{"id":4000,"date":"2025-06-25T14:58:50","date_gmt":"2025-06-25T11:58:50","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4000"},"modified":"2025-06-25T15:02:05","modified_gmt":"2025-06-25T12:02:05","slug":"precision-biosciences-receives-fda-rare-pediatric-disease-designation-for-pbgene-dmd-for-the-treatment-of-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/precision-biosciences-receives-fda-rare-pediatric-disease-designation-for-pbgene-dmd-for-the-treatment-of-duchenne-muscular-dystrophy\/","title":{"rendered":"Precision BioSciences, Duchenne Musk\u00fcler Distrofi Tedavisi \u0130\u00e7in PBGENE-DMD \u0130\u00e7in FDA Nadir Pediatrik Hastal\u0131k Tan\u0131m\u0131n\u0131 Ald\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Y\u00fcksek kar\u015f\u0131lanmam\u0131\u015f ihtiyaca sahip hastal\u0131klar i\u00e7in in vivo gen d\u00fczenleme terapileri geli\u015ftirmek amac\u0131yla yenilik\u00e7i tescilli ARCUS\u00ae platformunu kullanan klinik a\u015famada gen d\u00fczenleme \u015firketi Precision BioSciences, bug\u00fcn ABD G\u0131da ve \u0130la\u00e7 Dairesi&#039;nin (FDA) PBGENE-DMD&#039;ye Duchenne kas distrofisi (DMD) tedavisi i\u00e7in Nadir Pediatrik Hastal\u0131k Tan\u0131m\u0131 verdi\u011fini duyurdu.<\/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=\"#near-full-length-dystrophin-protein\">Neredeyse Tam Uzunlukta Distrofin Proteini<\/a><\/li><li><a href=\"#what-is-pbgene-dmd\">PBGENE-DMD nedir?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"near-full-length-dystrophin-protein\">Neredeyse Tam Uzunlukta Distrofin Proteini<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cNadir Pediatrik Hastal\u0131k Tan\u0131m\u0131&#039;n\u0131n al\u0131nmas\u0131, DMD ile ya\u015fayan erkek \u00e7ocuklar i\u00e7in yeni tedavi se\u00e7eneklerinin gerekli oldu\u011fu \u00f6nemli kar\u015f\u0131lanmam\u0131\u015f ihtiyac\u0131 vurgulamaktad\u0131r,\u201d dedi Precision BioSciences&#039;de Ba\u015f Geli\u015ftirme ve \u0130\u015fletme Sorumlusu Cindy Atwell. \u201cDistrofin geninin 45-55 ekzonlar\u0131n\u0131 \u00e7\u0131karmak i\u00e7in ARCUS gen d\u00fczenleme platformumuzun farkl\u0131la\u015ft\u0131r\u0131lm\u0131\u015f yeteneklerinden yararlanan s\u0131n\u0131f\u0131n\u0131n ilk PBGENE-DMD program\u0131m\u0131z, DMD&#039;li 60%&#039;ye kadar hasta i\u00e7in zaman i\u00e7inde anlaml\u0131 iyile\u015fme ve kal\u0131c\u0131 i\u015flevsel fayda sa\u011flama potansiyeline sahiptir. Yakla\u015f\u0131m\u0131m\u0131z, bir hastan\u0131n kendi DNA dizisini kal\u0131c\u0131 olarak d\u00fczenlemek ve bunun sonucunda insanlarda i\u015flevsel oldu\u011fu bilinen, neredeyse tam uzunlukta do\u011fal olarak \u00fcretilen distrofin proteini elde etmek i\u00e7in tasarlanm\u0131\u015ft\u0131r. \u015eu anda devam eden son IND etkinle\u015ftirici toksikoloji \u00e7al\u0131\u015fmalar\u0131n\u0131 tamamlamay\u0131 ve 2026&#039;da beklenen ilk klinik verileri \u00fcretmeyi d\u00f6rt g\u00f6zle bekliyoruz.\u201d<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-pbgene-dmd\">PBGENE-DMD nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">PBGENE-DMD, Precision&#039;\u0131n DMD tedavisi i\u00e7in geli\u015ftirme program\u0131d\u0131r. Yakla\u015f\u0131m, fonksiyonel sonu\u00e7lar\u0131 iyile\u015ftirmek i\u00e7in v\u00fccutta neredeyse tam uzunlukta distrofin proteinini geri kazand\u0131rmak amac\u0131yla distrofin geninin 45-55. ekzonlar\u0131n\u0131 \u00e7\u0131karmak i\u00e7in tek bir AAV&#039;de tek seferlik uygulama yoluyla verilen iki tamamlay\u0131c\u0131 ARCUS n\u00fckleaz\u0131n\u0131 kullan\u0131r. PBGENE-DMD, DMD hasta pop\u00fclasyonunun 60%&#039;sinden fazlas\u0131n\u0131 ele almay\u0131 ama\u00e7lamaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Klinik \u00f6ncesi \u00e7al\u0131\u015fmalarda, PBGENE-DMD, DMD&#039;nin ilerlemesinde rol oynayan temel kas tiplerini hedefleme yetene\u011fini g\u00f6sterdi ve insanla\u015ft\u0131r\u0131lm\u0131\u015f bir DMD fare modelinde \u00f6nemli, kal\u0131c\u0131 i\u015flevsel iyile\u015ftirmeler \u00fcretti. PBGENE-DMD, v\u00fccudun kardiyak doku ve \u00e7e\u015fitli temel iskelet kas gruplar\u0131 dahil olmak \u00fczere birden fazla kasta neredeyse tam uzunlukta i\u015flevsel bir distrofin proteini \u00fcretme yetene\u011fini geri kazand\u0131rd\u0131. Ek olarak, PBGENE-DMD, uzun vadeli dayan\u0131kl\u0131l\u0131k ve s\u00fcrekli i\u015flevsel iyile\u015ftirme i\u00e7in kritik oldu\u011funa inan\u0131lan uydu kas k\u00f6k h\u00fccrelerini d\u00fczenledi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha fazla bilgi edin:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/precision-biosciences-pbgene-dmd-restores-production-of-nearly-full-length-dystrophin-protein\/\">Precision BioSciences PBGENE-DMD, Neredeyse Tam Uzunlukta Distrofin Proteininin \u00dcretimini Geri Kazand\u0131r\u0131r<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Precision BioSciences, a clinical stage gene editing company utilizing its novel proprietary ARCUS\u00ae platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD). Near Full-Length [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4001,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,72,314,313],"class_list":["post-4000","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-full-length-dystrophin","tag-gene-editing","tag-pbgene-dmd","tag-precision-biosciences"],"subtitle":"Precision BioSciences, bug\u00fcn FDA'n\u0131n, PBGENE-DMD'ye Duchenne Musk\u00fcler Distrofi (DMD) tedavisi i\u00e7in Nadir Pediatrik Hastal\u0131k Tan\u0131m\u0131 verdi\u011fini duyurdu.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4000","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=4000"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4000\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4001"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4000"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4000"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4000"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}