{"id":3288,"date":"2025-03-19T16:03:20","date_gmt":"2025-03-19T13:03:20","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3288"},"modified":"2025-05-15T14:31:04","modified_gmt":"2025-05-15T11:31:04","slug":"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","status":"publish","type":"post","link":"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\/","title":{"rendered":"Precision BioSciences, 2025 Musk\u00fcler Distrofi Derne\u011fi (MDA) Klinik ve Bilimsel Konferans\u0131nda Duchenne Musk\u00fcler Distrofi (DMD) Tedavisi i\u00e7in PBGENE-DMD&#039;nin Klinik \u00d6ncesi Etkinlik ve Dayan\u0131kl\u0131l\u0131k Verilerini Sunuyor"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Kas uydu k\u00f6k h\u00fccrelerini d\u00fczenlemeyi i\u00e7eren bu distrofin gen d\u00fczeltme yakla\u015f\u0131m\u0131, sentetik yakla\u015f\u0131mlara k\u0131yasla dayan\u0131kl\u0131l\u0131\u011f\u0131 ve i\u015flevsel sonu\u00e7lar\u0131 potansiyel olarak art\u0131r\u0131r. DMD vakalar\u0131n\u0131n 60%&#039;sine kadar\u0131n\u0131n 45 ve 55 ekzonlar\u0131 aras\u0131ndaki defektlerden kaynaklanmas\u0131 nedeniyle, bu yakla\u015f\u0131m ekzon atlay\u0131c\u0131lar\u0131ndan daha \u00e7ok DMD hastalar\u0131n\u0131n \u00e7o\u011funlu\u011fu i\u00e7in uygulanabilirdir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fman\u0131n temel bulgular\u0131 \u015funlard\u0131r:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Fonksiyonel distrofin protein \u00fcretimi:<\/strong> PBGENE-DMD, kalp, diyafram ve iskelet kaslar\u0131 da dahil olmak \u00fczere birden fazla kasta fonksiyonel distrofin proteini \u00fcretme yetene\u011fini, terap\u00f6tik fayda sa\u011flamas\u0131 beklenen seviyelerde geri kazand\u0131rd\u0131.<\/li>\n\n\n\n<li><strong>Geli\u015fmi\u015f Kas Dayan\u0131kl\u0131l\u0131\u011f\u0131:<\/strong> Tedavi edilen fareler, tedavi edilmeyen hastal\u0131kl\u0131 muadillerine k\u0131yasla, eksantrik yaralanmaya kar\u015f\u0131 diren\u00e7te, kas dayan\u0131kl\u0131l\u0131\u011f\u0131n\u0131n artt\u0131\u011f\u0131n\u0131 g\u00f6steren bir g\u00f6sterge olan 66% iyile\u015fme g\u00f6sterdi.<\/li>\n\n\n\n<li><strong>Uzun Vadeli Fonksiyonel \u0130yile\u015fme:<\/strong> PBGENE-DMD ile tedavi edilen farelerde, kritik bir fonksiyonel \u00f6l\u00e7\u00fct olan maksimum kuvvet \u00e7\u0131k\u0131\u015f\u0131 (MFO), sa\u011fl\u0131kl\u0131 kontrol farelerindeki MFO&#039;nun 93%&#039;sine kadar ula\u015ft\u0131 ve PBGENE-DMD ile tedavi edilen farelerde 3 ila 6 ay aras\u0131nda iyile\u015fme g\u00f6zlendi.<\/li>\n\n\n\n<li><strong>Kal\u0131c\u0131 Sonu\u00e7lar:<\/strong> Kas uydu k\u00f6k h\u00fccreleri i\u00e7in bir belirte\u00e7 olan PAX7+ h\u00fccrelerinde PBGENE-DMD taraf\u0131ndan d\u00fczenlenen distrofin mRNA transkripti tespit edildi ve bu, standart gen terapisi yakla\u015f\u0131mlar\u0131na k\u0131yasla kal\u0131c\u0131 terap\u00f6tik etkilere sahip olma potansiyelini d\u00fc\u015f\u00fcnd\u00fcrmektedir.<\/li>\n<\/ul>","protected":false},"excerpt":{"rendered":"<p>This dystrophin gene correction approach which involves editing muscle satellite stem cells potentially enhances durability and functional outcomes compared to synthetic approaches. Since up to 60% of DMD cases are caused by defects between exons 45 and 55, this approach is more broadly applicable for the majority of DMD patients than exon skippers. Key findings [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3289,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,72,297,314,313],"class_list":["post-3288","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-full-length-dystrophin","tag-gene-editing","tag-muscular-dystrophy-association","tag-pbgene-dmd","tag-precision-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3288","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=3288"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3288\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3289"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3288"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3288"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3288"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}