{"id":3780,"date":"2025-04-30T15:15:46","date_gmt":"2025-04-30T12:15:46","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3780"},"modified":"2025-04-30T15:16:58","modified_gmt":"2025-04-30T12:16:58","slug":"tevard-biosciences-to-present-trna-based-therapy-rescued-full-length-dystrophin-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/tevard-biosciences-to-present-trna-based-therapy-rescued-full-length-dystrophin-in-duchenne-muscular-dystrophy\/","title":{"rendered":"Tevard Biosciences, Duchenne Musk\u00fcler Distrofi Modelinde tRNA Tabanl\u0131 Terapinin Tam Uzunlukta Distrofin ve Motor Fonksiyonunu Kurtard\u0131\u011f\u0131n\u0131 G\u00f6steren Verileri Sunuyor"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Duchenne Musk\u00fcler Distrofi (DMD) tedavisinde Tevard Biosciences&#039; tRNA tedavisinin potansiyelini g\u00f6steren klinik \u00f6ncesi sonu\u00e7lar sunulacakt\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Tevard Biosciences, \u00e7e\u015fitli genetik hastal\u0131klar\u0131 tedavi etmek i\u00e7in tRNA tabanl\u0131 ila\u00e7lar geli\u015ftirmede \u00f6nc\u00fc olan \u00f6zel bir biyoteknoloji firmas\u0131d\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>DMD hastal\u0131k modelinde yap\u0131lan \u00e7al\u0131\u015fmada, Tevard&#039;nin bask\u0131lay\u0131c\u0131 tRNA&#039;s\u0131n\u0131n distrofin proteininin tamam\u0131n\u0131 korudu\u011fu ve motor fonksiyonu geri kazand\u0131rd\u0131\u011f\u0131, herhangi bir olumsuz terap\u00f6tik etki g\u00f6stermedi\u011fi ortaya konuldu.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fman\u0131n bulgular\u0131n\u0131n ayr\u0131nt\u0131lar\u0131, \u015firket taraf\u0131ndan 13-17 May\u0131s 2025 tarihleri aras\u0131nda ger\u00e7ekle\u015fecek Amerikan Gen ve H\u00fccre Terapisi Derne\u011fi (ASGCT) Y\u0131ll\u0131k Toplant\u0131s\u0131&#039;nda s\u00f6zl\u00fc olarak sunulacak.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cToksikli\u011fe dair hi\u00e7bir kan\u0131t olmayan bir hastal\u0131k modelinde tam uzunlukta distrofin proteininin kurtar\u0131lmas\u0131n\u0131 ve motor fonksiyonunun restorasyonunu g\u00f6steren DMD&#039;deki tRNA program\u0131m\u0131z\u0131n klinik \u00f6ncesi \u00e7al\u0131\u015fmalar\u0131n\u0131n sonu\u00e7lar\u0131n\u0131 payla\u015fmay\u0131 d\u00f6rt g\u00f6zle bekliyoruz,\u201d dedi Tevard Biosciences&#039;nin Kurucu Orta\u011f\u0131, Ba\u015fkan\u0131 ve CEO&#039;su Daniel Fischer. \u201cBu erken veriler, bask\u0131lay\u0131c\u0131 tRNA yakla\u015f\u0131m\u0131m\u0131z\u0131n anlams\u0131z mutasyonlara sahip DMD hastalar\u0131 i\u00e7in olduk\u00e7a etkili bir tedavi sunma potansiyelini destekliyor. Bu sonu\u00e7lar\u0131n, \u00f6nc\u00fcm\u00fcz\u00fcn Dilate Kardiyomiyopati modelinde tam uzunlukta Titin proteinini restore etme yetene\u011fini g\u00f6sterdi\u011fi kardiyomiyopatiler de dahil olmak \u00fczere di\u011fer hastal\u0131klara tRNA platformumuzu uygulamak i\u00e7in de iyiye i\u015faret oldu\u011funa inan\u0131yoruz.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00d6nemli Noktalar:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>DMD geninde anlams\u0131z bir mutasyon i\u00e7eren ve insanlarda DMD patolojisinin temel y\u00f6nlerini \u00f6zetleyen D2-mdx fare modeli kullan\u0131larak yap\u0131lan \u00e7al\u0131\u015fmalardan elde edilen klinik \u00f6ncesi sonu\u00e7lar<\/li>\n\n\n\n<li>Tedavi edilen hayvanlar\u0131n kaslar\u0131, dozlamadan 6 hafta sonra doz ba\u011f\u0131ml\u0131 bir \u015fekilde tam uzunlukta distrofin proteini ifade etti<\/li>\n\n\n\n<li>Kurtar\u0131lan protein, 6. haftada vah\u015fi tip distrofin proteinine benzer bir \u015fekilde organize edilir<\/li>\n\n\n\n<li>Dozlamadan 12 hafta sonra, rotarod performans testinde latent s\u00fcredeki art\u0131\u015f ve \u00f6n ve arka bacak kavrama g\u00fcc\u00fcnde \u00f6nemli art\u0131\u015fla g\u00f6sterildi\u011fi gibi motor fonksiyonda \u00f6nemli bir iyile\u015fme oldu.<\/li>\n\n\n\n<li>Davran\u0131\u015fsal veya histolojik de\u011fi\u015fikliklerle \u00f6l\u00e7\u00fclen ana organlarda veya kan kimyas\u0131nda olumsuz tedavi etkilerine dair bir kan\u0131t yoktu<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Tam uzunluktaki distrofinin translasyonunu \u00f6nleyen erken sonland\u0131rma kodonunun bulundu\u011fu anlams\u0131z mutasyonlar, kas distrofisi olan hastalar\u0131n yakla\u015f\u0131k 15%&#039;sinde bulunur ve bozuklu\u011fun daha \u015fiddetli formlar\u0131yla ili\u015fkilidir. Bask\u0131lay\u0131c\u0131 tRNA&#039;lar, antikodonun anlams\u0131z mutasyonlar\u0131 okumak ve tam uzunluktaki, i\u015flevsel proteini geri y\u00fcklemek i\u00e7in de\u011fi\u015ftirildi\u011fi tRNA molek\u00fclleridir. Sadece \u00fc\u00e7 erken sonland\u0131rma kodonu (TGA, TAA ve TAG) oldu\u011fundan, s\u0131n\u0131rl\u0131 say\u0131da bask\u0131lay\u0131c\u0131 tRNA, anlams\u0131z mutasyonlar\u0131 olan t\u00fcm DMD hastalar\u0131n\u0131n tedavisine izin verebilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>Preclinical results demonstrating the potential of Tevard Biosciences&#8217; tRNA therapy for Duchenne Muscular Dystrophy (DMD) will be presented. Tevard Biosciences is a privately held biotechnology firm that is leading the way in developing tRNA-based medicines to treat a variety of genetic illnesses. In a DMD disease model, the study showed that Tevard&#8217;s suppressor tRNA preserved [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3782,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[360,359,357,358],"class_list":["post-3780","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-daniel-fischer","tag-full-length-dystrophin","tag-tevard-biosciences","tag-trna-therapy"],"subtitle":"Tevard Biosciences' tRNA bazl\u0131 tedavi, Duchenne Musk\u00fcler Distrofi hastalar\u0131na tam uzunlukta distrofin vaat ediyor.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3780","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=3780"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3780\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3782"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3780"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3780"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3780"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}