{"id":3122,"date":"2025-03-08T12:26:05","date_gmt":"2025-03-08T09:26:05","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3122"},"modified":"2025-03-08T15:41:54","modified_gmt":"2025-03-08T12:41:54","slug":"stitch-rna-new-method-for-duchenne-gene-therapies","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/stitch-rna-new-method-for-duchenne-gene-therapies\/","title":{"rendered":"Stitch RNA: Duchenne Gen Terapileri i\u00e7in Yeni Y\u00f6ntem"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Stitch RNA (StitchR) y\u00f6ntemi, Duchenne Musk\u00fcler Distrofi&#039;yi \u00f6nemli \u00f6l\u00e7\u00fcde tedavi edebilecek yeni ila\u00e7lar\u0131n geli\u015ftirilmesini h\u0131zland\u0131rabilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Kaslar\u0131n do\u011fru \u00e7al\u0131\u015fmas\u0131 i\u00e7in ihtiya\u00e7 duydu\u011fu belirli b\u00fcy\u00fck proteinleri olu\u015fturan DNA kodunun kayb\u0131, kaslar\u0131 zay\u0131flatan ve yok eden durumlara yol a\u00e7abilir. Gen tedavileri bu kodu tekrar kasa sokmaya \u00e7al\u0131\u015f\u0131r, ancak DNA kodu \u00e7ok uzun oldu\u011fu i\u00e7in bu zor olabilir. Ara\u015ft\u0131rmac\u0131lar kodu g\u00f6ndermenin yeni bir yolunu buldular. Art\u0131k kas h\u00fccrelerinin i\u00e7inde bir araya getirilecek par\u00e7alar halinde g\u00f6nderilecek. Bu, kas kaybeden veya zay\u0131flayan insanlara yard\u0131mc\u0131 olabilecek yeni gen tedavilerine yol a\u00e7abilir.<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u0130\u00e7indekiler<\/h2><nav><ul><li><a href=\"#the-dna-length-problem-in-gene-therapy\">Gen Terapisinde DNA Uzunlu\u011fu Sorunu<\/a><\/li><li><a href=\"#the-challenge-of-using-empty-shells-of-viruses\">Bo\u015f vir\u00fcs kabuklar\u0131n\u0131 kullanman\u0131n zorlu\u011fu<\/a><\/li><li><a href=\"#ribozymes-could-be-the-solution\">Ribozimler \u00e7\u00f6z\u00fcm olabilir<\/a><\/li><li><a href=\"#what-is-stitch-rna-and-how-does-it-work\">Stitch RNA Nedir ve Nas\u0131l \u00c7al\u0131\u015f\u0131r?<\/a><\/li><li><a href=\"#conclusion\">Sonu\u00e7<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<figure class=\"wp-block-image size-large\"><img decoding=\"async\" width=\"1024\" height=\"576\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/laboratory-clinical-trial-1024x576.jpg\" alt=\"Stitch RNA: Duchenne Gen Terapileri i\u00e7in Yeni Y\u00f6ntem\" class=\"wp-image-3124\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/laboratory-clinical-trial-1024x576.jpg 1024w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/laboratory-clinical-trial-300x169.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/laboratory-clinical-trial-768x432.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/laboratory-clinical-trial-1536x864.jpg 1536w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/laboratory-clinical-trial-18x10.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/laboratory-clinical-trial-747x420.jpg 747w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/laboratory-clinical-trial-150x84.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/laboratory-clinical-trial-696x392.jpg 696w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/laboratory-clinical-trial-1068x601.jpg 1068w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/laboratory-clinical-trial.jpg 1920w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><\/figure>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"the-dna-length-problem-in-gene-therapy\">Gen Terapisinde DNA Uzunlu\u011fu Sorunu<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Gen terapisi, kaslar\u0131n erimesine ve zay\u0131flamas\u0131na neden olan bir dizi hastal\u0131\u011f\u0131 tedavi etmek i\u00e7in yarat\u0131lm\u0131\u015ft\u0131r, ancak daha iyi \u00e7al\u0131\u015fmas\u0131 i\u00e7in hala daha fazla \u00e7al\u0131\u015fma yap\u0131lmaktad\u0131r. Kas h\u00fccrelerinin ihtiya\u00e7 duydu\u011fu proteinlerin baz\u0131lar\u0131 \u00e7ok b\u00fcy\u00fck oldu\u011fundan zor olabilir. Bir genin DNA kodu, v\u00fccuda bir proteinin nas\u0131l yap\u0131laca\u011f\u0131n\u0131 s\u00f6yler. Bu kodun uzunlu\u011fu, baz ad\u0131 verilen kimyasal bloklarla \u00f6l\u00e7\u00fcl\u00fcr.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">DNA baz kodunun daha b\u00fcy\u00fck proteinler i\u00e7in daha uzun olmas\u0131 gerekir. Duchenne kas distrofisi olan ki\u015filerde eksik olan distrofin proteininin kodlama dizisi 10.000 bazdan uzundur. \u00c7o\u011fu genin kodlama dizisi yaln\u0131zca yakla\u015f\u0131k 1.000 baz uzunlu\u011fundad\u0131r. \u00d6rne\u011fin, yeterli disferlin proteininiz yoksa, bacak ku\u015fa\u011f\u0131 kas distrofisi ve di\u011fer baz\u0131 kas distrofisi t\u00fcrlerine yakalanabilirsiniz. Kodunu olu\u015fturan baz zinciri 6.000&#039;den fazlad\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"the-challenge-of-using-empty-shells-of-viruses\">Bo\u015f vir\u00fcs kabuklar\u0131n\u0131 kullanman\u0131n zorlu\u011fu<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Ara\u015ft\u0131rmac\u0131lar, vir\u00fcslerin bo\u015f kabuklar\u0131n\u0131 kullanman\u0131n, v\u00fccudun h\u00fccrelerine kodlama dizileri sokmak i\u00e7in en etkili y\u00f6ntem oldu\u011funu ke\u015ffettiler. Kapsid olarak bilinen bu kabuklar, vir\u00fcsler taraf\u0131ndan genetik materyallerini kaps\u00fcllemek ve enfeksiyon i\u00e7in kom\u015fu h\u00fccrelere ta\u015f\u0131mak i\u00e7in kullan\u0131l\u0131r. Bunun yerine, tehlikeli viral kodlama olmadan laboratuvarlarda yapay olarak \u00fcretilebildikleri i\u00e7in gen terapisi sa\u011flamak i\u00e7in kullan\u0131labilirler. \u015eu anda, adeno-ili\u015fkili vir\u00fcs veya AAV olarak bilinen bir vir\u00fcs\u00fcn kapsidleri, gen terapisi i\u00e7in en etkili olanlard\u0131r. AAV, ba\u011f\u0131\u015f\u0131kl\u0131k sistemini harekete ge\u00e7irmeden \u00e7ok say\u0131da h\u00fccremize ula\u015fmada ustad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bununla birlikte, AAV kapsidi distrofin ve disferlinin kodlama dizilerini fiziksel olarak bar\u0131nd\u0131ramaz. Sadece vir\u00fcs\u00fcn kendi genetik kodu kadar uzun bir dizi i\u00e7erebilir, bu da 5.000 bazdan daha k\u0131sad\u0131r ve sadece 26 nanometre geni\u015fli\u011findedir (bunlardan bin tanesi bir insan sa\u00e7\u0131na s\u0131\u011far).<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"ribozymes-could-be-the-solution\">Ribozimler \u00e7\u00f6z\u00fcm olabilir<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Bu, yak\u0131n zamanda yap\u0131lan bir \u00e7al\u0131\u015fmayla yeni bir y\u00f6ntemle a\u015f\u0131ld\u0131. Ribozim olarak bilinen bir molek\u00fcl s\u0131n\u0131f\u0131 son birka\u00e7 on y\u0131lda tan\u0131mland\u0131 ve ara\u015ft\u0131r\u0131ld\u0131. Ribozimler, kendilerini ikiye b\u00f6lme yetene\u011fine sahip RNA dizileridir. Ribozimler, protein \u00fcretmek i\u00e7in kullan\u0131lan DNA kopyalar\u0131d\u0131r. Bunu, yeni kesilmi\u015f u\u00e7lara belirli kimyasal i\u015faretler b\u0131rakarak ger\u00e7ekle\u015ftirirler. New York ve Massachusetts&#039;ten yap\u0131lan ara\u015ft\u0131rmalara g\u00f6re, h\u00fccredeki belirli proteinler bu t\u00fcr belirte\u00e7leri tan\u0131yabilir ve u\u00e7lar\u0131 tekrar birbirine dikebilir. Bunun, bir gen terapisinin iki bile\u015feni gibi, iki ayr\u0131 RNA molek\u00fcl\u00fcn\u00fcn u\u00e7lar\u0131n\u0131 birle\u015ftirmek i\u00e7in kullan\u0131labilece\u011fine inan\u0131yorlard\u0131. <strong>Stitch RNA i\u00e7in y\u00f6nteme StitchR ad\u0131n\u0131 verdiler.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bilim insanlar\u0131 StitchR&#039;yi test etmek i\u00e7in iki RNA molek\u00fcl\u00fc olu\u015fturdular: biri disferlin kodlama dizisinin ilk k\u0131sm\u0131 ve ard\u0131ndan ikinci bir ribozim ve di\u011feri ise disferlin kodlama dizisinin kalan k\u0131sm\u0131 taraf\u0131ndan takip edilen bir ribozim. Bir fare modelinde uzuv ku\u015fa\u011f\u0131 kas dejenerasyonunu ba\u015flatmak i\u00e7in baz\u0131 AAV kapsidlerini bir molek\u00fclle ve di\u011fer AAV kapsidlerini di\u011fer molek\u00fclle y\u00fcklediler.<\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img decoding=\"async\" width=\"1024\" height=\"1024\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Stitch-RNA-Apples-1024x1024.jpg\" alt=\"Stitch RNA (StitchR) y\u00f6ntemi, Duchenne Musk\u00fcler Distrofi&#039;yi \u00f6nemli \u00f6l\u00e7\u00fcde tedavi edebilecek yeni ila\u00e7lar\u0131n geli\u015ftirilmesini h\u0131zland\u0131rabilir.\" class=\"wp-image-3139\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Stitch-RNA-Apples-1024x1024.jpg 1024w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Stitch-RNA-Apples-300x300.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Stitch-RNA-Apples-150x150.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Stitch-RNA-Apples-768x768.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Stitch-RNA-Apples-12x12.jpg 12w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Stitch-RNA-Apples-420x420.jpg 420w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Stitch-RNA-Apples-696x696.jpg 696w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Stitch-RNA-Apples-1068x1068.jpg 1068w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Stitch-RNA-Apples.jpg 1080w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><\/figure>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-stitch-rna-and-how-does-it-work\">Stitch RNA Nedir ve Nas\u0131l \u00c7al\u0131\u015f\u0131r?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ribozimler fare kas h\u00fccrelerine girdiklerinde aktif hale geliyor ve kar\u015f\u0131l\u0131k gelen molek\u00fclleri par\u00e7al\u0131yor.<\/strong> H\u00fccrenin onar\u0131m s\u00fcreci, kesiklerin b\u0131rakt\u0131\u011f\u0131 izleri belirleyerek iki par\u00e7ay\u0131 birbirine kayna\u015ft\u0131r\u0131yor ve disferlin proteininin farenin kaslar\u0131na geri d\u00f6nmesini sa\u011fl\u0131yor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu y\u00f6ntem ara\u015ft\u0131rmac\u0131lar taraf\u0131ndan distrofin kullan\u0131larak da test edildi.<\/strong> Distrofin kodlama dizisi, yar\u0131ya indirildikten sonra bile AAV kapsidi i\u00e7in hala \u00e7ok b\u00fcy\u00fck oldu\u011fundan, biraz daha k\u0131sa ama yine de i\u015flevsel bir protein i\u00e7in kodlama dizisi sa\u011flad\u0131lar. <strong>Y\u00f6ntem, Duchenne kas distrofisi fare modelinde bir dizi kas durumu \u00f6l\u00e7\u00fcm\u00fcn\u00fc iyile\u015ftirdi.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"conclusion\">Sonu\u00e7<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Duchenne kas distrofisi, disferlinopatiler ve di\u011fer bozukluklar i\u00e7in uzun y\u0131llard\u0131r prospektif tedaviler olarak geli\u015ftirilmi\u015f olsalar da, hedef h\u00fccre i\u00e7indeki b\u00fcy\u00fck genlerin kodlama dizilerini birle\u015ftiren y\u00f6ntemlerin \u00e7ok ba\u015far\u0131l\u0131 oldu\u011fu kan\u0131tlanmam\u0131\u015ft\u0131r. Fare modellerindeki sonu\u00e7lar \u00fcmit vericidir ve bu yeni yakla\u015f\u0131m baz\u0131 zorluklar\u0131n \u00fcstesinden gelmeye yard\u0131mc\u0131 olabilir. Bu ara\u015ft\u0131rman\u0131n yeni ila\u00e7larla sonu\u00e7lan\u0131p sonu\u00e7lanmayaca\u011f\u0131n\u0131 \u00f6\u011frenmek i\u00e7in daha fazla test yap\u0131lmas\u0131 gerekmektedir.<\/p>","protected":false},"excerpt":{"rendered":"<p>The Stitch RNA (StitchR) method could accelerate the development of new drugs that could significantly treat Duchenne Muscular Dystrophy. Loss of the DNA code that makes certain big proteins that muscles need to work right can lead to conditions that make muscles weak and waste away. Gene treatments try to get this code back into [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3125,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[61,283,284],"class_list":["post-3122","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-gene-therapy","tag-stitch-rna","tag-stitchr"],"subtitle":"Stitch RNA veya StitchR ad\u0131 verilen yeni bir y\u00f6ntemde, h\u00fccreler kas protein genlerini iki par\u00e7a halinde al\u0131yor ve bunlar\u0131 i\u015flevsel bir proteine d\u00f6n\u00fc\u015ft\u00fcr\u00fclen tek bir mRNA'ya birle\u015ftiriyorlar.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3122","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=3122"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3122\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3125"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3122"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3122"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3122"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}