{"id":3860,"date":"2025-05-15T13:01:14","date_gmt":"2025-05-15T10:01:14","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3860"},"modified":"2025-05-15T13:01:16","modified_gmt":"2025-05-15T10:01:16","slug":"precision-biosciences-pbgene-dmd-restores-production-of-nearly-full-length-dystrophin-protein","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/precision-biosciences-pbgene-dmd-restores-production-of-nearly-full-length-dystrophin-protein\/","title":{"rendered":"Precision BioSciences PBGENE-DMD, Neredeyse Tam Uzunlukta Distrofin Proteininin \u00dcretimini Geri Kazand\u0131r\u0131r"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Precision BioSciences, bug\u00fcn Duchenne Musk\u00fcler Distrofi (DMD) i\u00e7in \u015eirketin s\u0131n\u0131f\u0131n\u0131n ilk in vivo gen d\u00fczenleme yakla\u015f\u0131m\u0131 olan PBGENE-DMD&#039;nin stratejik \u00f6nceliklendirilmesini ve h\u0131zland\u0131r\u0131lmas\u0131n\u0131 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=\"#what-is-pbgene-dmd\">PBGENE-DMD nedir?<\/a><\/li><li><a href=\"#how-the-pbgene-dmd-approach-works\">PBGENE-DMD Yakla\u015f\u0131m\u0131 Nas\u0131l \u00c7al\u0131\u015f\u0131r?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-pbgene-dmd\">PBGENE-DMD nedir?<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>PBGENE-DMD, 45-55. eksonlar aras\u0131ndaki en yayg\u0131n &#039;s\u0131cak nokta&#039; b\u00f6lgesindeki distrofin mutasyonlar\u0131ndan etkilenen Duchenne Musk\u00fcler Distrofi hastalar\u0131n\u0131n \u00e7o\u011funlu\u011fu i\u00e7in s\u0131n\u0131f\u0131nda ilk olan bir in vivo gen d\u00fczenleme yakla\u015f\u0131m\u0131d\u0131r.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Son klinik aday PBGENE-DMD, zaman i\u00e7inde fonksiyonel fayday\u0131 kal\u0131c\u0131 olarak iyile\u015ftirmek i\u00e7in ikna edici klinik \u00f6ncesi veriler g\u00f6stermektedir.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>2025 y\u0131l\u0131nda PBGENE-DMD i\u00e7in Ara\u015ft\u0131rma Ama\u00e7l\u0131 Yeni \u0130la\u00e7 (IND) ve\/veya Klinik Ara\u015ft\u0131rma Ba\u015fvurusu (CTA) sunulmas\u0131 ve 2026 y\u0131l\u0131nda klinik verilerin beklenmesi hedefleniyor.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Devam\u0131n\u0131 oku: <a href=\"https:\/\/dmdwarrior.com\/tr\/precision-biosciences-presents-preclinical-efficacy-and-durability-data-on-pbgene-dmd-for-the-treatment-of-duchenne-muscular-dystrophy-dmd-at-the-2025-muscular-dystrophy-association-mda-clinical\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne Musk\u00fcler Distrofi (DMD) Tedavisinde PBGENE-DMD<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-the-pbgene-dmd-approach-works\">PBGENE-DMD Yakla\u015f\u0131m\u0131 Nas\u0131l \u00c7al\u0131\u015f\u0131r?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eu anda, \u00f6nemli ve kal\u0131c\u0131 fonksiyonel kas iyile\u015ftirmeleri sa\u011flayabilecek onayl\u0131 bir tedavi bulunmamaktad\u0131r. PBGENE-DMD, v\u00fccudun fonksiyonel distrofin proteininin do\u011fal \u00fcretimini yeniden sa\u011flamay\u0131 ama\u00e7layan distrofin geninin 45-55. eksonlar\u0131n\u0131 \u00e7\u0131karmak i\u00e7in tek bir AAV&#039;de iletilen iki tamamlay\u0131c\u0131 ARCUS n\u00fckleaz\u0131n\u0131 kullan\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">ASGCT&#039;de sunulan klinik \u00f6ncesi verilerde, PBGENE-DMD, insanla\u015ft\u0131r\u0131lm\u0131\u015f bir DMD fare modelinde \u00f6nemli ve kal\u0131c\u0131 i\u015flevsel iyile\u015fme g\u00f6sterdi. AAV verilmesinin ard\u0131ndan, PBGENE-DMD v\u00fccudun kardiyak ve iskelet kaslar\u0131 da dahil olmak \u00fczere bir\u00e7ok kasta i\u015flevsel bir distrofin proteini \u00fcretme yetene\u011fini geri kazand\u0131rd\u0131. 9 ay boyunca, PBGENE-DMD ile tedavi edilen fareler, \u00f6nemli ve s\u00fcrd\u00fcr\u00fclebilir i\u015flevsel kas iyile\u015fmesiyle sonu\u00e7lanan artan distrofin protein ekspresyonu g\u00f6sterdi. Ek olarak, yeni kas h\u00fccreleri i\u00e7in progenit\u00f6r h\u00fccreler olan kas uydu k\u00f6k h\u00fccrelerindeki PBGENE-DMD taraf\u0131ndan d\u00fczenlenen distrofin mRNA transkripti, uzun vadeli dayan\u0131kl\u0131l\u0131k potansiyelini destekler.<\/p>","protected":false},"excerpt":{"rendered":"<p>Precision BioSciences, today announced the strategic prioritization and acceleration of PBGENE-DMD, the Company\u2019s first-in-class in vivo gene editing approach for Duchenne Muscular Dystrophy (DMD). What is PBGENE-DMD? Read more: PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy (DMD) How the PBGENE-DMD Approach Works? There are currently no approved therapies that can drive significant and durable [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3863,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,72,314,313],"class_list":["post-3860","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":"PBGENE-DMD, kas rejenerasyonu ve i\u015flevinde \u00f6m\u00fcr boyu faydalar sa\u011flayabilecek tek seferlik, kal\u0131c\u0131 bir m\u00fcdahale sa\u011flama potansiyeline sahiptir. Klinik \u00f6ncesi modeller, PBGENE-DMD'nin insan v\u00fccudunun i\u015flevsel, neredeyse tam uzunlukta bir distrofin proteini \u00fcretimini geri kazand\u0131rarak maksimum kuvvet \u00e7\u0131kt\u0131s\u0131nda \u00f6nemli ve s\u00fcrekli bir iyile\u015fme sa\u011flad\u0131\u011f\u0131n\u0131 g\u00f6stermi\u015ftir.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3860","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=3860"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3860\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3863"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3860"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3860"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3860"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}