{"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\/hi\/precision-biosciences-pbgene-dmd-restores-production-of-nearly-full-length-dystrophin-protein\/","title":{"rendered":"Precision BioSciences PBGENE-DMD \u0932\u0917\u092d\u0917 \u092a\u0942\u0930\u094d\u0923 \u0932\u0902\u092c\u093e\u0908 \u0935\u093e\u0932\u0947 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u093e \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u092c\u0939\u093e\u0932 \u0915\u0930\u0924\u093e \u0939\u0948"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Precision BioSciences \u0928\u0947 \u0906\u091c PBGENE-DMD \u0915\u0940 \u0930\u0923\u0928\u0940\u0924\u093f\u0915 \u092a\u094d\u0930\u093e\u0925\u092e\u093f\u0915\u0924\u093e \u0914\u0930 \u0917\u0924\u093f \u092c\u0922\u093c\u093e\u0928\u0947 \u0915\u0940 \u0918\u094b\u0937\u0923\u093e \u0915\u0940, \u091c\u094b \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 (DMD) \u0915\u0947 \u0932\u093f\u090f \u0915\u0902\u092a\u0928\u0940 \u0915\u093e \u092a\u094d\u0930\u0925\u092e \u0907\u0928-\u0915\u094d\u0932\u093e\u0938 \u0907\u0928 \u0935\u093f\u0935\u094b \u091c\u0940\u0928 \u0938\u0902\u092a\u093e\u0926\u0928 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923 \u0939\u0948\u0964<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u0935\u093f\u0937\u092f\u0938\u0942\u091a\u0940<\/h2><nav><ul><li><a href=\"#what-is-pbgene-dmd\">PBGENE-DMD \u0915\u094d\u092f\u093e \u0939\u0948?<\/a><\/li><li><a href=\"#how-the-pbgene-dmd-approach-works\">PBGENE-DMD \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923 \u0915\u0948\u0938\u0947 \u0915\u093e\u092e \u0915\u0930\u0924\u093e \u0939\u0948?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-pbgene-dmd\">PBGENE-DMD \u0915\u094d\u092f\u093e \u0939\u0948?<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>PBGENE-DMD, \u090f\u0915\u094d\u0938\u0949\u0928 45-55 \u0915\u0947 \u092c\u0940\u091a \u0938\u092c\u0938\u0947 \u0906\u092e &#039;\u0939\u0949\u091f \u0938\u094d\u092a\u0949\u091f&#039; \u0915\u094d\u0937\u0947\u0924\u094d\u0930 \u092e\u0947\u0902 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0938\u0947 \u092a\u094d\u0930\u092d\u093e\u0935\u093f\u0924 \u0921\u094d\u092f\u0942\u091a\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u092c\u0939\u0941\u092e\u0924 \u0915\u0947 \u0932\u093f\u090f \u090f\u0915 \u092a\u094d\u0930\u0925\u092e \u0936\u094d\u0930\u0947\u0923\u0940 \u092e\u0947\u0902 \u0907\u0928 \u0935\u093f\u0935\u094b \u091c\u0940\u0928 \u0938\u0902\u092a\u093e\u0926\u0928 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923 \u0939\u0948\u0964<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0905\u0902\u0924\u093f\u092e \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0909\u092e\u094d\u092e\u0940\u0926\u0935\u093e\u0930 PBGENE-DMD \u0938\u092e\u092f \u0915\u0947 \u0938\u093e\u0925 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0932\u093e\u092d \u092e\u0947\u0902 \u0938\u094d\u0925\u093e\u092f\u0940 \u0938\u0941\u0927\u093e\u0930 \u0915\u0947 \u0932\u093f\u090f \u0906\u0915\u0930\u094d\u0937\u0915 \u092a\u0942\u0930\u094d\u0935 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0921\u0947\u091f\u093e \u092a\u094d\u0930\u0926\u0930\u094d\u0936\u093f\u0924 \u0915\u0930\u0924\u093e \u0939\u0948\u0964<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>2025 \u092e\u0947\u0902 PBGENE-DMD \u0915\u0947 \u0932\u093f\u090f \u090f\u0915 \u091c\u093e\u0902\u091a\u093e\u0924\u094d\u092e\u0915 \u0928\u0908 \u0926\u0935\u093e (IND) \u0914\u0930\/\u092f\u093e \u0915\u094d\u0932\u093f\u0928\u093f\u0915\u0932 \u092a\u0930\u0940\u0915\u094d\u0937\u0923 \u0906\u0935\u0947\u0926\u0928 (CTA) \u092a\u094d\u0930\u0938\u094d\u0924\u0941\u0924 \u0915\u0930\u0928\u0947 \u0915\u093e \u0938\u091f\u0940\u0915 \u0932\u0915\u094d\u0937\u094d\u092f, 2026 \u092e\u0947\u0902 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0921\u0947\u091f\u093e \u092e\u093f\u0932\u0928\u0947 \u0915\u0940 \u0909\u092e\u094d\u092e\u0940\u0926 \u0939\u0948\u0964<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u0914\u0930 \u092a\u0922\u093c\u0947\u0902: <a href=\"https:\/\/dmdwarrior.com\/hi\/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\">\u0921\u094d\u092f\u0942\u091a\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 (\u0921\u0940\u090f\u092e\u0921\u0940) \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u0932\u093f\u090f PBGENE-DMD<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-the-pbgene-dmd-approach-works\">PBGENE-DMD \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923 \u0915\u0948\u0938\u0947 \u0915\u093e\u092e \u0915\u0930\u0924\u093e \u0939\u0948?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0935\u0930\u094d\u0924\u092e\u093e\u0928 \u092e\u0947\u0902 \u0915\u094b\u0908 \u0938\u094d\u0935\u0940\u0915\u0943\u0924 \u0909\u092a\u091a\u093e\u0930 \u0928\u0939\u0940\u0902 \u0939\u0948 \u091c\u094b \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0914\u0930 \u091f\u093f\u0915\u093e\u090a \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0938\u0941\u0927\u093e\u0930 \u0932\u093e \u0938\u0915\u0924\u093e \u0939\u0948\u0964 PBGENE-DMD \u0926\u094b \u092a\u0942\u0930\u0915 ARCUS \u0928\u094d\u092f\u0942\u0915\u094d\u0932\u093f\u0910\u0938\u0947\u0938 \u0915\u093e \u0909\u092a\u092f\u094b\u0917 \u0915\u0930\u0924\u093e \u0939\u0948, \u091c\u094b \u090f\u0915 \u090f\u0915\u0932 AAV \u092e\u0947\u0902 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u091c\u0940\u0928 \u0915\u0947 \u090f\u0915\u094d\u0938\u0949\u0928 45-55 \u0915\u094b \u0928\u093f\u0915\u093e\u0932\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0935\u093f\u0924\u0930\u093f\u0924 \u0915\u093f\u090f \u091c\u093e\u0924\u0947 \u0939\u0948\u0902, \u091c\u093f\u0938\u0915\u093e \u0909\u0926\u094d\u0926\u0947\u0936\u094d\u092f \u0936\u0930\u0940\u0930 \u0915\u0947 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u0947 \u092a\u094d\u0930\u093e\u0915\u0943\u0924\u093f\u0915 \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u0915\u094b \u092c\u0939\u093e\u0932 \u0915\u0930\u0928\u093e \u0939\u0948\u0964<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">ASGCT \u092e\u0947\u0902 \u092a\u094d\u0930\u0938\u094d\u0924\u0941\u0924 \u0915\u093f\u090f \u091c\u093e \u0930\u0939\u0947 \u092a\u094d\u0930\u0940\u0915\u094d\u0932\u093f\u0928\u093f\u0915\u0932 \u0921\u0947\u091f\u093e \u092e\u0947\u0902, PBGENE-DMD \u0928\u0947 \u092e\u093e\u0928\u0935\u0915\u0943\u0924 DMD \u092e\u093e\u0909\u0938 \u092e\u0949\u0921\u0932 \u092e\u0947\u0902 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0914\u0930 \u091f\u093f\u0915\u093e\u090a \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0938\u0941\u0927\u093e\u0930 \u092a\u094d\u0930\u0926\u0930\u094d\u0936\u093f\u0924 \u0915\u093f\u092f\u093e\u0964 AAV \u0921\u093f\u0932\u0940\u0935\u0930\u0940 \u0915\u0947 \u092c\u093e\u0926, PBGENE-DMD \u0928\u0947 \u0939\u0943\u0926\u092f \u0914\u0930 \u0915\u0902\u0915\u093e\u0932 \u0915\u0940 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0938\u0939\u093f\u0924 \u0915\u0908 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u0935\u094d\u092f\u093e\u092a\u0915 \u0930\u0942\u092a \u0938\u0947 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u093e \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u0915\u0930\u0928\u0947 \u0915\u0940 \u0936\u0930\u0940\u0930 \u0915\u0940 \u0915\u094d\u0937\u092e\u0924\u093e \u0915\u094b \u092c\u0939\u093e\u0932 \u0915\u093f\u092f\u093e\u0964 9 \u092e\u0939\u0940\u0928\u094b\u0902 \u0915\u0947 \u0926\u094c\u0930\u093e\u0928, PBGENE-DMD \u0915\u0947 \u0938\u093e\u0925 \u0907\u0932\u093e\u091c \u0915\u093f\u090f \u0917\u090f \u091a\u0942\u0939\u094b\u0902 \u0928\u0947 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0905\u092d\u093f\u0935\u094d\u092f\u0915\u094d\u0924\u093f \u092e\u0947\u0902 \u0935\u0943\u0926\u094d\u0927\u093f \u0926\u093f\u0916\u093e\u0908, \u091c\u093f\u0938\u0915\u0947 \u092a\u0930\u093f\u0923\u093e\u092e\u0938\u094d\u0935\u0930\u0942\u092a \u092a\u0930\u094d\u092f\u093e\u092a\u094d\u0924 \u0914\u0930 \u0928\u093f\u0930\u0902\u0924\u0930 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0938\u0941\u0927\u093e\u0930 \u0939\u0941\u0906\u0964 \u0907\u0938\u0915\u0947 \u0905\u0932\u093e\u0935\u093e, \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0909\u092a\u0917\u094d\u0930\u0939 \u0938\u094d\u091f\u0947\u092e \u0915\u094b\u0936\u093f\u0915\u093e\u0913\u0902 \u092e\u0947\u0902 PBGENE-DMD-\u0938\u0902\u092a\u093e\u0926\u093f\u0924 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 mRNA \u092a\u094d\u0930\u0924\u093f\u0932\u0947\u0916, \u091c\u094b \u0928\u0908 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0915\u094b\u0936\u093f\u0915\u093e\u0913\u0902 \u0915\u0947 \u0932\u093f\u090f \u092a\u0942\u0930\u094d\u0935\u091c \u0915\u094b\u0936\u093f\u0915\u093e\u090f\u0901 \u0939\u0948\u0902, \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0938\u094d\u0925\u093e\u092f\u093f\u0924\u094d\u0935 \u0915\u0940 \u0915\u094d\u0937\u092e\u0924\u093e \u0915\u093e \u0938\u092e\u0930\u094d\u0925\u0928 \u0915\u0930\u0924\u0940 \u0939\u0948\u0902\u0964<\/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"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/3860","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/comments?post=3860"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/3860\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media\/3863"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media?parent=3860"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/categories?post=3860"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/tags?post=3860"}],"curies":[{"name":"\u0921\u092c\u094d\u0932\u094d\u092f\u0942\u092a\u0940","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}