{"id":4676,"date":"2025-09-29T17:43:53","date_gmt":"2025-09-29T14:43:53","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4676"},"modified":"2025-11-18T12:55:13","modified_gmt":"2025-11-18T09:55:13","slug":"tevard-biosciences-presents-data-demonstrating-production-of-full-length-protein-with-trna-based-therapy-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/tevard-biosciences-presents-data-demonstrating-production-of-full-length-protein-with-trna-based-therapy-for-duchenne-muscular-dystrophy\/","title":{"rendered":"Tevard Biosciences, Duchenne Kas Distrofisi i\u00e7in tRNA Tabanl\u0131 Terapi ile Tam Uzunlukta Protein \u00dcretimini G\u00f6steren Verileri Sunuyor"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Tevard Biosciences, Duchenne kas distrofisi (DMD) ve dilate kardiyomiyopati (DCM-TTNtv) modellerinde tam uzunluktaki fonksiyonel proteinlerin g\u00fc\u00e7l\u00fc bir \u015fekilde restore edildi\u011fini g\u00f6steren yeni klinik \u00f6ncesi verilerin sunuldu\u011funu duyurdu.<\/strong> \u00d6nemlisi, bulgular, son nesil bask\u0131lay\u0131c\u0131 tRNA&#039;lar (suptRNA) ile DMD modellerinde ortalama 70% vah\u015fi tip distrofin proteininin restorasyonunu g\u00f6steren verileri i\u00e7ermekte olup, bu da daha d\u00fc\u015f\u00fck dozlarda anlaml\u0131 klinik sonu\u00e7lar elde etme potansiyelini desteklemektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Veriler, Tevard Biosciences&#039;nin Ba\u015f Bilim Sorumlusu Dr. Elisabeth Gardiner taraf\u0131ndan &quot;Duchenne Musk\u00fcler Distrofi ve Dilate Kardiyomiyopati Tedavisinde Terap\u00f6tik Bask\u0131lay\u0131c\u0131 tRNA&#039;lar\u0131n Kullan\u0131m\u0131&quot; ba\u015fl\u0131kl\u0131 davetli s\u00f6zl\u00fc sunum ve poster oturumunda sunuldu. Sunumlar, 28 Eyl\u00fcl - 3 Ekim 2025 tarihleri aras\u0131nda H\u0131rvatistan&#039;\u0131n Dubrovnik kentinde d\u00fczenlenen 2025 Avrupa Biyokimya Dernekleri Federasyonu (FEBS) &quot;Aminoasil-tRNA Sentetaz Ara\u015ft\u0131rmalar\u0131nda S\u0131n\u0131rlar\u0131n Geni\u015fletilmesi&quot; \u00d6zel Toplant\u0131s\u0131&#039;nda ger\u00e7ekle\u015fti.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Dr. Gardiner, &quot;Bu sonu\u00e7lar, tasarlanm\u0131\u015f bask\u0131lay\u0131c\u0131 tRNA&#039;lar\u0131m\u0131z\u0131n, yaln\u0131zca biyolojik olarak anlaml\u0131 de\u011fil, ayn\u0131 zamanda klinik olarak da umut verici seviyelerde, tam uzunlukta, do\u011fal protein ifadesini geri getirebildi\u011fini g\u00f6steriyor&quot; dedi.<\/strong> &quot;Distrofin ve titin gibi yap\u0131sal proteinlerle u\u011fra\u015f\u0131rken, uygun katlanma, lokalizasyon ve protein-protein etkile\u015fimlerinin hayati \u00f6nem ta\u015f\u0131d\u0131\u011f\u0131 durumlarda, tam uzunlukta bir proteini geri y\u00fcklemek b\u00fcy\u00fck fark yarat\u0131r. Platformumuz, v\u00fccudun bekledi\u011fi \u015fekilde i\u015flev g\u00f6ren do\u011fal proteinler \u00fcretmek i\u00e7in do\u011fal h\u00fccresel mekanizmay\u0131 kullan\u0131r.&quot;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Sunulan Temel Bulgular<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>DMD program\u0131nda, Gln-TAA ve Arg-TGA anlams\u0131z mutasyonlar\u0131n\u0131 hedef alan AAV ile iletilen suptRNA&#039;lar, in vivo tam uzunlukta vah\u015fi tip distrofin seviyelerinin ortalama 70%&#039;sini geri kazand\u0131rd\u0131 ve bu durum motor fonksiyon iyile\u015fmesi ve proteomik biyobelirte\u00e7lerin normalle\u015fmesiyle g\u00fc\u00e7l\u00fc bir korelasyon g\u00f6sterdi.<\/strong><\/li>\n\n\n\n<li>DCM-TTNtv program\u0131nda, suptRNA tedavisi, iPSC&#039;den t\u00fcretilen insan kardiyomiyositlerinde d\u00f6rt g\u00fcn i\u00e7inde tam uzunlukta titin protein ekspresyonunu ve kas\u0131lma yetene\u011fini geri kazand\u0131rd\u0131. Canl\u0131 organizmada, AAV ile iletilen Arg-TGA suptRNA&#039;lar\u0131, bir TTNtv fare modelinde alt\u0131 hafta i\u00e7inde kalpte g\u00fc\u00e7l\u00fc tam uzunlukta titin \u00fcretimini sa\u011flad\u0131 ve proteomik homeostaz\u0131 geri kazand\u0131rd\u0131.<\/li>\n\n\n\n<li>Her iki program da sistemik uygulama sonras\u0131nda doz ba\u011f\u0131ml\u0131 transd\u00fcksiyon, protein kurtarma ve fonksiyonel iyile\u015fme g\u00f6sterdi; tespit edilebilir bir toksisite veya hedef d\u0131\u015f\u0131 etki g\u00f6r\u00fclmedi.<\/li>\n\n\n\n<li>\u00d6zellikle suptRNA ekspresyonu ve protein kurtarma, tedavi sonras\u0131 12 haftaya kadar s\u00fcrd\u00fcr\u00fcld\u00fc ve bu da tek bir intraven\u00f6z dozun ard\u0131ndan terap\u00f6tik etkinin kal\u0131c\u0131l\u0131\u011f\u0131n\u0131 vurgulad\u0131.<\/li>\n\n\n\n<li>Bu veriler, Tevard&#039;nin \u00f6nc\u00fc geli\u015ftirme \u00e7al\u0131\u015fmalar\u0131ndan biri olan DCM-TTNtv program\u0131ndan elde etti\u011fi sonu\u00e7lar\u0131 ilk kez a\u00e7\u0131klad\u0131\u011f\u0131 anlam\u0131na geliyor ve bu da bask\u0131lay\u0131c\u0131 tRNA platformunun genetik olarak farkl\u0131 endikasyonlarda ne kadar \u00e7ok y\u00f6nl\u00fc ve olgun oldu\u011funu daha da vurguluyor.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Tevard Biosciences Kurucu Orta\u011f\u0131, Ba\u015fkan\u0131 ve CEO&#039;su Daniel Fischer, &quot;Bu d\u00fcnya standartlar\u0131ndaki konferansta \u00f6ne \u00e7\u0131kan s\u00f6zl\u00fc sunum ve poster oturumu i\u00e7in se\u00e7ilmek, bask\u0131lay\u0131c\u0131 tRNA platformumuzun etkisini g\u00f6zler \u00f6n\u00fcne seriyor,&quot; dedi. &quot;Bask\u0131lay\u0131c\u0131 tRNA ve vekt\u00f6r tasar\u0131m\u0131ndaki son geli\u015fmeler, DMD ve DCM-TTNtv programlar\u0131m\u0131z\u0131 g\u00fcvenli ve etkili dozlarda klinikte g\u00fcvenle ilerletmek i\u00e7in gereken protein kurtarma seviyelerine ula\u015ft\u0131.&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>Tevard Biosciences announced the presentation of new preclinical data showing potent restoration of full-length functional proteins in models of Duchenne muscular dystrophy (DMD) and dilated cardiomyopathy (DCM-TTNtv). Importantly, the findings include data showing on average 70% restoration of wild-type dystrophin protein in DMD models with the latest generation of suppressor tRNAs (suptRNA), supporting the potential [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4677,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,357,271,358],"class_list":["post-4676","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-full-length-dystrophin","tag-tevard-biosciences","tag-trna","tag-trna-therapy"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4676","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=4676"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4676\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4677"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4676"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4676"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4676"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}