{"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\/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\/","title":{"rendered":"Precision BioSciences \u0928\u0947 2025 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u090f\u0938\u094b\u0938\u093f\u090f\u0936\u0928 (MDA) \u0915\u094d\u0932\u093f\u0928\u093f\u0915\u0932 \u0914\u0930 \u0935\u0948\u091c\u094d\u091e\u093e\u0928\u093f\u0915 \u0938\u092e\u094d\u092e\u0947\u0932\u0928 \u092e\u0947\u0902 \u0921\u094d\u092f\u0942\u091a\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 (DMD) \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u0932\u093f\u090f PBGENE-DMD \u092a\u0930 \u092a\u094d\u0930\u0940\u0915\u094d\u0932\u093f\u0928\u093f\u0915\u0932 \u092a\u094d\u0930\u092d\u093e\u0935\u0915\u093e\u0930\u093f\u0924\u093e \u0914\u0930 \u0938\u094d\u0925\u093e\u092f\u093f\u0924\u094d\u0935 \u0921\u0947\u091f\u093e \u092a\u094d\u0930\u0938\u094d\u0924\u0941\u0924 \u0915\u093f\u092f\u093e"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u092f\u0939 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u091c\u0940\u0928 \u0938\u0941\u0927\u093e\u0930 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923 \u091c\u093f\u0938\u092e\u0947\u0902 \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 \u0915\u094b \u0938\u0902\u092a\u093e\u0926\u093f\u0924 \u0915\u0930\u0928\u093e \u0936\u093e\u092e\u093f\u0932 \u0939\u0948, \u0938\u0902\u092d\u093e\u0935\u093f\u0924 \u0930\u0942\u092a \u0938\u0947 \u0938\u093f\u0902\u0925\u0947\u091f\u093f\u0915 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923\u094b\u0902 \u0915\u0940 \u0924\u0941\u0932\u0928\u093e \u092e\u0947\u0902 \u0938\u094d\u0925\u093e\u092f\u093f\u0924\u094d\u0935 \u0914\u0930 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u092a\u0930\u093f\u0923\u093e\u092e\u094b\u0902 \u0915\u094b \u092c\u0922\u093c\u093e\u0924\u093e \u0939\u0948\u0964 \u091a\u0942\u0902\u0915\u093f DMD \u0915\u0947 60% \u0924\u0915 \u0915\u0947 \u092e\u093e\u092e\u0932\u0947 \u090f\u0915\u094d\u0938\u0949\u0928 45 \u0914\u0930 55 \u0915\u0947 \u092c\u0940\u091a \u0926\u094b\u0937\u094b\u0902 \u0915\u0947 \u0915\u093e\u0930\u0923 \u0939\u094b\u0924\u0947 \u0939\u0948\u0902, \u0907\u0938\u0932\u093f\u090f \u092f\u0939 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923 \u090f\u0915\u094d\u0938\u0949\u0928 \u0938\u094d\u0915\u093f\u092a\u0930\u094d\u0938 \u0915\u0940 \u0924\u0941\u0932\u0928\u093e \u092e\u0947\u0902 DMD \u0915\u0947 \u0905\u0927\u093f\u0915\u093e\u0902\u0936 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u0905\u0927\u093f\u0915 \u0935\u094d\u092f\u093e\u092a\u0915 \u0930\u0942\u092a \u0938\u0947 \u0932\u093e\u0917\u0942 \u0939\u094b\u0924\u093e \u0939\u0948\u0964<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0905\u0927\u094d\u092f\u092f\u0928 \u0915\u0947 \u092a\u094d\u0930\u092e\u0941\u0916 \u0928\u093f\u0937\u094d\u0915\u0930\u094d\u0937 \u0907\u0938 \u092a\u094d\u0930\u0915\u093e\u0930 \u0939\u0948\u0902:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093c\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0909\u0924\u094d\u092a\u093e\u0926\u0928:<\/strong> PBGENE-DMD \u0928\u0947 \u0939\u0943\u0926\u092f, \u0921\u093e\u092f\u093e\u092b\u094d\u0930\u093e\u092e \u0914\u0930 \u0915\u0902\u0915\u093e\u0932 \u0915\u0940 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0938\u0939\u093f\u0924 \u0905\u0928\u0947\u0915 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \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 \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u0915\u0940 \u0936\u0930\u0940\u0930 \u0915\u0940 \u0915\u094d\u0937\u092e\u0924\u093e \u0915\u094b \u092c\u0939\u093e\u0932 \u0915\u093f\u092f\u093e, \u091c\u093f\u0938\u0938\u0947 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u0940\u092f \u0932\u093e\u092d \u092e\u093f\u0932\u0928\u0947 \u0915\u0940 \u0909\u092e\u094d\u092e\u0940\u0926 \u0925\u0940\u0964<\/li>\n\n\n\n<li><strong>\u092c\u0922\u093c\u0940 \u0939\u0941\u0908 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0932\u091a\u0940\u0932\u093e\u092a\u0928:<\/strong> \u0909\u092a\u091a\u093e\u0930\u093f\u0924 \u091a\u0942\u0939\u094b\u0902 \u092e\u0947\u0902, \u0909\u092a\u091a\u093e\u0930\u093f\u0924 \u0930\u094b\u0917\u0917\u094d\u0930\u0938\u094d\u0924 \u091a\u0942\u0939\u094b\u0902 \u0915\u0940 \u0924\u0941\u0932\u0928\u093e \u092e\u0947\u0902 \u0935\u093f\u0932\u0915\u094d\u0937\u0923 \u091a\u094b\u091f \u0915\u0947 \u092a\u094d\u0930\u0924\u093f \u092a\u094d\u0930\u0924\u093f\u0930\u094b\u0927 \u092e\u0947\u0902 66% \u0938\u0941\u0927\u093e\u0930 \u0926\u0947\u0916\u093e \u0917\u092f\u093e, \u091c\u094b \u0915\u093f \u092c\u0922\u093c\u0940 \u0939\u0941\u0908 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0924\u0928\u094d\u092f\u0915\u0924\u093e \u0915\u093e \u0938\u0942\u091a\u0915 \u0939\u0948\u0964<\/li>\n\n\n\n<li><strong>\u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0938\u0941\u0927\u093e\u0930:<\/strong> PBGENE-DMD \u0938\u0947 \u0909\u092a\u091a\u093e\u0930\u093f\u0924 \u091a\u0942\u0939\u094b\u0902 \u092e\u0947\u0902 \u0905\u0927\u093f\u0915\u0924\u092e \u092c\u0932 \u0928\u093f\u0930\u094d\u0917\u0924 (MFO), \u091c\u094b \u0915\u093f \u090f\u0915 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u092e\u0940\u091f\u094d\u0930\u093f\u0915 \u0939\u0948, \u0938\u094d\u0935\u0938\u094d\u0925 \u0928\u093f\u092f\u0902\u0924\u094d\u0930\u0923 \u091a\u0942\u0939\u094b\u0902 \u092e\u0947\u0902 MFO \u0915\u0947 93% \u0924\u0915 \u092a\u0939\u0941\u0902\u091a \u0917\u092f\u093e, \u091c\u092c\u0915\u093f PBGENE-DMD \u0909\u092a\u091a\u093e\u0930\u093f\u0924 \u091a\u0942\u0939\u094b\u0902 \u092e\u0947\u0902 3 \u0938\u0947 6 \u092e\u0939\u0940\u0928\u0947 \u0915\u0947 \u092c\u0940\u091a \u0938\u0941\u0927\u093e\u0930 \u0926\u0947\u0916\u093e \u0917\u092f\u093e\u0964<\/li>\n\n\n\n<li><strong>\u091f\u093f\u0915\u093e\u090a \u092a\u0930\u093f\u0923\u093e\u092e:<\/strong> 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 \u0915\u094b PAX7+ \u0915\u094b\u0936\u093f\u0915\u093e\u0913\u0902 \u092e\u0947\u0902 \u092a\u093e\u092f\u093e \u0917\u092f\u093e, \u091c\u094b \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 \u0915\u0947 \u0932\u093f\u090f \u090f\u0915 \u092e\u093e\u0930\u094d\u0915\u0930 \u0939\u0948, \u091c\u094b \u092e\u093e\u0928\u0915 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923\u094b\u0902 \u0915\u0940 \u0924\u0941\u0932\u0928\u093e \u092e\u0947\u0902 \u091f\u093f\u0915\u093e\u090a \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u0940\u092f \u092a\u094d\u0930\u092d\u093e\u0935\u094b\u0902 \u0915\u0940 \u0915\u094d\u0937\u092e\u0924\u093e \u0915\u093e \u0938\u0941\u091d\u093e\u0935 \u0926\u0947\u0924\u093e \u0939\u0948\u0964<\/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\/hi\/wp-json\/wp\/v2\/posts\/3288","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=3288"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/3288\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media\/3289"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media?parent=3288"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/categories?post=3288"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/tags?post=3288"}],"curies":[{"name":"\u0921\u092c\u094d\u0932\u094d\u092f\u0942\u092a\u0940","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}