{"id":8291,"date":"2026-07-25T07:45:55","date_gmt":"2026-07-25T04:45:55","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8291"},"modified":"2026-07-25T07:45:57","modified_gmt":"2026-07-25T04:45:57","slug":"gen6050x-exon-50-skipping-base-editing-drug-one-year-results","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/hi\/gen6050x-exon-50-skipping-base-editing-drug-one-year-results\/","title":{"rendered":"GEN6050X \u090f\u0915\u094d\u0938\u0949\u0928 50 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u092c\u0947\u0938 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0926\u0935\u093e: \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0915\u0947 \u0932\u093f\u090f \u090f\u0915 \u0938\u093e\u0932 \u0915\u0947 \u092a\u0930\u093f\u0923\u093e\u092e \u0909\u0924\u094d\u0938\u093e\u0939\u091c\u0928\u0915 \u092a\u094d\u0930\u0917\u0924\u093f \u0926\u0930\u094d\u0936\u093e\u0924\u0947 \u0939\u0948\u0902\u0964"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>GenAssist \u0925\u0947\u0930\u0947\u092a\u094d\u092f\u0942\u091f\u093f\u0915\u094d\u0938 \u0926\u094d\u0935\u093e\u0930\u093e \u0935\u093f\u0915\u0938\u093f\u0924 GEN6050X \u090f\u0915\u094d\u0938\u0949\u0928 50 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u092c\u0947\u0938 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0926\u0935\u093e, \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 \u092e\u0942\u0932\u094d\u092f\u093e\u0902\u0915\u0928 \u0915\u0940 \u091c\u093e \u0930\u0939\u0940 \u092a\u0939\u0932\u0940 \u092c\u0947\u0938 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u092e\u0947\u0902 \u0938\u0947 \u090f\u0915 \u0915\u0947 \u0930\u0942\u092a \u092e\u0947\u0902 \u0924\u0947\u091c\u0940 \u0938\u0947 \u0927\u094d\u092f\u093e\u0928 \u0906\u0915\u0930\u094d\u0937\u093f\u0924 \u0915\u0930 \u0930\u0939\u0940 \u0939\u0948\u0964.<\/strong> \u092a\u093e\u0930\u0902\u092a\u0930\u093f\u0915 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u0935\u093f\u092a\u0930\u0940\u0924, \u091c\u094b \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u091c\u0940\u0928 \u0915\u093e \u0938\u0902\u0915\u094d\u0937\u093f\u092a\u094d\u0924 \u0930\u0942\u092a \u092a\u094d\u0930\u0926\u093e\u0928 \u0915\u0930\u0924\u0940 \u0939\u0948\u0902, GEN6050X \u0915\u094b \u0921\u0940\u090f\u0928\u090f \u0915\u094b \u0938\u091f\u0940\u0915 \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\u092f\u093e \u0917\u092f\u093e \u0939\u0948 \u0924\u093e\u0915\u093f \u090f\u0915\u094d\u0938\u0949\u0928 50 \u0915\u094b \u091b\u094b\u0921\u093c \u0926\u093f\u092f\u093e \u091c\u093e\u090f, \u091c\u093f\u0938\u0938\u0947 \u0930\u0940\u0921\u093f\u0902\u0917 \u092b\u094d\u0930\u0947\u092e \u0915\u094b \u092c\u0939\u093e\u0932 \u0915\u093f\u092f\u093e \u091c\u093e \u0938\u0915\u0947 \u0914\u0930 \u0936\u0930\u0940\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0915\u093e \u090f\u0915 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0930\u0942\u092a \u0909\u0924\u094d\u092a\u0928\u094d\u0928 \u0915\u0930 \u0938\u0915\u0947\u0964 \u0939\u093e\u0932\u093e\u0902\u0915\u093f \u092f\u0939 \u092a\u094d\u0930\u093e\u092f\u094b\u0917\u093f\u0915 \u0909\u092a\u091a\u093e\u0930 \u0905\u092d\u0940 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0935\u093f\u0915\u093e\u0938 \u0915\u0947 \u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915 \u091a\u0930\u0923 \u092e\u0947\u0902 \u0939\u0948, \u0939\u093e\u0932 \u0939\u0940 \u092e\u0947\u0902 \u091c\u093e\u0930\u0940 \u090f\u0915 \u0935\u0930\u094d\u0937 \u0915\u0947 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0921\u0947\u091f\u093e \u0938\u0947 \u092a\u0924\u093e \u091a\u0932\u0924\u093e \u0939\u0948 \u0915\u093f \u092f\u0939 \u0915\u0941\u091b \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u0938\u093e\u0930\u094d\u0925\u0915 \u0914\u0930 \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0932\u093e\u092d \u092a\u094d\u0930\u0926\u093e\u0928 \u0915\u0930 \u0938\u0915\u0924\u093e \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-the-gen-6050-x-exon-50-skipping-base-editing-drug\">GEN6050X \u090f\u0915\u094d\u0938\u0949\u0928 50 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u092c\u0947\u0938 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0926\u0935\u093e \u0915\u094d\u092f\u093e \u0939\u0948?<\/a><\/li><li><a href=\"#one-year-clinical-results-of-gen-6050-x\">GEN6050X \u0915\u0947 \u090f\u0915 \u0935\u0930\u094d\u0937 \u0915\u0947 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u093f\u0923\u093e\u092e<\/a><ul><li><a href=\"#motor-function-outcomes\">\u092e\u094b\u091f\u0930 \u092b\u093c\u0902\u0915\u094d\u0936\u0928 \u092a\u0930\u093f\u0923\u093e\u092e<\/a><\/li><li><a href=\"#cardiac-function-results\">\u0939\u0943\u0926\u092f \u0915\u093e\u0930\u094d\u092f\u092a\u094d\u0930\u0923\u093e\u0932\u0940 \u0915\u0947 \u092a\u0930\u093f\u0923\u093e\u092e<\/a><\/li><li><a href=\"#pulmonary-function-results\">\u092b\u0941\u092b\u094d\u092b\u0941\u0938\u0940\u092f \u0915\u093e\u0930\u094d\u092f\u092a\u094d\u0930\u0923\u093e\u0932\u0940 \u0915\u0947 \u092a\u0930\u093f\u0923\u093e\u092e<\/a><\/li><\/ul><\/li><li><a href=\"#safety-profile-of-the-gen-6050-x-exon-50-skipping-base-editing-drug\">GEN6050X \u090f\u0915\u094d\u0938\u0949\u0928 50 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u092c\u0947\u0938 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0926\u0935\u093e \u0915\u093e \u0938\u0941\u0930\u0915\u094d\u0937\u093e \u092a\u094d\u0930\u094b\u092b\u093e\u0907\u0932<\/a><\/li><li><a href=\"#limitations-of-the-current-clinical-study\">\u0935\u0930\u094d\u0924\u092e\u093e\u0928 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0905\u0927\u094d\u092f\u092f\u0928 \u0915\u0940 \u0938\u0940\u092e\u093e\u090f\u0901<\/a><\/li><li><a href=\"#future-outlook-for-gen-6050-x\">GEN6050X \u0915\u0947 \u0932\u093f\u090f \u092d\u0935\u093f\u0937\u094d\u092f \u0915\u0940 \u0938\u0902\u092d\u093e\u0935\u0928\u093e\u090f\u0902<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 id=\"what-is-the-gen-6050-x-exon-50-skipping-base-editing-drug\" class=\"wp-block-heading\">GEN6050X \u090f\u0915\u094d\u0938\u0949\u0928 50 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u092c\u0947\u0938 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0926\u0935\u093e \u0915\u094d\u092f\u093e \u0939\u0948?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>GEN6050X \u0915\u0940 \u091c\u093e\u0902\u091a \u0935\u0930\u094d\u0924\u092e\u093e\u0928 \u092e\u0947\u0902 \u091a\u0940\u0928 \u0915\u0947 \u092a\u0947\u0915\u093f\u0902\u0917 \u092f\u0942\u0928\u093f\u092f\u0928 \u092e\u0947\u0921\u093f\u0915\u0932 \u0915\u0949\u0932\u0947\u091c \u0905\u0938\u094d\u092a\u0924\u093e\u0932 \u092e\u0947\u0902 \u0906\u092f\u094b\u091c\u093f\u0924 \u090f\u0915 \u0905\u0928\u094d\u0935\u0947\u0937\u0915-\u0906\u0930\u0902\u092d\u093f\u0924 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923 (NCT06392724) \u092e\u0947\u0902 \u0915\u0940 \u091c\u093e \u0930\u0939\u0940 \u0939\u0948\u0964.<\/strong> \u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915 \u0905\u0927\u094d\u092f\u092f\u0928 \u092e\u0947\u0902 \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0938\u0947 \u092a\u0940\u0921\u093c\u093f\u0924 \u0924\u0940\u0928 \u091a\u0932\u0928\u0947-\u092b\u093f\u0930\u0928\u0947 \u092e\u0947\u0902 \u0938\u0915\u094d\u0937\u092e \u0932\u0921\u093c\u0915\u094b\u0902 \u0915\u094b \u0936\u093e\u092e\u093f\u0932 \u0915\u093f\u092f\u093e \u0917\u092f\u093e, \u091c\u093f\u0928\u0915\u0940 \u0909\u092e\u094d\u0930 6.5 \u0938\u0947 10 \u0935\u0930\u094d\u0937 \u0915\u0947 \u092c\u0940\u091a \u0925\u0940\u0964 \u092a\u094d\u0930\u0924\u094d\u092f\u0947\u0915 \u092a\u094d\u0930\u0924\u093f\u092d\u093e\u0917\u0940 \u0915\u094b 5 \u00d7 10\u00b9\u00b3 vg\/kg \u0915\u0940 \u0916\u0941\u0930\u093e\u0915 \u092a\u0930 GEN6050X \u0915\u093e \u090f\u0915 \u090f\u0915\u0932 \u0905\u0902\u0924\u0903\u0936\u093f\u0930\u093e \u091c\u0932\u0938\u0947\u0915 \u0926\u093f\u092f\u093e \u0917\u092f\u093e\u0964 \u0928\u0935\u0940\u0928\u0924\u092e \u092b\u0949\u0932\u094b-\u0905\u092a \u092e\u0947\u0902, \u0924\u0940\u0928\u094b\u0902 \u092a\u094d\u0930\u0924\u093f\u092d\u093e\u0917\u093f\u092f\u094b\u0902 \u0928\u0947 \u090f\u0915 \u0935\u0930\u094d\u0937 \u0915\u0940 \u0928\u093f\u0917\u0930\u093e\u0928\u0940 \u092a\u0942\u0930\u0940 \u0915\u0930 \u0932\u0940 \u0925\u0940, \u091c\u092c\u0915\u093f \u0926\u094b \u092a\u094d\u0930\u0924\u093f\u092d\u093e\u0917\u093f\u092f\u094b\u0902 \u0928\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u092c\u093e\u0926 18 \u092e\u0939\u0940\u0928\u0947 \u092a\u0942\u0930\u0947 \u0915\u0930 \u0932\u093f\u090f \u0925\u0947\u0964 \u0905\u0927\u093f\u0915 \u091c\u093e\u0928\u0947\u0902: <a href=\"https:\/\/dmdwarrior.com\/hi\/mutations-amenable-to-exon-50-skipping\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u0930\u094b\u0917 \u092e\u0947\u0902 \u090f\u0915\u094d\u0938\u0949\u0928 50 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u0932\u093f\u090f \u0909\u092a\u092f\u0941\u0915\u094d\u0924 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928<\/a><\/p>\n\n\n\n<h2 id=\"one-year-clinical-results-of-gen-6050-x\" class=\"wp-block-heading\">GEN6050X \u0915\u0947 \u090f\u0915 \u0935\u0930\u094d\u0937 \u0915\u0947 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u093f\u0923\u093e\u092e<\/h2>\n\n\n\n<h3 id=\"motor-function-outcomes\" class=\"wp-block-heading\">\u092e\u094b\u091f\u0930 \u092b\u093c\u0902\u0915\u094d\u0936\u0928 \u092a\u0930\u093f\u0923\u093e\u092e<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0907\u0938 \u0905\u0927\u094d\u092f\u092f\u0928 \u0915\u093e \u0938\u092c\u0938\u0947 \u0909\u0924\u094d\u0938\u093e\u0939\u091c\u0928\u0915 \u092a\u0939\u0932\u0942 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0928\u093f\u0937\u094d\u0915\u0930\u094d\u0937\u094b\u0902 \u0915\u0940 \u0938\u094d\u0925\u093e\u092f\u093f\u0924\u094d\u0935\u0924\u093e \u0939\u0948\u0964 \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, \u092e\u094b\u091f\u0930 \u0915\u093e\u0930\u094d\u092f\u0915\u094d\u0937\u092e\u0924\u093e \u0906\u092e \u0924\u094c\u0930 \u092a\u0930 \u0938\u092e\u092f \u0915\u0947 \u0938\u093e\u0925 \u0918\u091f\u0924\u0940 \u091c\u093e\u0924\u0940 \u0939\u0948\u0964.<\/strong> \u0939\u093e\u0932\u093e\u0902\u0915\u093f, \u090f\u0915 \u0935\u0930\u094d\u0937 \u092c\u093e\u0926, \u091c\u093e\u0902\u091a\u0915\u0930\u094d\u0924\u093e\u0913\u0902 \u0928\u0947 \u092c\u0924\u093e\u092f\u093e \u0915\u093f \u0928\u0949\u0930\u094d\u0925 \u0938\u094d\u091f\u093e\u0930 \u090f\u092e\u094d\u092c\u0941\u0932\u0947\u091f\u0930\u0940 \u0905\u0938\u0947\u0938\u092e\u0947\u0902\u091f (\u090f\u0928\u090f\u0938\u090f\u090f) \u0938\u094d\u0915\u094b\u0930 \u0914\u0930 \u092a\u0930\u092b\u0949\u0930\u094d\u092e\u0947\u0902\u0938 \u0911\u092b \u0905\u092a\u0930 \u0932\u093f\u092e\u094d\u092c 2.0 (\u092a\u0940\u092f\u0942\u090f\u0932 2.0) \u0938\u094d\u0915\u094b\u0930 \u0938\u094d\u0925\u093f\u0930 \u0930\u0939\u0947 \u092f\u093e \u0909\u0928\u092e\u0947\u0902 \u092e\u093e\u092e\u0942\u0932\u0940 \u0938\u0941\u0927\u093e\u0930 \u0926\u0947\u0916\u093e \u0917\u092f\u093e\u0964 \u092a\u094d\u0930\u0924\u093f\u092d\u093e\u0917\u093f\u092f\u094b\u0902 \u0915\u0940 \u0938\u0902\u0916\u094d\u092f\u093e \u092c\u0939\u0941\u0924 \u0915\u092e \u0939\u094b\u0928\u0947 \u0915\u0947 \u092c\u093e\u0935\u091c\u0942\u0926, \u0921\u0940\u090f\u092e\u0921\u0940 \u091c\u0948\u0938\u0940 \u092a\u094d\u0930\u0917\u0924\u093f\u0936\u0940\u0932 \u092c\u0940\u092e\u093e\u0930\u0940 \u092e\u0947\u0902 \u0932\u0902\u092c\u0947 \u0938\u092e\u092f \u0924\u0915 \u092e\u094b\u091f\u0930 \u092b\u0902\u0915\u094d\u0936\u0928 \u0915\u094b \u092c\u0928\u093e\u090f \u0930\u0916\u0928\u093e \u090f\u0915 \u0909\u0924\u094d\u0938\u093e\u0939\u091c\u0928\u0915 \u092a\u0930\u093f\u0923\u093e\u092e \u092e\u093e\u0928\u093e \u091c\u093e\u0924\u093e \u0939\u0948\u0964.<\/p>\n\n\n\n<h3 id=\"cardiac-function-results\" class=\"wp-block-heading\">\u0939\u0943\u0926\u092f \u0915\u093e\u0930\u094d\u092f\u092a\u094d\u0930\u0923\u093e\u0932\u0940 \u0915\u0947 \u092a\u0930\u093f\u0923\u093e\u092e<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u0930\u093f\u092a\u094b\u0930\u094d\u091f \u0915\u093f\u090f \u0917\u090f \u0932\u093e\u092d \u0915\u0947\u0935\u0932 \u0915\u0902\u0915\u093e\u0932 \u0915\u0940 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0924\u0915 \u0939\u0940 \u0938\u0940\u092e\u093f\u0924 \u0928\u0939\u0940\u0902 \u0925\u0947\u0964. <strong>\u0936\u094b\u0927\u0915\u0930\u094d\u0924\u093e\u0913\u0902 \u0928\u0947 \u0939\u0943\u0926\u092f \u0915\u093e\u0930\u094d\u092f\u092a\u094d\u0930\u0923\u093e\u0932\u0940 \u092e\u0947\u0902 \u0938\u0915\u093e\u0930\u093e\u0924\u094d\u092e\u0915 \u0930\u0941\u091d\u093e\u0928 \u092d\u0940 \u0926\u0947\u0916\u0947, \u091c\u094b \u0935\u093f\u0936\u0947\u0937 \u0930\u0942\u092a \u0938\u0947 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0939\u0948 \u0915\u094d\u092f\u094b\u0902\u0915\u093f \u0939\u0943\u0926\u092f \u0930\u094b\u0917 \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 \u091c\u091f\u093f\u0932\u0924\u093e\u0913\u0902 \u0915\u093e \u090f\u0915 \u092a\u094d\u0930\u092e\u0941\u0916 \u0915\u093e\u0930\u0923 \u0939\u0948\u0964.<\/strong> \u0905\u0927\u094d\u092f\u092f\u0928 \u092e\u0947\u0902 \u092a\u094d\u0930\u0924\u094d\u092f\u0947\u0915 \u0930\u094b\u0917\u0940 \u0915\u0947 \u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915 \u092e\u093e\u092a \u0915\u0940 \u0924\u0941\u0932\u0928\u093e \u092e\u0947\u0902 \u092c\u093e\u090f\u0902 \u0935\u0947\u0902\u091f\u094d\u0930\u093f\u0915\u0941\u0932\u0930 \u0907\u091c\u0947\u0915\u094d\u0936\u0928 \u0905\u0902\u0936 (LVEF) \u092e\u0947\u0902 \u0914\u0938\u0924\u0928 5.32 \u092a\u094d\u0930\u0924\u093f\u0936\u0924 \u0915\u093e \u0938\u0941\u0927\u093e\u0930 \u092a\u093e\u092f\u093e \u0917\u092f\u093e\u0964 \u092f\u0926\u093f \u092c\u0921\u093c\u0947 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923\u094b\u0902 \u092e\u0947\u0902 \u0907\u0938\u0915\u0940 \u092a\u0941\u0937\u094d\u091f\u093f \u0939\u094b \u091c\u093e\u0924\u0940 \u0939\u0948, \u0924\u094b \u0939\u0943\u0926\u092f \u0915\u093e\u0930\u094d\u092f\u092a\u094d\u0930\u0923\u093e\u0932\u0940 \u092e\u0947\u0902 \u092f\u0939 \u0938\u0941\u0927\u093e\u0930 \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0938\u094d\u0935\u093e\u0938\u094d\u0925\u094d\u092f \u0915\u0947 \u0932\u093f\u090f \u092b\u093e\u092f\u0926\u0947\u092e\u0902\u0926 \u0938\u093e\u092c\u093f\u0924 \u0939\u094b \u0938\u0915\u0924\u093e \u0939\u0948\u0964.<\/p>\n\n\n\n<h3 id=\"pulmonary-function-results\" class=\"wp-block-heading\">\u092b\u0941\u092b\u094d\u092b\u0941\u0938\u0940\u092f \u0915\u093e\u0930\u094d\u092f\u092a\u094d\u0930\u0923\u093e\u0932\u0940 \u0915\u0947 \u092a\u0930\u093f\u0923\u093e\u092e<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u092c\u093e\u0926 \u092b\u0947\u092b\u0921\u093c\u094b\u0902 \u0915\u0940 \u0915\u093e\u0930\u094d\u092f\u0915\u094d\u0937\u092e\u0924\u093e \u092e\u0947\u0902 \u092d\u0940 \u0938\u0941\u0927\u093e\u0930 \u0926\u093f\u0916\u093e\u0908 \u0926\u093f\u092f\u093e\u0964 \u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915 \u0938\u094d\u0925\u093f\u0924\u093f \u0915\u0940 \u0924\u0941\u0932\u0928\u093e \u092e\u0947\u0902, \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0940 \u092b\u094b\u0930\u094d\u0938\u094d\u0921 \u0935\u093e\u0907\u091f\u0932 \u0915\u0948\u092a\u0947\u0938\u093f\u091f\u0940 (FVC) \u092e\u0947\u0902 \u0914\u0938\u0924\u0928 11.72 \u092a\u094d\u0930\u0924\u093f\u0936\u0924 \u0914\u0930 \u092a\u0940\u0915 \u090f\u0915\u094d\u0938\u092a\u093f\u0930\u0947\u091f\u0930\u0940 \u092b\u094d\u0932\u094b (PEF) \u092e\u0947\u0902 25.29 \u092a\u094d\u0930\u0924\u093f\u0936\u0924 \u0915\u093e \u0938\u0941\u0927\u093e\u0930 \u0939\u0941\u0906\u0964.<\/strong> \u092f\u0947 \u092e\u093e\u092a \u092b\u0947\u092b\u0921\u093c\u094b\u0902 \u0915\u0940 \u092e\u091c\u092c\u0942\u0924\u0940 \u0914\u0930 \u0938\u093e\u0902\u0938 \u0932\u0947\u0928\u0947 \u0915\u0940 \u0915\u094d\u0937\u092e\u0924\u093e \u0915\u093e \u0906\u0915\u0932\u0928 \u0915\u0930\u0924\u0947 \u0939\u0948\u0902, \u091c\u094b \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u0930\u094b\u0917 \u092c\u0922\u093c\u0928\u0947 \u0915\u0947 \u0938\u093e\u0925 \u0927\u0940\u0930\u0947-\u0927\u0940\u0930\u0947 \u0915\u092e \u0939\u094b\u0924\u0940 \u091c\u093e\u0924\u0940 \u0939\u0948\u0902\u0964 \u0936\u094b\u0927\u0915\u0930\u094d\u0924\u093e\u0913\u0902 \u0928\u0947 \u0938\u0941\u091d\u093e\u0935 \u0926\u093f\u092f\u093e \u0915\u093f \u092f\u0947 \u0928\u093f\u0937\u094d\u0915\u0930\u094d\u0937 \u0938\u0902\u0915\u0947\u0924 \u0926\u0947 \u0938\u0915\u0924\u0947 \u0939\u0948\u0902 \u0915\u093f GEN6050X \u0915\u0902\u0915\u093e\u0932 \u0915\u0940 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0938\u0947 \u092a\u0930\u0947 \u092d\u0940 \u0932\u093e\u092d \u092a\u094d\u0930\u0926\u093e\u0928 \u0915\u0930\u0924\u093e \u0939\u0948, \u0939\u093e\u0932\u093e\u0902\u0915\u093f \u0907\u0928 \u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915 \u0928\u093f\u0937\u094d\u0915\u0930\u094d\u0937\u094b\u0902 \u0915\u0940 \u092a\u0941\u0937\u094d\u091f\u093f \u0915\u0947 \u0932\u093f\u090f \u092c\u0921\u093c\u0947 \u0905\u0927\u094d\u092f\u092f\u0928\u094b\u0902 \u0915\u0940 \u0906\u0935\u0936\u094d\u092f\u0915\u0924\u093e \u0939\u0948\u0964.<\/p>\n\n\n\n<h2 id=\"safety-profile-of-the-gen-6050-x-exon-50-skipping-base-editing-drug\" class=\"wp-block-heading\">GEN6050X \u090f\u0915\u094d\u0938\u0949\u0928 50 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u092c\u0947\u0938 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0926\u0935\u093e \u0915\u093e \u0938\u0941\u0930\u0915\u094d\u0937\u093e \u092a\u094d\u0930\u094b\u092b\u093e\u0907\u0932<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0915\u093f\u0938\u0940 \u092d\u0940 \u092a\u094d\u0930\u093e\u092f\u094b\u0917\u093f\u0915 \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u093e \u0915\u0947 \u0932\u093f\u090f \u0938\u0941\u0930\u0915\u094d\u0937\u093e \u0938\u092c\u0938\u0947 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u092a\u094d\u0930\u0936\u094d\u0928\u094b\u0902 \u092e\u0947\u0902 \u0938\u0947 \u090f\u0915 \u092c\u0928\u0940 \u0939\u0941\u0908 \u0939\u0948\u0964. <strong>\u090f\u0915 \u0935\u0930\u094d\u0937 \u0915\u0947 \u0905\u0928\u0941\u0935\u0930\u094d\u0924\u0940 \u0905\u0927\u094d\u092f\u092f\u0928 \u0915\u0947 \u0905\u0928\u0941\u0938\u093e\u0930, GEN6050X \u0906\u092e\u0924\u094c\u0930 \u092a\u0930 \u0905\u091a\u094d\u091b\u0940 \u0924\u0930\u0939 \u0938\u0947 \u0938\u0939\u0928 \u0915\u093f\u092f\u093e \u0917\u092f\u093e\u0964 \u0915\u0941\u091b \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u094b \u0916\u0941\u0930\u093e\u0915 \u0932\u0947\u0928\u0947 \u0915\u0947 \u0924\u0941\u0930\u0902\u0924 \u092c\u093e\u0926 \u0917\u0902\u092d\u0940\u0930 \u092a\u094d\u0930\u0924\u093f\u0915\u0942\u0932 \u0918\u091f\u0928\u093e\u0913\u0902 \u0915\u093e \u0905\u0928\u0941\u092d\u0935 \u0939\u0941\u0906, \u0932\u0947\u0915\u093f\u0928 \u0936\u094b\u0927\u0915\u0930\u094d\u0924\u093e\u0913\u0902 \u0928\u0947 \u092c\u0924\u093e\u092f\u093e \u0915\u093f \u092f\u0947 \u0918\u091f\u0928\u093e\u090f\u0902 \u0915\u094d\u0937\u0923\u093f\u0915, \u092a\u094d\u0930\u092c\u0902\u0927\u0928\u0940\u092f \u0925\u0940\u0902 \u0914\u0930 \u092c\u093f\u0928\u093e \u0915\u093f\u0938\u0940 \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u092a\u0930\u093f\u0923\u093e\u092e \u0915\u0947 \u092a\u0942\u0930\u0940 \u0924\u0930\u0939 \u0938\u0947 \u0920\u0940\u0915 \u0939\u094b \u0917\u0908\u0902\u0964.<\/strong> \u0935\u093f\u0938\u094d\u0924\u093e\u0930\u093f\u0924 \u0905\u0928\u0941\u0935\u0930\u094d\u0924\u0940 \u0905\u0927\u094d\u092f\u092f\u0928 \u0915\u0947 \u0926\u094c\u0930\u093e\u0928, \u0936\u094b\u0927\u0915\u0930\u094d\u0924\u093e\u0913\u0902 \u0928\u0947 \u0909\u092a\u091a\u093e\u0930\u093f\u0924 \u092a\u094d\u0930\u0924\u093f\u092d\u093e\u0917\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u0909\u092a\u091a\u093e\u0930 \u0938\u0947 \u0938\u0902\u092c\u0902\u0927\u093f\u0924 \u0915\u094b\u0908 \u092d\u0940 \u0928\u092f\u093e \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u0915\u0940\u092f \u0930\u0942\u092a \u0938\u0947 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0932\u0915\u094d\u0937\u0923 \u092f\u093e \u092a\u094d\u0930\u092f\u094b\u0917\u0936\u093e\u0932\u093e \u0938\u0902\u092c\u0902\u0927\u0940 \u0905\u0938\u093e\u092e\u093e\u0928\u094d\u092f\u0924\u093e\u090f\u0902 \u0928\u0939\u0940\u0902 \u092a\u093e\u0908\u0902\u0964.<\/p>\n\n\n\n<h2 id=\"limitations-of-the-current-clinical-study\" class=\"wp-block-heading\">\u0935\u0930\u094d\u0924\u092e\u093e\u0928 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0905\u0927\u094d\u092f\u092f\u0928 \u0915\u0940 \u0938\u0940\u092e\u093e\u090f\u0901<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0939\u093e\u0932\u093e\u0902\u0915\u093f \u092f\u0947 \u0928\u093f\u0937\u094d\u0915\u0930\u094d\u0937 \u0906\u0936\u093e\u091c\u0928\u0915 \u0939\u0948\u0902, \u0932\u0947\u0915\u093f\u0928 \u0907\u0928\u0915\u093e \u0938\u093e\u0935\u0927\u093e\u0928\u0940\u092a\u0942\u0930\u094d\u0935\u0915 \u0935\u093f\u0936\u094d\u0932\u0947\u0937\u0923 \u0915\u0930\u0928\u093e \u0906\u0935\u0936\u094d\u092f\u0915 \u0939\u0948\u0964 \u0935\u0930\u094d\u0924\u092e\u093e\u0928 \u0905\u0927\u094d\u092f\u092f\u0928 \u092e\u0947\u0902 \u0915\u0947\u0935\u0932 \u0924\u0940\u0928 \u092e\u0930\u0940\u091c\u093c \u0936\u093e\u092e\u093f\u0932 \u0939\u0948\u0902, \u0907\u0938\u0932\u093f\u090f \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u092a\u094d\u0930\u092d\u093e\u0935\u0936\u0940\u0932\u0924\u093e \u092f\u093e \u0938\u0941\u0930\u0915\u094d\u0937\u093e \u0915\u0947 \u092c\u093e\u0930\u0947 \u092e\u0947\u0902 \u0915\u094b\u0908 \u0928\u093f\u0936\u094d\u091a\u093f\u0924 \u0928\u093f\u0937\u094d\u0915\u0930\u094d\u0937 \u0928\u093f\u0915\u093e\u0932\u0928\u093e \u0938\u0902\u092d\u0935 \u0928\u0939\u0940\u0902 \u0939\u0948\u0964 GEN6050X \u090f\u0915\u094d\u0938\u0949\u0928 50 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u092c\u0947\u0938 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0926\u0935\u093e \u0915\u094b \u0935\u094d\u092f\u093e\u092a\u0915 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0909\u092a\u092f\u094b\u0917 \u0915\u0947 \u0932\u093f\u090f \u0935\u093f\u091a\u093e\u0930 \u0915\u0930\u0928\u0947 \u0938\u0947 \u092a\u0939\u0932\u0947 \u0905\u0927\u093f\u0915 \u092a\u094d\u0930\u0924\u093f\u092d\u093e\u0917\u093f\u092f\u094b\u0902 \u0914\u0930 \u0932\u0902\u092c\u0940 \u0905\u0935\u0932\u094b\u0915\u0928 \u0905\u0935\u0927\u093f \u0935\u093e\u0932\u0947 \u092c\u0921\u093c\u0947 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923\u094b\u0902 \u0915\u0940 \u0906\u0935\u0936\u094d\u092f\u0915\u0924\u093e \u0939\u094b\u0917\u0940\u0964.<\/p>\n\n\n\n<h2 id=\"future-outlook-for-gen-6050-x\" class=\"wp-block-heading\">GEN6050X \u0915\u0947 \u0932\u093f\u090f \u092d\u0935\u093f\u0937\u094d\u092f \u0915\u0940 \u0938\u0902\u092d\u093e\u0935\u0928\u093e\u090f\u0902<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u092f\u0926\u093f \u092d\u0935\u093f\u0937\u094d\u092f \u0915\u0947 \u0905\u0927\u094d\u092f\u092f\u0928 \u0907\u0928 \u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915 \u092a\u0930\u093f\u0923\u093e\u092e\u094b\u0902 \u0915\u0940 \u092a\u0941\u0937\u094d\u091f\u093f \u0915\u0930\u0924\u0947 \u0939\u0948\u0902, \u0924\u094b GEN6050X \u090f\u0915\u094d\u0938\u0949\u0928 50 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u092c\u0947\u0938 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0926\u0935\u093e \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0915\u0947 \u0932\u093f\u090f \u0938\u091f\u0940\u0915 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u093e \u092e\u0947\u0902 \u090f\u0915 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u092a\u094d\u0930\u0917\u0924\u093f \u0915\u093e \u092a\u094d\u0930\u0924\u093f\u0928\u093f\u0927\u093f\u0924\u094d\u0935 \u0915\u0930 \u0938\u0915\u0924\u0940 \u0939\u0948\u0964.<\/strong> \u091c\u0940\u0928 \u0915\u0947 \u090f\u0915 \u0939\u093f\u0938\u094d\u0938\u0947 \u0915\u094b \u092c\u0926\u0932\u0928\u0947 \u0915\u0947 \u092c\u091c\u093e\u092f \u092c\u0947\u0938 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0915\u0947 \u092e\u093e\u0927\u094d\u092f\u092e \u0938\u0947 \u0905\u0902\u0924\u0930\u094d\u0928\u093f\u0939\u093f\u0924 \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0915\u094b \u0938\u0940\u0927\u0947 \u0920\u0940\u0915 \u0915\u0930\u0915\u0947, \u092f\u0939 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923 \u0921\u0940\u090f\u092e\u0921\u0940 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u0932\u093f\u090f \u0928\u0908 \u0938\u0902\u092d\u093e\u0935\u0928\u093e\u090f\u0902 \u0916\u094b\u0932 \u0938\u0915\u0924\u093e \u0939\u0948 \u091c\u094b \u090f\u0915\u094d\u0938\u0949\u0928 50 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0915\u0947 \u0932\u093f\u090f \u0909\u092a\u092f\u0941\u0915\u094d\u0924 \u0939\u0948\u0902\u0964. <strong>\u0939\u093e\u0932\u093e\u0902\u0915\u093f \u092f\u0939 \u0925\u0947\u0930\u0947\u092a\u0940 \u0905\u092d\u0940 \u092d\u0940 \u091c\u093e\u0902\u091a \u0915\u0947 \u0926\u093e\u092f\u0930\u0947 \u092e\u0947\u0902 \u0939\u0948, \u0932\u0947\u0915\u093f\u0928 \u090f\u0915 \u0938\u093e\u0932 \u0915\u0947 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0906\u0902\u0915\u0921\u093c\u094b\u0902 \u0938\u0947 \u0938\u093e\u0935\u0927\u093e\u0928\u0940\u092a\u0942\u0930\u094d\u0935\u0915 \u0906\u0936\u093e\u0935\u093e\u0926 \u092e\u093f\u0932\u0924\u093e \u0939\u0948 \u0915\u093f \u092c\u0947\u0938 \u090f\u0921\u093f\u091f\u093f\u0902\u0917 \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u0915\u0947 \u0909\u092a\u091a\u093e\u0930\u094b\u0902 \u0915\u0947 \u092c\u0922\u093c\u0924\u0947 \u092a\u0930\u093f\u0926\u0943\u0936\u094d\u092f \u092e\u0947\u0902 \u090f\u0915 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u092f\u094b\u0917\u0926\u093e\u0928 \u092c\u0928 \u0938\u0915\u0924\u093e \u0939\u0948\u0964.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0905\u092d\u0940 \u0926\u0947\u0916\u0947\u0902<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMDWarrioR \u0915\u094d\u0932\u093f\u0928\u093f\u0915\u0932 \u091f\u094d\u0930\u093e\u092f\u0932\u094d\u0938 \u0939\u092c<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>The GEN6050X exon 50 skipping base editing drug, developed by GenAssist Therapeutics, is attracting growing attention as one of the first base editing therapies being evaluated for Duchenne muscular dystrophy (DMD). Unlike conventional gene therapies that deliver a shortened version of the dystrophin gene, GEN6050X is designed to precisely edit DNA so that exon 50 [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":8293,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[123,278,279,475,280,178,277,276],"class_list":["post-8291","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-china","tag-exon-50","tag-exon-50-skipping","tag-exon-50-skipping-therapy","tag-exon-50-skipping-treatment","tag-exon-skipping-therapies","tag-gen6050x","tag-genassist"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/8291","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=8291"}],"version-history":[{"count":2,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/8291\/revisions"}],"predecessor-version":[{"id":8294,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/8291\/revisions\/8294"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media\/8293"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media?parent=8291"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/categories?post=8291"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/tags?post=8291"}],"curies":[{"name":"\u0921\u092c\u094d\u0932\u094d\u092f\u0942\u092a\u0940","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}