{"id":3561,"date":"2025-04-08T11:41:34","date_gmt":"2025-04-08T08:41:34","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3561"},"modified":"2025-04-08T11:41:36","modified_gmt":"2025-04-08T08:41:36","slug":"the-immune-system-may-be-decreasing-the-effectiveness-of-gene-therapy-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/the-immune-system-may-be-decreasing-the-effectiveness-of-gene-therapy-for-duchenne-muscular-dystrophy\/","title":{"rendered":"Ba\u011f\u0131\u015f\u0131kl\u0131k Sistemi Duchenne Musk\u00fcler Distrofisi \u0130\u00e7in Gen Terapisinin Etkinli\u011fini Azalt\u0131yor Olabilir"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Duchenne Musk\u00fcler Distrofi (DMD) hastalar\u0131nda distrofin eksikli\u011fi oldu\u011fundan, gen tedavisi yoluyla tekrar verilmesi, ba\u011f\u0131\u015f\u0131kl\u0131k sisteminin bunu bir antijen veya yabanc\u0131 protein olarak alg\u0131lamas\u0131na neden olabilir; bu da onu \u00fcreten kas\u0131 tahrip eden bir reaksiyona yol a\u00e7abilir ve tedavinin etkinli\u011fini tehlikeye atabilir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Ba\u011f\u0131\u015f\u0131kl\u0131k Sistemi Distrofin \u00dcretimini Engelleyebilir<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Sonu\u00e7lar, eksik protein distrofini geri kazand\u0131rmay\u0131 ama\u00e7layan Elevidys (delandistrogene moxeparvovec) gibi gen terapisinin, v\u00fccudun ba\u011f\u0131\u015f\u0131kl\u0131k sistemi taraf\u0131ndan engellenebilece\u011fini ve muhtemelen herhangi bir terap\u00f6tik etki g\u00f6stermesini engelleyebilece\u011fini g\u00f6stermektedir.<a href=\"https:\/\/dmdwarrior.com\/tr\/frequently-asked-questions-about-elevidys-used-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Elevidys Hakk\u0131nda S\u0131k\u00e7a Sorulan Sorular<\/a>)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7o\u011funlukla erkek \u00e7ocuklar\u0131n\u0131 etkileyen DMD, zay\u0131flat\u0131c\u0131 bir genetik bozukluktur. Erken \u00f6l\u00fcme, erken ergenlikte hareket kayb\u0131na ve amans\u0131z kas par\u00e7alanmas\u0131na neden olur. \u015eu anda bir tedavi olmad\u0131\u011f\u0131ndan, gen terapisi ilerleme i\u00e7in en umut verici yollardan biri olarak g\u00f6r\u00fclm\u00fc\u015ft\u00fcr.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Distrofin ad\u0131 verilen hayati bir kas proteinini geri kazand\u0131ran tedavinin, bu hastal\u0131ktan etkilenen ki\u015filerin hayatlar\u0131n\u0131 de\u011fi\u015ftirece\u011fi y\u00f6n\u00fcndeki g\u00fc\u00e7l\u00fc umutlarla, Elevidys, 2024 y\u0131l\u0131nda ABD G\u0131da ve \u0130la\u00e7 Dairesi&#039;nden geni\u015fletilmi\u015f onay ald\u0131. Faz 3 EMBARK denemesinin en son sonu\u00e7lar\u0131, daha kasvetli bir tablo \u00e7iziyor.<a href=\"https:\/\/dmdwarrior.com\/tr\/roche-announces-new-results-from-the-embark-study-of-elevidys-in-outpatients-with-duchenne-muscular-dystrophy-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">EMBARK Elevidys \u00c7al\u0131\u015fmas\u0131<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">DMD Gen Terapilerinde Ba\u011f\u0131\u015f\u0131kl\u0131k Sistemi \u0130kilemi<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Sonu\u00e7lar tatmin edici de\u011fildi. Portsmouth \u00dcniversitesi T\u0131p, Eczac\u0131l\u0131k ve Biyolojik Bilimler Fak\u00fcltesi&#039;nden GT makalesinin k\u0131demli yazarlar\u0131ndan Profes\u00f6r Darek Gorecki&#039;ye g\u00f6re, ba\u011f\u0131\u015f\u0131kl\u0131k sisteminin yeniden tan\u0131t\u0131lan proteine verdi\u011fi yan\u0131t, tedavinin kas fonksiyonunda g\u00f6zle g\u00f6r\u00fcl\u00fcr bir iyile\u015fme sa\u011flamamas\u0131n\u0131n nedeni olabilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu tip reaksiyon baz\u0131 hastalarda \u00f6zellikle yayg\u0131n olabilir. Kronik kas iltihab\u0131, &quot;geri d\u00f6n\u00fc\u015f\u00fcml\u00fc lifler&quot; olarak adland\u0131r\u0131lanlar arac\u0131l\u0131\u011f\u0131yla eser miktarda distrofin maruziyeti veya erken geli\u015fim s\u0131ras\u0131nda normalde silinmeyen reaktif ba\u011f\u0131\u015f\u0131kl\u0131k h\u00fccrelerinin varl\u0131\u011f\u0131 risk fakt\u00f6rleridir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fma ayr\u0131ca DMD&#039;nin ne kadar karma\u015f\u0131k oldu\u011funu vurguluyor. Herhangi bir etkili tedavi muhtemelen distrofinin kas\u0131n \u00f6tesine ge\u00e7erek beyin, kemikler ve di\u011fer dokular\u0131 da kapsayan sistemik etkilerini hesaba katmal\u0131d\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Yine de bilim insanlar\u0131 umutlu. Profes\u00f6r Gorecki, &quot;DMD i\u00e7in gen terapisi hen\u00fcz bitmedi.&quot; dedi. &quot;Bu, her hasta i\u00e7in tedavileri \u00f6zelle\u015ftirmek ve do\u011frudan imm\u00fcnojeniteyi ele almak i\u00e7in daha ileri gitmemiz gerekti\u011fini hat\u0131rlat\u0131yor.&quot;<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fma, laboratuvardan hayat de\u011fi\u015ftiren t\u0131bba giden yolculu\u011fun nadiren basit oldu\u011fu ve en geli\u015fmi\u015f genetik tedavilerin bile ki\u015fiselle\u015ftirilmi\u015f, multidisipliner bir yakla\u015f\u0131m gerektirece\u011fi konusundaki giderek artan bilgiye katk\u0131da bulunuyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu ara\u015ft\u0131rma ve tezi g\u00f6z \u00f6n\u00fcne ald\u0131\u011f\u0131m\u0131zda, Elevidys gen tedavisinin neden $3 milyon dolara sat\u0131ld\u0131\u011f\u0131n\u0131 sormam\u0131z gerekmez mi?<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/clinical-trials-review-of-elevidys-gene-therapy-is-elevidys-cost-worthy\/\">Elevidys Gen Terapisinin Klinik Ara\u015ft\u0131rma \u0130ncelemesi: Elevidys Maliyetine De\u011fer mi?<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Since patients with Duchenne Muscular Dystrophy (DMD) lack dystrophin, reintroducing it through gene therapy may cause the immune system to mistake it for an antigen, or foreign protein, which could lead to a reaction that destroys the muscle that produces it and jeopardize the effectiveness of the treatment. The Immune System May Inhibit Dystrophin Production [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3567,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[33,61,349,203,65],"class_list":["post-3561","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-elevidys","tag-gene-therapy","tag-immune-system","tag-roche","tag-sarepta"],"subtitle":"Geni\u015f \u00e7apl\u0131 bir klinik ara\u015ft\u0131rman\u0131n kayda de\u011fer bir fayda g\u00f6stermemesi \u00fczerine, Gene Therapy dergisinde yay\u0131nlanan son bir makale, Duchenne kas distrofisi (DMD) i\u00e7in gen terapisinin etkinli\u011fi konusunda ba\u011f\u0131\u015f\u0131kl\u0131k sistemiyle ilgili \u00f6nemli sorular\u0131 g\u00fcndeme getiriyor.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3561","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=3561"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3561\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3567"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3561"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3561"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3561"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}