini ortaya koymaktad\u0131r. <strong>RAG-18, 2024 y\u0131l\u0131nda ABD G\u0131da ve \u0130la\u00e7 \u0130daresi&#039;nden (FDA) Yetim \u0130la\u00e7 Stat\u00fcs\u00fc (ODD) ve Nadir Pediatrik Hastal\u0131k Stat\u00fcs\u00fc (RPDD) (FDA) alarak, DMD hastalar\u0131 i\u00e7in umut vadeden bir tedavi se\u00e7ene\u011fi olarak \u00f6ne \u00e7\u0131km\u0131\u015ft\u0131r.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu Sayfay\u0131 Takip Edin<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne i\u00e7in T\u00fcm Klinik Ara\u015ft\u0131rmalar<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced the successful dosing of the first patient in its investigator-initiated trial (IIT) of RAG-18 at Peking Union Medical College Hospital (PUMCH), Chinese Academy of Medical Sciences in Beijing. This milestone marks a transformative moment in the fight against Duchenne Muscular Dystrophy (DMD), [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5316,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[123,466,467,465,468],"class_list":["post-5315","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-china","tag-ractigen-therapeutics","tag-rag-18","tag-sarna","tag-utrn-expression"],"subtitle":"Ractigen Therapeutics, Pekin'deki Peking Union T\u0131p Fak\u00fcltesi Hastanesi'nde ara\u015ft\u0131rmac\u0131lar\u0131n ba\u015flatt\u0131\u011f\u0131 saRNA terapisi RAG-18 denemesinde ilk hastaya ba\u015far\u0131l\u0131 bir \u015fekilde doz uyguland\u0131\u011f\u0131n\u0131 duyurdu.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5315","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=5315"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5315\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/5316"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=5315"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=5315"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=5315"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}