{"id":7752,"date":"2026-04-28T20:01:26","date_gmt":"2026-04-28T17:01:26","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=7752"},"modified":"2026-05-15T13:57:36","modified_gmt":"2026-05-15T10:57:36","slug":"faq-pbgene-dmd-gene-editing-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/hi\/faq-pbgene-dmd-gene-editing-therapy\/","title":{"rendered":"PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940: \u0932\u093e\u092d, \u092a\u093e\u0924\u094d\u0930\u0924\u093e \u0914\u0930 \u0905\u0915\u094d\u0938\u0930 \u092a\u0942\u091b\u0947 \u091c\u093e\u0928\u0947 \u0935\u093e\u0932\u0947 \u092a\u094d\u0930\u0936\u094d\u0928"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940, \u0921\u094d\u092f\u0942\u0936\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 \u0932\u093f\u090f Precision BioSciences \u0926\u094d\u0935\u093e\u0930\u093e \u0935\u093f\u0915\u0938\u093f\u0924 \u090f\u0915 \u092a\u094d\u0930\u093e\u092f\u094b\u0917\u093f\u0915, \u0905\u092a\u0928\u0940 \u0924\u0930\u0939 \u0915\u093e \u092a\u0939\u0932\u093e \u0909\u092a\u091a\u093e\u0930 \u0939\u0948\u0964.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u092a\u0930\u0902\u092a\u0930\u093e\u0917\u0924 \u0909\u092a\u091a\u093e\u0930\u094b\u0902 \u0915\u0947 \u0935\u093f\u092a\u0930\u0940\u0924, PBGENE-DMD ARCUS \u091c\u0940\u0928 \u0938\u0902\u092a\u093e\u0926\u0928 \u0924\u0915\u0928\u0940\u0915 \u0926\u094d\u0935\u093e\u0930\u093e \u0938\u0902\u091a\u093e\u0932\u093f\u0924 \u091c\u0940\u0928 \u0928\u093f\u0937\u094d\u0915\u093e\u0938\u0928 \u0930\u0923\u0928\u0940\u0924\u093f \u0915\u093e \u0909\u092a\u092f\u094b\u0917 \u0915\u0930\u0924\u093e \u0939\u0948\u0964 \u0907\u0938\u0947 \u090f\u0915 \u090f\u0915\u0932 \u090f\u0921\u0947\u0928\u094b-\u090f\u0938\u094b\u0938\u093f\u090f\u091f\u0947\u0921 \u0935\u093e\u092f\u0930\u0938 (AAV) \u0935\u0947\u0915\u094d\u091f\u0930 \u0915\u0947 \u092e\u093e\u0927\u094d\u092f\u092e \u0938\u0947 \u092a\u0939\u0941\u0902\u091a\u093e\u092f\u093e \u091c\u093e\u0924\u093e \u0939\u0948, \u091c\u093f\u0938\u092e\u0947\u0902 \u0926\u094b ARCUS \u0928\u094d\u092f\u0942\u0915\u094d\u0932\u093f\u090f\u091c\u093c \u090f\u0928\u094d\u0915\u094b\u0921 \u0939\u094b\u0924\u0947 \u0939\u0948\u0902 \u091c\u094b \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u091c\u0940\u0928 \u0915\u094b \u0938\u094d\u0925\u093e\u092f\u0940 \u0930\u0942\u092a \u0938\u0947 \u0938\u0902\u092a\u093e\u0926\u093f\u0924 \u0915\u0930\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0921\u093f\u091c\u093c\u093e\u0907\u0928 \u0915\u093f\u090f \u0917\u090f \u0939\u0948\u0902\u0964.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0907\u0938 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923 \u0915\u093e \u0909\u0926\u094d\u0926\u0947\u0936\u094d\u092f \u0928\u093f\u092e\u094d\u0928\u0932\u093f\u0916\u093f\u0924 \u0939\u0948:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u091c\u0940\u0928 \u0915\u0947 \u092d\u0940\u0924\u0930 \u0921\u0940\u090f\u0928\u090f \u0915\u0947 \u0926\u094b\u0937\u092a\u0942\u0930\u094d\u0923 \u092d\u093e\u0917\u094b\u0902 \u0915\u094b \u0939\u091f\u093e \u0926\u0947\u0902<\/li>\n\n\n\n<li><strong>\u0936\u0930\u0940\u0930 \u0915\u094b \u0932\u0917\u092d\u0917 \u092a\u0942\u0930\u094d\u0923 \u0932\u0902\u092c\u093e\u0908 \u0935\u093e\u0932\u093e, \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 \u092c\u0928\u093e\u0928\u0947 \u092e\u0947\u0902 \u0938\u0915\u094d\u0937\u092e \u092c\u0928\u093e\u0928\u093e<\/strong><\/li>\n\n\n\n<li>\u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0914\u0930 \u0938\u0902\u092d\u093e\u0935\u093f\u0924 \u0930\u0942\u092a \u0938\u0947 \u0938\u094d\u0925\u093e\u092f\u0940 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0938\u0941\u0927\u093e\u0930 \u092a\u094d\u0930\u0926\u093e\u0928 \u0915\u0930\u0947\u0902<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u092a\u094d\u0930\u0940\u0915\u094d\u0932\u093f\u0928\u093f\u0915\u0932 \u0921\u0947\u091f\u093e \u0938\u0947 \u092a\u0924\u093e \u091a\u0932\u0924\u093e \u0939\u0948 \u0915\u093f PBGENE-DMD \u0915\u0902\u0915\u093e\u0932 \u0915\u0940 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902, \u0921\u093e\u092f\u093e\u092b\u094d\u0930\u093e\u092e \u0914\u0930 \u0939\u0943\u0926\u092f \u090a\u0924\u0915 \u0938\u0939\u093f\u0924 \u0915\u0908 \u092a\u094d\u0930\u0915\u093e\u0930 \u0915\u0940 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0905\u092d\u093f\u0935\u094d\u092f\u0915\u094d\u0924\u093f \u0915\u094b \u092c\u0939\u093e\u0932 \u0915\u0930 \u0938\u0915\u0924\u093e \u0939\u0948, \u0938\u093e\u0925 \u0939\u0940 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0915\u0940 \u0938\u0948\u091f\u0947\u0932\u093e\u0907\u091f \u0938\u094d\u091f\u0947\u092e \u0915\u094b\u0936\u093f\u0915\u093e\u0913\u0902 \u0915\u094b \u092d\u0940 \u0938\u0902\u092a\u093e\u0926\u093f\u0924 \u0915\u0930 \u0938\u0915\u0924\u093e \u0939\u0948, \u091c\u094b \u0928\u093f\u0930\u0902\u0924\u0930 \u0932\u093e\u092d \u0915\u0947 \u0932\u093f\u090f \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0939\u0948\u0902\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=\"#why-is-pbgene-dmd-gene-editing-therapy-important\">PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u094d\u092f\u094b\u0902 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0939\u0948?<\/a><ul><li><a href=\"#permanent-gene-editing\">\u0938\u094d\u0925\u093e\u092f\u0940 \u091c\u0940\u0928 \u0938\u0902\u092a\u093e\u0926\u0928<\/a><\/li><li><a href=\"#near-full-length-dystrophin-production\">\u0932\u0917\u092d\u0917 \u092a\u0942\u0930\u094d\u0923-\u0932\u0902\u092c\u093e\u0908 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0909\u0924\u094d\u092a\u093e\u0926\u0928<\/a><\/li><li><a href=\"#targeting-muscle-stem-cells\">\u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0938\u094d\u091f\u0947\u092e \u0915\u094b\u0936\u093f\u0915\u093e\u0913\u0902 \u0915\u094b \u0932\u0915\u094d\u0937\u093f\u0924 \u0915\u0930\u0928\u093e<\/a><\/li><li><a href=\"#multi-tissue-impact\">\u092c\u0939\u0941-\u090a\u0924\u0915 \u092a\u094d\u0930\u092d\u093e\u0935<\/a><\/li><li><a href=\"#regulatory-recognition\">\u0928\u093f\u092f\u093e\u092e\u0915 \u092e\u093e\u0928\u094d\u092f\u0924\u093e<\/a><\/li><\/ul><\/li><li><a href=\"#who-can-receive-pbgene-dmd-gene-editing-therapy-eligibility-fa-qs\">PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u094c\u0928 \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0915\u0930 \u0938\u0915\u0924\u093e \u0939\u0948? (\u092a\u093e\u0924\u094d\u0930\u0924\u093e \u0938\u0902\u092c\u0902\u0927\u0940 \u0905\u0915\u094d\u0938\u0930 \u092a\u0942\u091b\u0947 \u091c\u093e\u0928\u0947 \u0935\u093e\u0932\u0947 \u092a\u094d\u0930\u0936\u094d\u0928)<\/a><ul><li><a href=\"#\u2705-eligible-patients\">\u2705 \u092a\u093e\u0924\u094d\u0930 \u092e\u0930\u0940\u091c\u093c<\/a><\/li><li><a href=\"#faq-question-1777392685572\">\u0915\u094c\u0928 \u0938\u0947 \u090f\u0915\u094d\u0938\u0949\u0928 \u0935\u093f\u0932\u094b\u092a\u0928 PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u0932\u093f\u090f \u092a\u093e\u0924\u094d\u0930 \u0939\u0948\u0902?<\/a><\/li><li><a href=\"#faq-question-1777392706521\">\u0907\u0938\u0915\u0947 \u0932\u093f\u090f \u0915\u093f\u0928 \u0936\u093e\u0930\u0940\u0930\u093f\u0915 \u0915\u094d\u0937\u092e\u0924\u093e\u0913\u0902 \u0915\u0940 \u0906\u0935\u0936\u094d\u092f\u0915\u0924\u093e \u0939\u094b\u0924\u0940 \u0939\u0948?<\/a><\/li><li><a href=\"#faq-question-1777392815309\">\u0915\u094d\u092f\u093e \u091f\u0940\u0915\u093e\u0915\u0930\u0923 \u0905\u0928\u093f\u0935\u093e\u0930\u094d\u092f \u0939\u0948?<\/a><\/li><li><a href=\"#faq-question-1777392838933\">\u0915\u094d\u092f\u093e \u0938\u0939\u092e\u0924\u093f \u0906\u0935\u0936\u094d\u092f\u0915 \u0939\u0948?<\/a><\/li><li><a href=\"#faq-question-1777392849177\">\u0915\u094d\u092f\u093e \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0905\u0928\u0941\u0935\u0930\u094d\u0924\u0940 \u0915\u093e\u0930\u094d\u0930\u0935\u093e\u0908 \u0906\u0935\u0936\u094d\u092f\u0915 \u0939\u0948?<\/a><\/li><\/ul><\/li><li><a href=\"#who-is-not-eligible-for-pbgene-dmd-exclusion-fa-qs\">PBGENE-DMD \u0915\u0947 \u0932\u093f\u090f \u0915\u094c\u0928 \u092a\u093e\u0924\u094d\u0930 \u0928\u0939\u0940\u0902 \u0939\u0948? (\u0905\u092a\u0935\u093e\u0926 \u0938\u0902\u092c\u0902\u0927\u0940 \u0905\u0915\u094d\u0938\u0930 \u092a\u0942\u091b\u0947 \u091c\u093e\u0928\u0947 \u0935\u093e\u0932\u0947 \u092a\u094d\u0930\u0936\u094d\u0928)<\/a><ul><li><a href=\"#\u274c-ineligible-patients\">\u274c \u0905\u092a\u093e\u0924\u094d\u0930 \u0930\u094b\u0917\u0940<\/a><\/li><li><a href=\"#faq-question-1777393251291\">\u0915\u094d\u092f\u093e \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0930\u093e \u091a\u0941\u0915\u0947 \u092e\u0930\u0940\u091c \u0907\u0938\u092e\u0947\u0902 \u092d\u093e\u0917 \u0932\u0947 \u0938\u0915\u0924\u0947 \u0939\u0948\u0902?<\/a><\/li><li><a href=\"#faq-question-1777393281660\">\u090f\u0915\u094d\u0938\u0949\u0928 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u092f\u093e \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u092c\u093e\u0930\u0947 \u092e\u0947\u0902 \u0915\u094d\u092f\u093e \u0916\u094d\u092f\u093e\u0932 \u0939\u0948?<\/a><\/li><li><a href=\"#faq-question-1777393309651\">\u0915\u094d\u092f\u093e \u0905\u0928\u094d\u092f \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923\u094b\u0902 \u092e\u0947\u0902 \u0936\u093e\u092e\u093f\u0932 \u092e\u0930\u0940\u091c \u092a\u093e\u0924\u094d\u0930 \u0939\u0948\u0902?<\/a><\/li><li><a href=\"#faq-question-1777393318621\">\u0915\u094d\u092f\u093e \u090f\u090f\u0935\u0940 \u090f\u0902\u091f\u0940\u092c\u0949\u0921\u0940-\u092a\u0949\u091c\u093f\u091f\u093f\u0935 \u092e\u0930\u0940\u091c \u092a\u093e\u0924\u094d\u0930 \u0939\u0948\u0902?<\/a><\/li><li><a href=\"#faq-question-1777393328468\">\u0915\u094d\u092f\u093e \u0921\u0940\u090f\u092e\u0921\u0940 \u0915\u0947 \u0938\u092d\u0940 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u092a\u093e\u0924\u094d\u0930 \u0939\u0948\u0902?<\/a><\/li><li><a href=\"#faq-question-1777393410826\">\u0915\u093f\u0928 \u0939\u0943\u0926\u092f \u0938\u0902\u092c\u0902\u0927\u0940 \u0938\u094d\u0925\u093f\u0924\u093f\u092f\u094b\u0902 \u0915\u0947 \u0915\u093e\u0930\u0923 \u092e\u0930\u0940\u091c\u094b\u0902 \u0915\u094b \u0907\u0938 \u0905\u0927\u094d\u092f\u092f\u0928 \u0938\u0947 \u092c\u093e\u0939\u0930 \u0930\u0916\u093e \u091c\u093e\u0924\u093e \u0939\u0948?<\/a><\/li><li><a href=\"#faq-question-1777393452605\">\u0915\u094d\u092f\u093e \u092a\u094d\u0930\u0924\u093f\u0930\u0915\u094d\u0937\u093e\u0926\u092e\u0928 \u0938\u0902\u092c\u0902\u0927\u0940 \u092e\u0924\u092d\u0947\u0926\u094b\u0902 \u0935\u093e\u0932\u0947 \u092e\u0930\u0940\u091c \u092d\u093e\u0917 \u0932\u0947 \u0938\u0915\u0924\u0947 \u0939\u0948\u0902?<\/a><\/li><\/ul><\/li><li><a href=\"#pbgene-dmd-gene-editing-therapy-fa-qs-from-families\">PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940: \u092a\u0930\u093f\u0935\u093e\u0930\u094b\u0902 \u0926\u094d\u0935\u093e\u0930\u093e \u092a\u0942\u091b\u0947 \u091c\u093e\u0928\u0947 \u0935\u093e\u0932\u0947 \u0905\u0915\u094d\u0938\u0930 \u092a\u0942\u091b\u0947 \u091c\u093e\u0928\u0947 \u0935\u093e\u0932\u0947 \u092a\u094d\u0930\u0936\u094d\u0928<\/a><ul><li><a href=\"#faq-question-1777394327672\">\u0915\u094d\u092f\u093e Elevidys \u0938\u0947 \u0909\u092a\u091a\u093e\u0930\u093f\u0924 \u092c\u091a\u094d\u091a\u093e PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u092d\u0940 \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0915\u0930 \u0938\u0915\u0924\u093e \u0939\u0948?<\/a><\/li><li><a href=\"#faq-question-1777394381372\">\u0915\u094d\u092f\u093e \u092e\u0947\u0930\u0947 \u092c\u0947\u091f\u0947 \u0915\u094b, \u091c\u093f\u0938\u0915\u0947 \u0921\u0940\u090f\u092e\u0921\u0940 \u091c\u0940\u0928 \u092e\u0947\u0902 \u090f\u0915\u094d\u0938\u0949\u0928 43 \u0915\u093e \u0935\u093f\u0932\u094b\u092a\u0928 \u0939\u0948, PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u092e\u093f\u0932 \u0938\u0915\u0924\u0940 \u0939\u0948?<\/a><\/li><li><a href=\"#faq-question-1777394586338\">\u0915\u094d\u092f\u093e \u092e\u0947\u0930\u0947 \u092c\u0947\u091f\u0947 \u0915\u094b, \u091c\u093f\u0938\u0915\u0947 \u091c\u0940\u0928 \u092e\u0947\u0902 \u090f\u0915\u094d\u0938\u0949\u0928 48-55 \u0915\u093e \u0935\u093f\u0932\u094b\u092a\u0928 \u0939\u0948, PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u092e\u093f\u0932 \u0938\u0915\u0924\u0940 \u0939\u0948?<\/a><\/li><li><a href=\"#faq-question-1777395554562\">\u0915\u094d\u092f\u093e \u090f\u0915\u094d\u0938\u0949\u0928 45 \u0921\u093f\u0932\u0940\u0936\u0928 \u0935\u093e\u0932\u0947 \u0921\u0940\u090f\u092e\u0921\u0940 \u0938\u0947 \u092a\u0940\u0921\u093c\u093f\u0924 \u0932\u0921\u093c\u0915\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f PBGENE-DMD \u090f\u0915 \u0909\u092a\u091a\u093e\u0930 \u0935\u093f\u0915\u0932\u094d\u092a \u0939\u0948?<\/a><\/li><\/ul><\/li><li><a href=\"#precision-bio-sciences-presents-new-preclinical-data-supporting-the-advancement-of-pbgene-dmd-into-clinic\">Precision BioSciences \u0928\u0947 PBGENE-DMD \u0915\u094b \u0915\u094d\u0932\u093f\u0928\u093f\u0915 \u092e\u0947\u0902 \u0906\u0917\u0947 \u092c\u0922\u093c\u093e\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0938\u092e\u0930\u094d\u0925\u0928 \u0926\u0947\u0928\u0947 \u0935\u093e\u0932\u0947 \u0928\u090f \u092a\u094d\u0930\u0940\u0915\u094d\u0932\u093f\u0928\u093f\u0915\u0932 \u0921\u0947\u091f\u093e \u092a\u094d\u0930\u0938\u094d\u0924\u0941\u0924 \u0915\u093f\u090f \u0939\u0948\u0902\u0964<\/a><\/li><li><a href=\"#final-thoughts\">\u0905\u0902\u0924\u093f\u092e \u0935\u093f\u091a\u093e\u0930<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"why-is-pbgene-dmd-gene-editing-therapy-important\"><strong>PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u094d\u092f\u094b\u0902 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0939\u0948?<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">PBGENE-DMD \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u0930\u094b\u0917 \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u0940 \u0930\u0923\u0928\u0940\u0924\u093f \u092e\u0947\u0902 \u090f\u0915 \u092c\u0921\u093c\u093e \u092c\u0926\u0932\u093e\u0935 \u0926\u0930\u094d\u0936\u093e\u0924\u093e \u0939\u0948, \u091c\u094b \u092e\u094c\u091c\u0942\u0926\u093e \u0909\u092a\u091a\u093e\u0930\u094b\u0902 \u0915\u0940 \u0924\u0941\u0932\u0928\u093e \u092e\u0947\u0902 \u0915\u0908 \u0938\u0902\u092d\u093e\u0935\u093f\u0924 \u0932\u093e\u092d \u092a\u094d\u0930\u0926\u093e\u0928 \u0915\u0930\u0924\u093e \u0939\u0948:<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"permanent-gene-editing\"><strong>\u0938\u094d\u0925\u093e\u092f\u0940 \u091c\u0940\u0928 \u0938\u0902\u092a\u093e\u0926\u0928<\/strong><\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u0935\u0930\u094d\u0924\u092e\u093e\u0928 \u092e\u0947\u0902 \u0909\u092a\u0932\u092c\u094d\u0927 \u0905\u0927\u093f\u0915\u093e\u0902\u0936 \u0909\u092a\u091a\u093e\u0930, \u091c\u0948\u0938\u0947 \u0915\u093f \u090f\u0915\u094d\u0938\u0949\u0928 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917, \u092e\u0947\u0902 \u092c\u093e\u0930-\u092c\u093e\u0930 \u0916\u0941\u0930\u093e\u0915 \u0915\u0940 \u0906\u0935\u0936\u094d\u092f\u0915\u0924\u093e \u0939\u094b\u0924\u0940 \u0939\u0948 \u0914\u0930 \u0907\u0928\u0915\u093e \u092a\u094d\u0930\u092d\u093e\u0935 \u0905\u0938\u094d\u0925\u093e\u092f\u0940 \u0939\u094b\u0924\u093e \u0939\u0948\u0964 PBGENE-DMD \u0915\u093e \u0909\u0926\u094d\u0926\u0947\u0936\u094d\u092f \u0921\u0940\u090f\u0928\u090f \u0915\u094b \u0938\u094d\u0925\u093e\u092f\u0940 \u0930\u0942\u092a \u0938\u0947 \u0938\u0902\u0936\u094b\u0927\u093f\u0924 \u0915\u0930\u0928\u093e \u0939\u0948, \u091c\u093f\u0938\u0938\u0947 \u0938\u0902\u092d\u0935\u0924\u0903 \u090f\u0915 \u092c\u093e\u0930 \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u093e \u0935\u093f\u0915\u0932\u094d\u092a \u0909\u092a\u0932\u092c\u094d\u0927 \u0939\u094b \u0938\u0915\u0924\u093e \u0939\u0948\u0964.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"near-full-length-dystrophin-production\"><strong>\u0932\u0917\u092d\u0917 \u092a\u0942\u0930\u094d\u0923-\u0932\u0902\u092c\u093e\u0908 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0909\u0924\u094d\u092a\u093e\u0926\u0928<\/strong><\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u092e\u093e\u0907\u0915\u094d\u0930\u094b\u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u0935\u093f\u092a\u0930\u0940\u0924, \u091c\u094b \u091b\u094b\u091f\u0947 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u093e \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u0915\u0930\u0924\u0940 \u0939\u0948\u0902, PBGENE-DMD \u0915\u094b \u0905\u0927\u093f\u0915 \u092a\u0942\u0930\u094d\u0923 \u0914\u0930 \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\u094b \u092c\u0939\u093e\u0932 \u0915\u0930\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0921\u093f\u091c\u093c\u093e\u0907\u0928 \u0915\u093f\u092f\u093e \u0917\u092f\u093e \u0939\u0948, \u091c\u093f\u0938\u0938\u0947 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u093f\u0923\u093e\u092e\u094b\u0902 \u092e\u0947\u0902 \u0938\u0941\u0927\u093e\u0930 \u0939\u094b \u0938\u0915\u0924\u093e \u0939\u0948\u0964.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"targeting-muscle-stem-cells\"><strong>\u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0938\u094d\u091f\u0947\u092e \u0915\u094b\u0936\u093f\u0915\u093e\u0913\u0902 \u0915\u094b \u0932\u0915\u094d\u0937\u093f\u0924 \u0915\u0930\u0928\u093e<\/strong><\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u0938\u0948\u091f\u0947\u0932\u093e\u0907\u091f \u0915\u094b\u0936\u093f\u0915\u093e\u0913\u0902 \u0915\u094b \u0938\u0902\u092a\u093e\u0926\u093f\u0924 \u0915\u0930\u0915\u0947, PBGENE-DMD \u0928\u093f\u0930\u0902\u0924\u0930 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u092a\u0941\u0928\u0930\u094d\u091c\u0928\u0928 \u0914\u0930 \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0938\u094d\u0925\u093e\u092f\u093f\u0924\u094d\u0935 \u0915\u093e \u0938\u092e\u0930\u094d\u0925\u0928 \u0915\u0930 \u0938\u0915\u0924\u093e \u0939\u0948, \u091c\u094b \u0905\u0928\u094d\u092f \u0909\u092a\u091a\u093e\u0930\u094b\u0902 \u0915\u0940 \u090f\u0915 \u092a\u094d\u0930\u092e\u0941\u0916 \u0938\u0940\u092e\u093e \u0939\u0948\u0964.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"multi-tissue-impact\"><strong>\u092c\u0939\u0941-\u090a\u0924\u0915 \u092a\u094d\u0930\u092d\u093e\u0935<\/strong><\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u092a\u0942\u0930\u094d\u0935-\u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0905\u0927\u094d\u092f\u092f\u0928\u094b\u0902 \u092e\u0947\u0902 \u0928\u093f\u092e\u094d\u0928\u0932\u093f\u0916\u093f\u0924 \u0915\u094d\u0937\u0947\u0924\u094d\u0930\u094b\u0902 \u092e\u0947\u0902 \u092a\u094d\u0930\u092d\u093e\u0935 \u092a\u094d\u0930\u0926\u0930\u094d\u0936\u093f\u0924 \u0915\u093f\u090f \u0917\u090f \u0939\u0948\u0902:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0915\u0902\u0915\u093e\u0932 \u0915\u0940 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940<\/li>\n\n\n\n<li>\u0939\u0943\u0926\u092f \u0915\u0940 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940<\/li>\n\n\n\n<li>\u0921\u093e\u092f\u093e\u092b\u094d\u0930\u093e\u092e<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u0921\u0940\u090f\u092e\u0921\u0940 \u0915\u0940 \u092a\u094d\u0930\u0917\u0924\u093f \u092e\u0947\u0902 \u0939\u0943\u0926\u092f \u0914\u0930 \u0936\u094d\u0935\u0938\u0928 \u0935\u093f\u092b\u0932\u0924\u093e \u0915\u0940 \u092d\u0942\u092e\u093f\u0915\u093e \u0915\u094b \u0926\u0947\u0916\u0924\u0947 \u0939\u0941\u090f \u092f\u0939 \u0935\u093f\u0936\u0947\u0937 \u0930\u0942\u092a \u0938\u0947 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0939\u0948\u0964.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"regulatory-recognition\"><strong>\u0928\u093f\u092f\u093e\u092e\u0915 \u092e\u093e\u0928\u094d\u092f\u0924\u093e<\/strong><\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">PBGENE-DMD \u0915\u094b FDA \u0938\u0947 \u0915\u0908 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u092a\u0926\u0928\u093e\u092e \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0939\u0941\u090f \u0939\u0948\u0902, \u091c\u093f\u0928\u092e\u0947\u0902 \u0936\u093e\u092e\u093f\u0932 \u0939\u0948\u0902:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u092b\u093e\u0938\u094d\u091f \u091f\u094d\u0930\u0948\u0915 \u092a\u0926\u0928\u093e\u092e<\/li>\n\n\n\n<li>\u0905\u0928\u093e\u0925 \u0914\u0937\u0927\u093f \u092a\u0926\u0928\u093e\u092e<\/li>\n\n\n\n<li>\u0926\u0941\u0930\u094d\u0932\u092d \u092c\u093e\u0932 \u0930\u094b\u0917 \u092a\u0926\u0928\u093e\u092e<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"who-can-receive-pbgene-dmd-gene-editing-therapy-eligibility-fa-qs\"><strong>PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u094c\u0928 \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0915\u0930 \u0938\u0915\u0924\u093e \u0939\u0948? (\u092a\u093e\u0924\u094d\u0930\u0924\u093e \u0938\u0902\u092c\u0902\u0927\u0940 \u0905\u0915\u094d\u0938\u0930 \u092a\u0942\u091b\u0947 \u091c\u093e\u0928\u0947 \u0935\u093e\u0932\u0947 \u092a\u094d\u0930\u0936\u094d\u0928)<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0928\u093f\u092e\u094d\u0928\u0932\u093f\u0916\u093f\u0924 \u092e\u093e\u0928\u0926\u0902\u0921 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923 \u0915\u0940 \u092a\u093e\u0924\u094d\u0930\u0924\u093e \u0906\u0935\u0936\u094d\u092f\u0915\u0924\u093e\u0913\u0902 \u092a\u0930 \u0906\u0927\u093e\u0930\u093f\u0924 \u0939\u0948\u0902\u0964 .<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"\u2705-eligible-patients\">\u2705 <strong>\u092a\u093e\u0924\u094d\u0930 \u092e\u0930\u0940\u091c\u093c<\/strong><\/h3>\n\n\n<div id=\"rank-math-faq\" class=\"rank-math-block\">\n<div class=\"rank-math-list\">\n<div id=\"faq-question-1777392685572\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0915\u094c\u0928 \u0938\u0947 \u090f\u0915\u094d\u0938\u0949\u0928 \u0935\u093f\u0932\u094b\u092a\u0928 PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u0932\u093f\u090f \u092a\u093e\u0924\u094d\u0930 \u0939\u0948\u0902?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u092e\u0930\u0940\u091c\u094b\u0902 \u092e\u0947\u0902 \u090f\u0915\u094d\u0938\u0949\u0928 45-55 \u0915\u0947 \u092d\u0940\u0924\u0930 \u092a\u0942\u0930\u0940 \u0924\u0930\u0939 \u0938\u0947 \u0938\u092e\u093e\u0939\u093f\u0924 \u090f\u0915 \u092a\u0941\u0937\u094d\u091f \u0921\u0940\u090f\u092e\u0921\u0940 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0939\u094b\u0928\u093e \u0906\u0935\u0936\u094d\u092f\u0915 \u0939\u0948\u0964.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1777392706521\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0907\u0938\u0915\u0947 \u0932\u093f\u090f \u0915\u093f\u0928 \u0936\u093e\u0930\u0940\u0930\u093f\u0915 \u0915\u094d\u0937\u092e\u0924\u093e\u0913\u0902 \u0915\u0940 \u0906\u0935\u0936\u094d\u092f\u0915\u0924\u093e \u0939\u094b\u0924\u0940 \u0939\u0948?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>2 \u0938\u0947 &lt;4 \u0935\u0930\u094d\u0937 \u0915\u0940 \u0906\u092f\u0941:<\/p>\n<p>\u2022 \u0915\u092e \u0938\u0947 \u0915\u092e 10 \u092e\u0940\u091f\u0930 \u0924\u0915 \u0938\u094d\u0935\u0924\u0902\u0924\u094d\u0930 \u0930\u0942\u092a \u0938\u0947 \u091a\u0932\u0928\u0947 \u0915\u0940 \u0915\u094d\u0937\u092e\u0924\u093e<br \/>\u2022 \u092b\u0930\u094d\u0936 \u0938\u0947 \u0909\u0920\u0928\u0947 \u0915\u0940 \u0915\u094d\u0937\u092e\u0924\u093e <a href=\"https:\/\/dmdwarrior.com\/hi\/gowers-sign-in-duchenne-muscular-dystrophy\/\" data-type=\"post\" data-id=\"7263\">(\u0917\u094b\u0935\u0930\u094d\u0938 \u0915\u0940 \u091a\u093e\u0932 \u0915\u094b \u0905\u0928\u0941\u092e\u0924\u093f \u0926\u0940 \u0917\u0908)<\/a><\/p>\n<p>4 \u0938\u0947 7 \u0935\u0930\u094d\u0937 \u0915\u0940 \u0906\u092f\u0941:<\/p>\n<p>\u2022 \u0915\u092e \u0938\u0947 \u0915\u092e 100 \u092e\u0940\u091f\u0930 \u0924\u0915 \u0938\u094d\u0935\u0924\u0902\u0924\u094d\u0930 \u0930\u0942\u092a \u0938\u0947 \u091a\u0932\u0928\u0947 \u0915\u0940 \u0915\u094d\u0937\u092e\u0924\u093e<br \/>\u2022 \u090f\u0928\u090f\u0938\u090f\u090f \u0938\u094d\u0915\u094b\u0930 16 \u0914\u0930 29 \u0915\u0947 \u092c\u0940\u091a<\/p>\n<p>\u0914\u0930 \u0905\u0927\u093f\u0915 \u091c\u093e\u0928\u0947\u0902: <a href=\"https:\/\/dmdwarrior.com\/hi\/north-star-ambulatory-assessment-nsaa-in-duchenne-dmd\/\" target=\"_blank\" data-type=\"post\" data-id=\"4959\" rel=\"noreferrer noopener\">\u090f\u0928\u090f\u0938\u090f\u090f<\/a><\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1777392815309\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0915\u094d\u092f\u093e \u091f\u0940\u0915\u093e\u0915\u0930\u0923 \u0905\u0928\u093f\u0935\u093e\u0930\u094d\u092f \u0939\u0948?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u091c\u0940 \u0939\u093e\u0901\u0964 \u092e\u0930\u0940\u091c\u094b\u0902 \u0915\u094b \u0928\u093f\u092f\u092e\u093f\u0924 \u092c\u091a\u092a\u0928 \u0915\u0947 \u091f\u0940\u0915\u093e\u0915\u0930\u0923 \u0915\u0947 \u0938\u093e\u0925 \u0905\u0926\u094d\u092f\u0924\u0928 \u0930\u0939\u0928\u093e \u091a\u093e\u0939\u093f\u090f\u0964.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1777392838933\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0915\u094d\u092f\u093e \u0938\u0939\u092e\u0924\u093f \u0906\u0935\u0936\u094d\u092f\u0915 \u0939\u0948?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u091c\u0940 \u0939\u093e\u0901\u0964 \u092e\u093e\u0924\u093e-\u092a\u093f\u0924\u093e \u092f\u093e \u0915\u093e\u0928\u0942\u0928\u0940 \u0905\u092d\u093f\u092d\u093e\u0935\u0915\u094b\u0902 \u0915\u094b \u0932\u093f\u0916\u093f\u0924 \u0930\u0942\u092a \u092e\u0947\u0902 \u0938\u0942\u091a\u093f\u0924 \u0938\u0939\u092e\u0924\u093f \u092a\u094d\u0930\u0926\u093e\u0928 \u0915\u0930\u0928\u0940 \u0939\u094b\u0917\u0940, \u0914\u0930 \u091c\u0939\u093e\u0902 \u0932\u093e\u0917\u0942 \u0939\u094b \u0935\u0939\u093e\u0902 \u092c\u091a\u094d\u091a\u094b\u0902 \u0915\u094b \u092d\u0940 \u0938\u0939\u092e\u0924\u093f \u0926\u0947\u0928\u0940 \u0939\u094b\u0917\u0940\u0964.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1777392849177\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0915\u094d\u092f\u093e \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0905\u0928\u0941\u0935\u0930\u094d\u0924\u0940 \u0915\u093e\u0930\u094d\u0930\u0935\u093e\u0908 \u0906\u0935\u0936\u094d\u092f\u0915 \u0939\u0948?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u091c\u0940 \u0939\u093e\u0901\u0964 \u092e\u0930\u0940\u091c\u094b\u0902 \u0915\u094b \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0905\u0928\u0941\u0935\u0930\u094d\u0924\u0940 \u0905\u0927\u094d\u092f\u092f\u0928 (\u090f\u0932\u091f\u0940\u090f\u092b\u092f\u0942) \u092e\u0947\u0902 \u092d\u093e\u0917 \u0932\u0947\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0938\u0939\u092e\u0924 \u0939\u094b\u0928\u093e \u0939\u094b\u0917\u093e\u0964.<\/p>\n\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"who-is-not-eligible-for-pbgene-dmd-exclusion-fa-qs\"><strong>PBGENE-DMD \u0915\u0947 \u0932\u093f\u090f \u0915\u094c\u0928 \u092a\u093e\u0924\u094d\u0930 \u0928\u0939\u0940\u0902 \u0939\u0948? (\u0905\u092a\u0935\u093e\u0926 \u0938\u0902\u092c\u0902\u0927\u0940 \u0905\u0915\u094d\u0938\u0930 \u092a\u0942\u091b\u0947 \u091c\u093e\u0928\u0947 \u0935\u093e\u0932\u0947 \u092a\u094d\u0930\u0936\u094d\u0928)<\/strong><\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"\u274c-ineligible-patients\">\u274c <strong>\u0905\u092a\u093e\u0924\u094d\u0930 \u0930\u094b\u0917\u0940<\/strong><\/h3>\n\n\n<div id=\"rank-math-faq\" class=\"rank-math-block\">\n<div class=\"rank-math-list\">\n<div id=\"faq-question-1777393251291\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0915\u094d\u092f\u093e \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0930\u093e \u091a\u0941\u0915\u0947 \u092e\u0930\u0940\u091c \u0907\u0938\u092e\u0947\u0902 \u092d\u093e\u0917 \u0932\u0947 \u0938\u0915\u0924\u0947 \u0939\u0948\u0902?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u0928\u0939\u0940\u0902\u0964 \u0915\u093f\u0938\u0940 \u092d\u0940 \u0907\u0924\u093f\u0939\u093e\u0938 \u092e\u0947\u0902:<\/p>\n<p>\u2022 \u092a\u093f\u0924\u094d\u0930\u0948\u0915 \u0909\u092a\u091a\u093e\u0930<br \/>\u2022 \u091c\u0940\u0928 \u0938\u0902\u092a\u093e\u0926\u0928<br \/>\u2022 \u0915\u094b\u0936\u093f\u0915\u093e-\u0906\u0927\u093e\u0930\u093f\u0924 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u093e<br \/>\u2192 \u0907\u0938\u0915\u0947 \u092a\u0930\u093f\u0923\u093e\u092e\u0938\u094d\u0935\u0930\u0942\u092a \u0928\u093f\u0937\u094d\u0915\u093e\u0938\u0928 \u0939\u094b\u0924\u093e \u0939\u0948\u0964.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1777393281660\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u090f\u0915\u094d\u0938\u0949\u0928 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u092f\u093e \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u092c\u093e\u0930\u0947 \u092e\u0947\u0902 \u0915\u094d\u092f\u093e \u0916\u094d\u092f\u093e\u0932 \u0939\u0948?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u0928\u093f\u092e\u094d\u0928\u0932\u093f\u0916\u093f\u0924 \u0938\u094d\u0925\u093f\u0924\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u094b \u0905\u0927\u094d\u092f\u092f\u0928 \u0938\u0947 \u092c\u093e\u0939\u0930 \u0930\u0916\u093e \u091c\u093e\u090f\u0917\u093e:<\/p>\n<p>\u2022 \u092a\u093f\u091b\u0932\u0947 6 \u092e\u0939\u0940\u0928\u094b\u0902 \u0915\u0947 \u092d\u0940\u0924\u0930 \u0910\u0938\u0940 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u093e \u092a\u0926\u094d\u0927\u0924\u093f\u092f\u094b\u0902 \u0915\u093e \u0909\u092a\u092f\u094b\u0917 \u0915\u093f\u092f\u093e \u0939\u0948<br \/>\u2022 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u092c\u093e\u0926 \u0915\u092e \u0938\u0947 \u0915\u092e 5 \u0935\u0930\u094d\u0937\u094b\u0902 \u0924\u0915 \u0907\u0928\u094d\u0939\u0947\u0902 \u092c\u0902\u0926 \u0915\u0930\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0924\u0948\u092f\u093e\u0930 \u0928\u0939\u0940\u0902 \u0939\u0948\u0902<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1777393309651\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0915\u094d\u092f\u093e \u0905\u0928\u094d\u092f \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923\u094b\u0902 \u092e\u0947\u0902 \u0936\u093e\u092e\u093f\u0932 \u092e\u0930\u0940\u091c \u092a\u093e\u0924\u094d\u0930 \u0939\u0948\u0902?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u0928\u0939\u0940\u0902, \u091c\u092c \u0924\u0915 \u0915\u093f \u092a\u0930\u0940\u0915\u094d\u0937\u0923 \u0905\u0935\u0932\u094b\u0915\u0928 \u0938\u0902\u092c\u0902\u0927\u0940 (\u0917\u0948\u0930-\u0939\u0938\u094d\u0924\u0915\u094d\u0937\u0947\u092a\u0915\u093e\u0930\u0940) \u0928 \u0939\u094b\u0964.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1777393318621\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0915\u094d\u092f\u093e \u090f\u090f\u0935\u0940 \u090f\u0902\u091f\u0940\u092c\u0949\u0921\u0940-\u092a\u0949\u091c\u093f\u091f\u093f\u0935 \u092e\u0930\u0940\u091c \u092a\u093e\u0924\u094d\u0930 \u0939\u0948\u0902?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u0928\u0939\u0940\u0902\u0964 \u090f\u090f\u0935\u09409 \u090f\u0902\u091f\u0940\u092c\u0949\u0921\u0940 \u092a\u0930\u0940\u0915\u094d\u0937\u0923 \u0915\u093e \u0938\u0915\u093e\u0930\u093e\u0924\u094d\u092e\u0915 \u092a\u0930\u093f\u0923\u093e\u092e \u0906\u0928\u0947 \u092a\u0930 \u092a\u094d\u0930\u0924\u093f\u092d\u093e\u0917\u0940 \u092d\u093e\u0917 \u0928\u0939\u0940\u0902 \u0932\u0947 \u0938\u0915\u0924\u0947\u0964.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1777393328468\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0915\u094d\u092f\u093e \u0921\u0940\u090f\u092e\u0921\u0940 \u0915\u0947 \u0938\u092d\u0940 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u092a\u093e\u0924\u094d\u0930 \u0939\u0948\u0902?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u090f\u0915\u094d\u0938\u0949\u0928 1-44 \u0914\u0930\/\u092f\u093e \u090f\u0915\u094d\u0938\u0949\u0928 56-79 \u092e\u0947\u0902 \u0930\u094b\u0917\u091c\u0928\u0915 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0935\u093e\u0932\u0947 \u092a\u094d\u0930\u0924\u093f\u092d\u093e\u0917\u0940\u0964.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1777393410826\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0915\u093f\u0928 \u0939\u0943\u0926\u092f \u0938\u0902\u092c\u0902\u0927\u0940 \u0938\u094d\u0925\u093f\u0924\u093f\u092f\u094b\u0902 \u0915\u0947 \u0915\u093e\u0930\u0923 \u092e\u0930\u0940\u091c\u094b\u0902 \u0915\u094b \u0907\u0938 \u0905\u0927\u094d\u092f\u092f\u0928 \u0938\u0947 \u092c\u093e\u0939\u0930 \u0930\u0916\u093e \u091c\u093e\u0924\u093e \u0939\u0948?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u091c\u093f\u0928 \u092e\u0930\u0940\u091c\u094b\u0902 \u0915\u094b \u092f\u0947 \u0938\u092e\u0938\u094d\u092f\u093e \u0939\u0948:<\/p>\n<p>\u2022 <strong>\u092c\u093e\u090f\u0901 \u0928\u093f\u0932\u092f \u0907\u091c\u0947\u0915\u094d\u0936\u0928 \u0905\u0902\u0936 (LVEF) &lt; % 50<\/strong><br \/>\u2022 \u0939\u0943\u0926\u092f \u0930\u094b\u0917 \u0915\u0947 \u0938\u093e\u0915\u094d\u0937\u094d\u092f \u0915\u094b \u092c\u093e\u0939\u0930 \u0930\u0916\u093e \u0917\u092f\u093e \u0939\u0948\u0964.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1777393452605\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0915\u094d\u092f\u093e \u092a\u094d\u0930\u0924\u093f\u0930\u0915\u094d\u0937\u093e\u0926\u092e\u0928 \u0938\u0902\u092c\u0902\u0927\u0940 \u092e\u0924\u092d\u0947\u0926\u094b\u0902 \u0935\u093e\u0932\u0947 \u092e\u0930\u0940\u091c \u092d\u093e\u0917 \u0932\u0947 \u0938\u0915\u0924\u0947 \u0939\u0948\u0902?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u0928\u0939\u0940\u0902\u0964 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u0932\u093f\u090f \u092a\u094d\u0930\u0924\u093f\u0930\u0915\u094d\u0937\u093e \u092a\u094d\u0930\u0923\u093e\u0932\u0940 \u0915\u094b \u0926\u092c\u093e\u0928\u0947 \u0915\u0940 \u0906\u0935\u0936\u094d\u092f\u0915\u0924\u093e \u0939\u094b\u0924\u0940 \u0939\u0948, \u0907\u0938\u0932\u093f\u090f \u0910\u0938\u0947 \u092e\u0930\u0940\u091c \u0907\u0938\u0915\u0947 \u0932\u093f\u090f \u092a\u093e\u0924\u094d\u0930 \u0928\u0939\u0940\u0902 \u0939\u0948\u0902\u0964.<\/p>\n\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"pbgene-dmd-gene-editing-therapy-fa-qs-from-families\">PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940: \u092a\u0930\u093f\u0935\u093e\u0930\u094b\u0902 \u0926\u094d\u0935\u093e\u0930\u093e \u092a\u0942\u091b\u0947 \u091c\u093e\u0928\u0947 \u0935\u093e\u0932\u0947 \u0905\u0915\u094d\u0938\u0930 \u092a\u0942\u091b\u0947 \u091c\u093e\u0928\u0947 \u0935\u093e\u0932\u0947 \u092a\u094d\u0930\u0936\u094d\u0928<\/h2>\n\n\n<div id=\"rank-math-faq\" class=\"rank-math-block\">\n<div class=\"rank-math-list\">\n<div id=\"faq-question-1777394327672\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0915\u094d\u092f\u093e Elevidys \u0938\u0947 \u0909\u092a\u091a\u093e\u0930\u093f\u0924 \u092c\u091a\u094d\u091a\u093e PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u092d\u0940 \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0915\u0930 \u0938\u0915\u0924\u093e \u0939\u0948?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u0928\u0939\u0940\u0902\u0964 \u091c\u093f\u0928 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u094b \u092a\u0939\u0932\u0947 Elevidys \u091c\u0948\u0938\u0940 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u092e\u093f\u0932 \u091a\u0941\u0915\u0940 \u0939\u0948, \u0935\u0947 PBGENE-DMD \u0915\u0947 \u0932\u093f\u090f \u092a\u093e\u0924\u094d\u0930 \u0928\u0939\u0940\u0902 \u0939\u0948\u0902 \u0915\u094d\u092f\u094b\u0902\u0915\u093f \u092a\u0939\u0932\u0947 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u093e \u0905\u0928\u0941\u092d\u0935 \u0939\u094b\u0928\u093e \u090f\u0915 \u0905\u092a\u0935\u0930\u094d\u091c\u0928 \u092e\u093e\u0928\u0926\u0902\u0921 \u0939\u0948\u0964 \u0907\u0938\u0915\u0947 \u0905\u0932\u093e\u0935\u093e, AAV \u0935\u0948\u0915\u094d\u091f\u0930 \u0915\u0947 \u092a\u094d\u0930\u0924\u093f \u092a\u094d\u0930\u0924\u093f\u0930\u0915\u094d\u0937\u093e \u092a\u094d\u0930\u0924\u093f\u0915\u094d\u0930\u093f\u092f\u093e \u0938\u0941\u0930\u0915\u094d\u0937\u093e \u0914\u0930 \u092a\u094d\u0930\u092d\u093e\u0935\u0936\u0940\u0932\u0924\u093e \u0915\u094b \u0915\u092e \u0915\u0930 \u0938\u0915\u0924\u0940 \u0939\u0948, \u0914\u0930 \u0935\u0930\u094d\u0924\u092e\u093e\u0928 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u094d\u0930\u094b\u091f\u094b\u0915\u0949\u0932 \u092e\u0947\u0902 \u091c\u0940\u0928-\u0906\u0927\u093e\u0930\u093f\u0924 \u0909\u092a\u091a\u093e\u0930\u094b\u0902 \u0915\u094b \u0926\u094b\u0939\u0930\u093e\u0928\u0947 \u0938\u0947 \u0906\u092e\u0924\u094c\u0930 \u092a\u0930 \u092c\u091a\u093e \u091c\u093e\u0924\u093e \u0939\u0948\u0964.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1777394381372\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0915\u094d\u092f\u093e \u092e\u0947\u0930\u0947 \u092c\u0947\u091f\u0947 \u0915\u094b, \u091c\u093f\u0938\u0915\u0947 \u0921\u0940\u090f\u092e\u0921\u0940 \u091c\u0940\u0928 \u092e\u0947\u0902 \u090f\u0915\u094d\u0938\u0949\u0928 43 \u0915\u093e \u0935\u093f\u0932\u094b\u092a\u0928 \u0939\u0948, PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u092e\u093f\u0932 \u0938\u0915\u0924\u0940 \u0939\u0948?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u0928\u0902\u092c\u0930 PBGENE-DMD \u0915\u094b \u090f\u0915\u094d\u0938\u0949\u0928 45-55 \u0915\u0947 \u092d\u0940\u0924\u0930 \u092a\u0942\u0930\u0940 \u0924\u0930\u0939 \u0938\u0947 \u0938\u092e\u093e\u0939\u093f\u0924 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u0921\u093f\u091c\u093c\u093e\u0907\u0928 \u0915\u093f\u092f\u093e \u0917\u092f\u093e \u0939\u0948\u0964 \u090f\u0915\u094d\u0938\u0949\u0928 43 \u0915\u093e \u0935\u093f\u0932\u094b\u092a\u0928 \u0907\u0938 \u0932\u0915\u094d\u0937\u094d\u092f \u0938\u0940\u092e\u093e \u0938\u0947 \u092c\u093e\u0939\u0930 \u0906\u0924\u093e \u0939\u0948, \u0907\u0938\u0932\u093f\u090f \u092f\u0939 \u0935\u0930\u094d\u0924\u092e\u093e\u0928 \u092a\u093e\u0924\u094d\u0930\u0924\u093e \u092e\u093e\u0928\u0926\u0902\u0921\u094b\u0902 \u0915\u094b \u092a\u0942\u0930\u093e \u0928\u0939\u0940\u0902 \u0915\u0930\u0924\u093e \u0939\u0948\u0964.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1777394586338\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0915\u094d\u092f\u093e \u092e\u0947\u0930\u0947 \u092c\u0947\u091f\u0947 \u0915\u094b, \u091c\u093f\u0938\u0915\u0947 \u091c\u0940\u0928 \u092e\u0947\u0902 \u090f\u0915\u094d\u0938\u0949\u0928 48-55 \u0915\u093e \u0935\u093f\u0932\u094b\u092a\u0928 \u0939\u0948, PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u092e\u093f\u0932 \u0938\u0915\u0924\u0940 \u0939\u0948?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u0924\u0915\u0928\u0940\u0915\u0940 \u0930\u0942\u092a \u0938\u0947, \u0935\u0939 \u092a\u093e\u0924\u094d\u0930 \u0939\u094b \u0938\u0915\u0924\u093e \u0939\u0948 \u0915\u094d\u092f\u094b\u0902\u0915\u093f \u092f\u0939 \u0935\u093f\u0932\u094b\u092a\u0928 \u0932\u0915\u094d\u0937\u093f\u0924 \u090f\u0915\u094d\u0938\u0949\u0928 45-55 \u0938\u0940\u092e\u093e \u0915\u0947 \u092d\u0940\u0924\u0930 \u0906\u0924\u093e \u0939\u0948\u0964 \u0939\u093e\u0932\u093e\u0902\u0915\u093f, \u090f\u0915\u094d\u0938\u0949\u0928 48-55 \u0915\u0947 \u0935\u093f\u0932\u094b\u092a\u0928 \u0906\u092e\u0924\u094c\u0930 \u092a\u0930 \u092c\u0947\u0915\u0930 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0938\u0947 \u091c\u0941\u0921\u093c\u0947 \u0939\u094b\u0924\u0947 \u0939\u0948\u0902, \u091c\u093f\u0938\u0915\u093e \u0906\u092e\u0924\u094c\u0930 \u092a\u0930 \u0939\u0932\u094d\u0915\u093e \u092a\u094d\u0930\u092d\u093e\u0935 \u0939\u094b\u0924\u093e \u0939\u0948\u0964 \u0907\u0938\u0932\u093f\u090f, \u0907\u0938 \u0909\u092a\u0938\u092e\u0942\u0939 \u092e\u0947\u0902 Precision BioSciences \u0915\u0947 \u0905\u0928\u0941\u0938\u0902\u0927\u093e\u0928 \u0915\u094b \u0906\u0917\u0947 \u092c\u0922\u093c\u093e\u0928\u0947 \u0914\u0930 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u093f\u0923\u093e\u092e\u094b\u0902 \u0915\u094b \u092a\u094d\u0930\u0915\u093e\u0936\u093f\u0924 \u0915\u0930\u0928\u0947 \u0915\u0940 \u092a\u094d\u0930\u0924\u0940\u0915\u094d\u0937\u093e \u0915\u0930\u0928\u093e \u0938\u092e\u091d\u0926\u093e\u0930\u0940 \u0939\u094b\u0917\u0940\u0964 \u092c\u094b\u0928 \u092e\u0948\u0930\u094b \u0915\u0940 \u0935\u0943\u0926\u094d\u0927\u093f \u0926\u0930 \u0927\u0940\u092e\u0940 \u0939\u094b\u0928\u0947 \u0915\u0947 \u0915\u093e\u0930\u0923, \u0938\u093e\u0935\u0927\u093e\u0928\u0940\u092a\u0942\u0930\u094d\u0935\u0915 \u0928\u093f\u0917\u0930\u093e\u0928\u0940 \u0914\u0930 \u0927\u0948\u0930\u094d\u092f \u0930\u0916\u0928\u093e \u0909\u091a\u093f\u0924 \u0939\u0948\u0964.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1777395554562\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\"><strong>\u0915\u094d\u092f\u093e \u090f\u0915\u094d\u0938\u0949\u0928 45 \u0921\u093f\u0932\u0940\u0936\u0928 \u0935\u093e\u0932\u0947 \u0921\u0940\u090f\u092e\u0921\u0940 \u0938\u0947 \u092a\u0940\u0921\u093c\u093f\u0924 \u0932\u0921\u093c\u0915\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f PBGENE-DMD \u090f\u0915 \u0909\u092a\u091a\u093e\u0930 \u0935\u093f\u0915\u0932\u094d\u092a \u0939\u0948?<\/strong><\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u091c\u0940 \u0939\u093e\u0902\u0964 PBGENE-DMD \u0909\u0928 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u0921\u093f\u091c\u093c\u093e\u0907\u0928 \u0915\u093f\u092f\u093e \u0917\u092f\u093e \u0939\u0948 \u091c\u093f\u0928\u092e\u0947\u0902 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u092a\u0942\u0930\u0940 \u0924\u0930\u0939 \u0938\u0947 \u090f\u0915\u094d\u0938\u0949\u0928 45-55 \u0915\u0947 \u092d\u0940\u0924\u0930 \u0938\u092e\u093e\u0939\u093f\u0924 \u0939\u0948\u0902, \u0914\u0930 \u090f\u0915\u094d\u0938\u0949\u0928 45 \u0915\u093e \u0935\u093f\u0932\u094b\u092a\u0928 \u0907\u0938 \u0932\u0915\u094d\u0937\u093f\u0924 \u0938\u0940\u092e\u093e \u0915\u0947 \u0905\u0902\u0924\u0930\u094d\u0917\u0924 \u0906\u0924\u093e \u0939\u0948\u0964 \u0907\u0938\u0932\u093f\u090f, \u0907\u0938 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0935\u093e\u0932\u0947 \u0932\u0921\u093c\u0915\u0947 \u092a\u093e\u0924\u094d\u0930 \u0939\u094b \u0938\u0915\u0924\u0947 \u0939\u0948\u0902, \u092c\u0936\u0930\u094d\u0924\u0947 \u0935\u0947 \u0906\u092f\u0941, \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0938\u094d\u0925\u093f\u0924\u093f \u0914\u0930 \u0938\u092e\u0917\u094d\u0930 \u0938\u094d\u0935\u093e\u0938\u094d\u0925\u094d\u092f \u0938\u0902\u092c\u0902\u0927\u0940 \u0906\u0935\u0936\u094d\u092f\u0915\u0924\u093e\u0913\u0902 \u091c\u0948\u0938\u0947 \u0905\u0928\u094d\u092f \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092e\u093e\u0928\u0926\u0902\u0921\u094b\u0902 \u0915\u094b \u092d\u0940 \u092a\u0942\u0930\u093e \u0915\u0930\u0924\u0947 \u0939\u094b\u0902\u0964.<\/p>\n\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n\n\n<p class=\"wp-block-paragraph\">\u0914\u0930 \u0905\u0927\u093f\u0915 \u091c\u093e\u0928\u0947\u0902: <a href=\"https:\/\/precisionbiosciences.com\/arcus-genome-editing\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0906\u0930\u094d\u0915\u0938 \u091c\u0940\u0928\u094b\u092e \u0938\u0902\u092a\u093e\u0926\u0928<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"precision-bio-sciences-presents-new-preclinical-data-supporting-the-advancement-of-pbgene-dmd-into-clinic\">Precision BioSciences \u0928\u0947 PBGENE-DMD \u0915\u094b \u0915\u094d\u0932\u093f\u0928\u093f\u0915 \u092e\u0947\u0902 \u0906\u0917\u0947 \u092c\u0922\u093c\u093e\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0938\u092e\u0930\u094d\u0925\u0928 \u0926\u0947\u0928\u0947 \u0935\u093e\u0932\u0947 \u0928\u090f \u092a\u094d\u0930\u0940\u0915\u094d\u0932\u093f\u0928\u093f\u0915\u0932 \u0921\u0947\u091f\u093e \u092a\u094d\u0930\u0938\u094d\u0924\u0941\u0924 \u0915\u093f\u090f \u0939\u0948\u0902\u0964<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Precision BioSciences \u0928\u0947 \u0905\u092e\u0947\u0930\u093f\u0915\u0928 \u0938\u094b\u0938\u093e\u0907\u091f\u0940 \u0911\u092b \u091c\u0940\u0928 \u090f\u0902\u0921 \u0938\u0947\u0932 \u0925\u0947\u0930\u0947\u092a\u0940 (ASGCT) 2026 \u0915\u0940 \u0935\u093e\u0930\u094d\u0937\u093f\u0915 \u092c\u0948\u0920\u0915 \u092e\u0947\u0902 \u090f\u0915 \u092e\u094c\u0916\u093f\u0915 \u092a\u094d\u0930\u0938\u094d\u0924\u0941\u0924\u093f \u092e\u0947\u0902 \u0905\u092a\u0928\u0947 PBGENE-DMD \u0915\u093e\u0930\u094d\u092f\u0915\u094d\u0930\u092e \u0938\u0947 \u0928\u090f \u092a\u094d\u0930\u0940\u0915\u094d\u0932\u093f\u0928\u093f\u0915\u0932 \u0921\u0947\u091f\u093e \u0915\u0940 \u0918\u094b\u0937\u0923\u093e \u0915\u0940\u0964.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915 \u0915\u093f\u0936\u094b\u0930 \u0905\u0935\u0938\u094d\u0925\u093e \u0935\u093e\u0932\u0947 \u091a\u0942\u0939\u094b\u0902 \u092e\u0947\u0902 PBGENE-DMD \u0915\u0947 \u092a\u094d\u0930\u092f\u094b\u0917 \u0938\u0947 \u0915\u0902\u0915\u093e\u0932 \u0915\u0940 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u0940 \u092c\u0939\u093e\u0932\u0940 \u0926\u0947\u0930 \u0938\u0947 \u0915\u093f\u0936\u094b\u0930 \u0905\u0935\u0938\u094d\u0925\u093e \u0935\u093e\u0932\u0947 \u091a\u0942\u0939\u094b\u0902 \u0915\u0940 \u0924\u0941\u0932\u0928\u093e \u092e\u0947\u0902 3 \u0917\u0941\u0928\u093e \u0905\u0927\u093f\u0915 \u0914\u0930 \u0936\u094d\u0935\u0938\u0928 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u0940 \u092c\u0939\u093e\u0932\u0940 12 \u0917\u0941\u0928\u093e \u0905\u0927\u093f\u0915 \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0939\u0941\u0908, \u091c\u092c\u0915\u093f \u0938\u092e\u093e\u0928 \u0916\u0941\u0930\u093e\u0915 \u0938\u094d\u0924\u0930 \u092a\u0930 \u0910\u0938\u093e \u0928\u0939\u0940\u0902 \u0939\u0941\u0906\u0964.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0936\u094d\u0935\u0938\u0928 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u090a\u0924\u0915\u094b\u0902 \u092e\u0947\u0902 \u092e\u091c\u092c\u0942\u0924 \u092a\u094d\u0930\u092d\u093e\u0935\u0915\u093e\u0930\u093f\u0924\u093e \u0926\u0947\u0916\u0940 \u0917\u0908, \u091c\u094b % 5 \u0915\u0940 \u0905\u092a\u0947\u0915\u094d\u0937\u093f\u0924 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u092c\u0939\u093e\u0932\u0940 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u0940\u092f \u0938\u0940\u092e\u093e \u0938\u0947 \u0905\u0927\u093f\u0915 \u0925\u0940, \u091c\u093f\u0938\u092e\u0947\u0902 \u091a\u0942\u0939\u094b\u0902 \u0928\u0947 \u0921\u093e\u092f\u093e\u092b\u094d\u0930\u093e\u092e \u092e\u0947\u0902 % 12 \u0924\u0915 \u0914\u0930 \u0907\u0902\u091f\u0930\u0915\u094b\u0938\u094d\u091f\u0932 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 % 30 \u0924\u0915 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092c\u0939\u093e\u0932\u0940 \u0939\u093e\u0938\u093f\u0932 \u0915\u0940, \u092f\u0947 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u093e\u0902 \u0921\u0940\u090f\u092e\u0921\u0940 \u0935\u093e\u0932\u0947 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u0936\u094d\u0935\u0938\u0928 \u0935\u093f\u092b\u0932\u0924\u093e \u0915\u094b \u0930\u094b\u0915\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0915\u093e\u0930\u094d\u092f \u0915\u0930\u0924\u0940 \u0939\u0948\u0902\u0964.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u092e\u093e\u0928\u0935\u0940\u0915\u0943\u0924 \u0921\u0940\u090f\u092e\u0921\u0940 \u092e\u093e\u0909\u0938 \u092e\u0949\u0921\u0932 \u092e\u0947\u0902 \u0915\u093f\u090f \u0917\u090f \u0935\u093f\u0937 \u0935\u093f\u091c\u094d\u091e\u093e\u0928 \u0905\u0927\u094d\u092f\u092f\u0928 \u0938\u0947 \u092a\u0924\u093e \u091a\u0932\u093e \u0915\u093f PBGENE-DMD \u0909\u092a\u091a\u093e\u0930 \u0938\u0947 \u0915\u0908 \u0916\u0941\u0930\u093e\u0915 \u0938\u094d\u0924\u0930\u094b\u0902 \u092a\u0930 \u0938\u0940\u0930\u092e \u0915\u094d\u0930\u093f\u090f\u091f\u093f\u0928 \u0915\u093e\u0907\u0928\u0947\u091c \u092e\u0947\u0902 45% \u0938\u0947 \u0905\u0927\u093f\u0915 \u0915\u0940 \u0915\u092e\u0940 \u0906\u0908, \u0938\u093e\u0925 \u0939\u0940 \u0935\u093e\u0939\u0928-\u0909\u092a\u091a\u093e\u0930\u093f\u0924 \u0928\u093f\u092f\u0902\u0924\u094d\u0930\u0923\u094b\u0902 \u0915\u0940 \u0924\u0941\u0932\u0928\u093e \u092e\u0947\u0902 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0915\u0940 \u0935\u093f\u0915\u0943\u0924\u093f \u092e\u0947\u0902 \u0938\u0941\u0927\u093e\u0930 \u0939\u0941\u0906, \u091c\u094b \u0915\u093e\u0930\u094d\u092f\u0915\u094d\u0930\u092e \u0915\u0940 \u0938\u0941\u0930\u0915\u094d\u0937\u093e \u092a\u094d\u0930\u094b\u092b\u093c\u093e\u0907\u0932 \u0915\u093e \u0938\u092e\u0930\u094d\u0925\u0928 \u0915\u0930\u0924\u093e \u0939\u0948\u0964.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>PBGENE-DMD \u0928\u0947 \u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915-\u0915\u093f\u0936\u094b\u0930 \u091a\u0942\u0939\u094b\u0902 \u092e\u0947\u0902 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928-\u092a\u0949\u091c\u093f\u091f\u093f\u0935 \u092b\u093e\u0907\u092c\u0930 \u0915\u0947 \u0909\u091a\u094d\u091a \u0938\u094d\u0924\u0930 \u0915\u094b \u092a\u094d\u0930\u0947\u0930\u093f\u0924 \u0915\u093f\u092f\u093e, \u091c\u093f\u0938\u092e\u0947\u0902 \u0924\u0940\u0928 \u092e\u0939\u0940\u0928\u0947 \u092c\u093e\u0926 \u0915\u0902\u0915\u093e\u0932 \u0914\u0930 \u0936\u094d\u0935\u0938\u0928 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u090a\u0924\u0915\u094b\u0902 \u092e\u0947\u0902 \u0926\u0947\u0930 \u0938\u0947 \u0915\u093f\u0936\u094b\u0930 \u091a\u0942\u0939\u094b\u0902 \u0915\u0940 \u0924\u0941\u0932\u0928\u093e \u092e\u0947\u0902 \u0938\u094d\u0924\u0930 2-3 \u0917\u0941\u0928\u093e \u0905\u0927\u093f\u0915 \u0925\u093e, \u091c\u094b 70 \u092a\u094d\u0930\u0924\u093f\u0936\u0924 \u0924\u0915 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928-\u092a\u0949\u091c\u093f\u091f\u093f\u0935 \u092b\u093e\u0907\u092c\u0930 \u0924\u0915 \u092a\u0939\u0941\u0902\u091a \u0917\u092f\u093e\u0964.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915 \u0914\u0930 \u0926\u0947\u0930 \u0938\u0947 \u0915\u093f\u0936\u094b\u0930 \u0905\u0935\u0938\u094d\u0925\u093e \u0935\u093e\u0932\u0947 \u091a\u0942\u0939\u094b\u0902 \u0926\u094b\u0928\u094b\u0902 \u092e\u0947\u0902 \u0939\u0943\u0926\u092f \u0915\u0940 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u0938\u092e\u093e\u0928 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u0940\u092f \u092a\u094d\u0930\u092d\u093e\u0935\u0915\u093e\u0930\u093f\u0924\u093e \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0939\u0941\u0908\u0964.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0938\u0941\u0930\u0915\u094d\u0937\u093e \u0915\u0947 \u0905\u0932\u093e\u0935\u093e, PBGENE-DMD \u0928\u0947 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u0940 \u092c\u0939\u093e\u0932\u0940 \u0914\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928-\u092a\u0949\u091c\u093f\u091f\u093f\u0935 \u092e\u093e\u092f\u094b\u092b\u093e\u0907\u092c\u0930 \u0915\u0947 \u092e\u093e\u0927\u094d\u092f\u092e \u0938\u0947 \u0938\u092e\u092f \u0915\u0947 \u0938\u093e\u0925 \u0928\u093f\u0930\u0902\u0924\u0930 \u092a\u094d\u0930\u092d\u093e\u0935\u0915\u093e\u0930\u093f\u0924\u093e \u092a\u094d\u0930\u0926\u0930\u094d\u0936\u093f\u0924 \u0915\u0940 \u0939\u0948, \u091c\u094b \u091f\u093f\u0915\u093e\u090a \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0915\u093e\u0930\u094d\u092f\u092a\u094d\u0930\u0923\u093e\u0932\u0940 \u092e\u0947\u0902 \u092a\u0930\u093f\u0923\u0924 \u0939\u094b\u0924\u0940 \u0939\u0948\u0964.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0909\u092a\u091a\u093e\u0930\u093f\u0924 \u091a\u0942\u0939\u094b\u0902 \u0928\u0947 \u0938\u094d\u0935\u0938\u094d\u0925 \u091c\u093e\u0928\u0935\u0930\u094b\u0902 \u0915\u0940 \u0905\u0927\u093f\u0915\u0924\u092e \u092c\u0932 \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u0915\u094d\u0937\u092e\u0924\u093e \u0915\u093e &#039;92 \u092a\u094d\u0930\u0924\u093f\u0936\u0924&#039; \u0924\u0915 \u092c\u0928\u093e\u090f \u0930\u0916\u093e, \u091c\u092c\u0915\u093f \u0905\u0928\u0941\u092a\u091a\u093e\u0930\u093f\u0924, \u0930\u094b\u0917\u0917\u094d\u0930\u0938\u094d\u0924 \u091a\u0942\u0939\u094b\u0902 \u0915\u0940 \u092c\u0932 \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u0915\u094d\u0937\u092e\u0924\u093e \u092e\u0947\u0902 \u0909\u0924\u094d\u0924\u0930\u094b\u0924\u094d\u0924\u0930 \u0917\u093f\u0930\u093e\u0935\u091f \u0926\u0947\u0916\u0940 \u0917\u0908\u0964.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"final-thoughts\">\u0905\u0902\u0924\u093f\u092e \u0935\u093f\u091a\u093e\u0930<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">PBGENE-DMD \u091c\u0940\u0928 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u092e\u0947\u0902 \u0938\u094d\u0925\u093e\u092f\u0940 \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u0938\u0941\u0927\u093e\u0930 \u0915\u0940 \u0926\u093f\u0936\u093e \u092e\u0947\u0902 \u090f\u0915 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0915\u0926\u092e \u0939\u0948\u0964 \u092e\u0942\u0932 \u0915\u093e\u0930\u0923 \u0915\u094b \u0932\u0915\u094d\u0937\u093f\u0924 \u0915\u0930\u0915\u0947, \u092f\u0939 \u0932\u0917\u092d\u0917 \u092a\u0942\u0930\u094d\u0923 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0938\u094d\u0924\u0930 \u0915\u0940 \u092c\u0939\u093e\u0932\u0940 \u0915\u094b \u0938\u0902\u092d\u0935 \u092c\u0928\u093e \u0938\u0915\u0924\u0940 \u0939\u0948\u0964 \u0907\u0938\u0915\u093e \u090f\u0915 \u092c\u093e\u0930 \u0915\u093e \u0909\u092a\u091a\u093e\u0930 \u092c\u093e\u0930-\u092c\u093e\u0930 \u0926\u0940 \u091c\u093e\u0928\u0947 \u0935\u093e\u0932\u0940 \u0925\u0947\u0930\u0947\u092a\u0940 \u0938\u0947 \u092c\u0947\u0939\u0924\u0930 \u092a\u0930\u093f\u0923\u093e\u092e \u0926\u0947 \u0938\u0915\u0924\u093e \u0939\u0948\u0964 \u0939\u093e\u0932\u093e\u0902\u0915\u093f, \u0938\u0916\u094d\u0924 \u092a\u093e\u0924\u094d\u0930\u0924\u093e \u092e\u093e\u0928\u0926\u0902\u0921 \u0907\u0938\u0915\u0940 \u0909\u092a\u0932\u092c\u094d\u0927\u0924\u093e \u0915\u094b \u0938\u0940\u092e\u093f\u0924 \u0915\u0930\u0924\u0947 \u0939\u0948\u0902\u0964 \u0938\u0941\u0930\u0915\u094d\u0937\u093e, \u0938\u094d\u0925\u093e\u092f\u093f\u0924\u094d\u0935 \u0914\u0930 \u0935\u093e\u0938\u094d\u0924\u0935\u093f\u0915 \u092a\u0930\u093f\u0923\u093e\u092e\u094b\u0902 \u0915\u093e \u0905\u0927\u094d\u092f\u092f\u0928 \u0905\u092d\u0940 \u091c\u093e\u0930\u0940 \u0939\u0948\u0964 \u091a\u0932 \u0930\u0939\u0947 \u092a\u0930\u0940\u0915\u094d\u0937\u0923 \u0907\u0938\u0915\u0947 \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u094d\u0930\u092d\u093e\u0935 \u0915\u094b \u092a\u0930\u093f\u092d\u093e\u0937\u093f\u0924 \u0915\u0930\u0947\u0902\u0917\u0947\u0964.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0907\u0938 \u092a\u0947\u091c \u0915\u094b \u092b\u093c\u0949\u0932\u094b \u0915\u0930\u0947\u0902<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/hi\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u0915\u0947 \u0932\u093f\u090f \u0938\u092d\u0940 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>PBGENE-DMD Gene Editing Therapy is an investigational, first-in-class treatment developed by Precision BioSciences for Duchenne muscular dystrophy (DMD). Unlike traditional therapies, PBGENE-DMD uses a gene excision strategy powered by ARCUS gene editing technology. It is delivered via a single adeno-associated virus (AAV) vector that encodes two ARCUS nucleases designed to permanently edit the dystrophin gene. [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":7753,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[726,359,725,314,724],"class_list":["post-7752","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-arcus-gene-editing","tag-full-length-dystrophin","tag-gene-editing-therapy","tag-pbgene-dmd","tag-pbgene-dmd-therapy"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/7752","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=7752"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/7752\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media\/7753"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media?parent=7752"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/categories?post=7752"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/tags?post=7752"}],"curies":[{"name":"\u0921\u092c\u094d\u0932\u094d\u092f\u0942\u092a\u0940","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}