{"id":2261,"date":"2026-04-10T14:26:46","date_gmt":"2026-04-10T11:26:46","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=2261"},"modified":"2026-04-24T13:04:53","modified_gmt":"2026-04-24T10:04:53","slug":"new-gene-therapies-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/hi\/new-gene-therapies-for-duchenne-muscular-dystrophy\/","title":{"rendered":"\u0921\u094d\u092f\u0942\u091a\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\u0902\u092d\u093e\u0935\u093f\u0924 \u0906\u0917\u093e\u092e\u0940 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>\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 \u0938\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u093e \u092a\u0930\u093f\u0926\u0943\u0936\u094d\u092f \u0924\u0947\u091c\u0940 \u0938\u0947 \u0935\u093f\u0915\u0938\u093f\u0924 \u0939\u094b \u0930\u0939\u093e \u0939\u0948, \u091c\u093f\u0938\u0938\u0947 \u0907\u0938 \u0935\u093f\u0928\u093e\u0936\u0915\u093e\u0930\u0940 \u0928\u094d\u092f\u0942\u0930\u094b\u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0935\u093f\u0915\u093e\u0930 \u0938\u0947 \u092a\u094d\u0930\u092d\u093e\u0935\u093f\u0924 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0914\u0930 \u092a\u0930\u093f\u0935\u093e\u0930\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u0928\u0908 \u0909\u092e\u094d\u092e\u0940\u0926 \u091c\u0917 \u0930\u0939\u0940 \u0939\u0948\u0964.<\/strong> \u091c\u093f\u0928 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923\u094b\u0902 \u092a\u0930 \u0938\u092c\u0938\u0947 \u0905\u0927\u093f\u0915 \u0927\u094d\u092f\u093e\u0928 \u0926\u093f\u092f\u093e \u091c\u093e \u0930\u0939\u093e \u0939\u0948 \u0909\u0928\u092e\u0947\u0902 Solid Biosciences SGT-003, RegenxBio RGX-202 \u0914\u0930 Genethon GNT-0004 \u0936\u093e\u092e\u093f\u0932 \u0939\u0948\u0902, \u091c\u094b \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u093e \u092e\u0947\u0902 \u0928\u0935\u093e\u091a\u093e\u0930 \u0915\u0940 \u0905\u0917\u0932\u0940 \u0932\u0939\u0930 \u0915\u093e \u092a\u094d\u0930\u0924\u093f\u0928\u093f\u0927\u093f\u0924\u094d\u0935 \u0915\u0930\u0924\u0947 \u0939\u0948\u0902\u0964. <strong>\u092f\u0947 \u0909\u092d\u0930\u0924\u0940 \u0939\u0941\u0908 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u093e \u092a\u0926\u094d\u0927\u0924\u093f\u092f\u093e\u0901\u2014\u091c\u093f\u0928\u094d\u0939\u0947\u0902 \u0905\u0917\u0932\u0940 \u092a\u0940\u0922\u093c\u0940 \u0915\u0940 \u0921\u0940\u090f\u092e\u0921\u0940 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940, \u092e\u093e\u0907\u0915\u094d\u0930\u094b-\u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u091c\u0940\u0928 \u0938\u094d\u0925\u093e\u0928\u093e\u0902\u0924\u0930\u0923 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923 \u0914\u0930 \u090f\u090f\u0935\u0940-\u0906\u0927\u093e\u0930\u093f\u0924 \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u0909\u092a\u091a\u093e\u0930 \u092d\u0940 \u0915\u0939\u093e \u091c\u093e\u0924\u093e \u0939\u0948\u2014\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 \u092e\u0942\u0932 \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u0915\u093e\u0930\u0923 \u0915\u094b \u0926\u0942\u0930 \u0915\u0930\u0928\u0947 \u0915\u093e \u0932\u0915\u094d\u0937\u094d\u092f \u0930\u0916\u0924\u0940 \u0939\u0948\u0902\u0964.<\/strong> \u0932\u0915\u094d\u0937\u0923\u094b\u0902 \u0915\u0947 \u092a\u094d\u0930\u092c\u0902\u0927\u0928 \u092a\u0930 \u0927\u094d\u092f\u093e\u0928 \u0915\u0947\u0902\u0926\u094d\u0930\u093f\u0924 \u0915\u0930\u0928\u0947 \u0935\u093e\u0932\u0947 \u092a\u093e\u0930\u0902\u092a\u0930\u093f\u0915 \u0909\u092a\u091a\u093e\u0930\u094b\u0902 \u0915\u0947 \u0935\u093f\u092a\u0930\u0940\u0924, \u092f\u0947 \u0909\u0928\u094d\u0928\u0924 \u0930\u0923\u0928\u0940\u0924\u093f\u092f\u093e\u0901 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \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\u0928\u0947 \u0914\u0930 \u0938\u0902\u092d\u093e\u0935\u093f\u0924 \u0930\u0942\u092a \u0938\u0947 \u092e\u094c\u0932\u093f\u0915 \u0938\u094d\u0924\u0930 \u092a\u0930 \u0930\u094b\u0917 \u0915\u0940 \u092a\u094d\u0930\u0917\u0924\u093f \u0915\u094b \u092c\u0926\u0932\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0921\u093f\u091c\u093c\u093e\u0907\u0928 \u0915\u0940 \u0917\u0908 \u0939\u0948\u0902\u0964.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\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 \u091c\u0940\u0928 \u092e\u0947\u0902 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0915\u0947 \u0915\u093e\u0930\u0923 \u0939\u094b\u0924\u0940 \u0939\u0948, \u091c\u093f\u0938\u0938\u0947 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0928\u093e\u092e\u0915 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u0940 \u0915\u092e\u0940 \u0939\u094b \u091c\u093e\u0924\u0940 \u0939\u0948, \u091c\u094b \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0915\u0940 \u0905\u0916\u0902\u0921\u0924\u093e \u092c\u0928\u093e\u090f \u0930\u0916\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0906\u0935\u0936\u094d\u092f\u0915 \u0939\u0948\u0964 \u0938\u092e\u092f \u0915\u0947 \u0938\u093e\u0925, \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940\u092f \u0924\u0902\u0924\u0941 \u0915\u094d\u0937\u0940\u0923 \u0939\u094b \u091c\u093e\u0924\u0947 \u0939\u0948\u0902, \u091c\u093f\u0938\u0915\u0947 \u092a\u0930\u093f\u0923\u093e\u092e\u0938\u094d\u0935\u0930\u0942\u092a \u092a\u094d\u0930\u0917\u0924\u093f\u0936\u0940\u0932 \u0915\u092e\u091c\u094b\u0930\u0940, \u091a\u0932\u0928\u0947-\u092b\u093f\u0930\u0928\u0947 \u092e\u0947\u0902 \u0905\u0938\u092e\u0930\u094d\u0925\u0924\u093e, \u0936\u094d\u0935\u0938\u0928 \u0938\u0902\u092c\u0902\u0927\u0940 \u091c\u091f\u093f\u0932\u0924\u093e\u090f\u0902 \u0914\u0930 \u0939\u0943\u0926\u092f \u0938\u0902\u092c\u0902\u0927\u0940 \u0935\u093f\u0915\u093e\u0930 \u0939\u094b \u091c\u093e\u0924\u0947 \u0939\u0948\u0902\u0964. <strong>\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\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u093e \u0935\u093f\u0915\u093e\u0938 \u090f\u0915 \u092a\u094d\u0930\u0924\u093f\u092e\u093e\u0928 \u092c\u0926\u0932\u093e\u0935 \u0915\u093e \u092a\u094d\u0930\u0924\u093f\u0928\u093f\u0927\u093f\u0924\u094d\u0935 \u0915\u0930\u0924\u093e \u0939\u0948 - \u0938\u0939\u093e\u092f\u0915 \u0926\u0947\u0916\u092d\u093e\u0932 \u0938\u0947 \u0939\u091f\u0915\u0930 \u0930\u094b\u0917-\u0938\u0902\u0936\u094b\u0927\u0915 \u0939\u0938\u094d\u0924\u0915\u094d\u0937\u0947\u092a\u094b\u0902 \u0915\u0940 \u0913\u0930 \u091c\u094b \u091c\u0940\u0935\u0928\u0915\u093e\u0932 \u0914\u0930 \u091c\u0940\u0935\u0928 \u0915\u0940 \u0917\u0941\u0923\u0935\u0924\u094d\u0924\u093e \u0926\u094b\u0928\u094b\u0902 \u0915\u094b \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0930\u0942\u092a \u0938\u0947 \u092c\u0922\u093c\u093e \u0938\u0915\u0924\u0947 \u0939\u0948\u0902\u0964.<\/strong><\/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=\"#the-scientific-foundation-behind-gene-therapy-in-dmd\">\u0921\u0940\u090f\u092e\u0921\u0940 \u092e\u0947\u0902 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u092a\u0940\u091b\u0947 \u0915\u093e \u0935\u0948\u091c\u094d\u091e\u093e\u0928\u093f\u0915 \u0906\u0927\u093e\u0930<\/a><\/li><li><a href=\"#solid-biosciences-sgt-003\">1\u091f\u0940\u092a\u09401\u091f\u0940<\/a><ul><\/ul><\/li><li><a href=\"#regenx-bio-rgx-202\">\u0930\u0947\u091c\u0947\u0928\u0915\u094d\u0938\u092c\u093e\u092f\u094b RGX-202<\/a><ul><\/ul><\/li><li><a href=\"#genethon-gnt-0004\">\u091c\u0940\u0928\u0947\u0925\u0949\u0928 GNT-0004<\/a><ul><\/ul><\/li><li><a href=\"#emerging-gene-therapy-research-in-china-and-russia\">\u091a\u0940\u0928 \u0914\u0930 \u0930\u0942\u0938 \u092e\u0947\u0902 \u0909\u092d\u0930\u0924\u0947 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0905\u0928\u0941\u0938\u0902\u0927\u093e\u0928<\/a><ul><li><a href=\"#china\">\u091a\u0940\u0928<\/a><\/li><li><a href=\"#russia\">\u0930\u0942\u0938<\/a><\/li><li><a href=\"#a-hopeful-outlook\">\u090f\u0915 \u0906\u0936\u093e\u0935\u093e\u0926\u0940 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923<\/a><\/li><\/ul><\/li><li><a href=\"#challenges-and-limitations-of-gene-therapy-in-dmd\">\u092e\u0927\u0941\u092e\u0947\u0939 \u0930\u094b\u0917 \u092e\u0947\u0902 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0940 \u091a\u0941\u0928\u094c\u0924\u093f\u092f\u093e\u0901 \u0914\u0930 \u0938\u0940\u092e\u093e\u090f\u0901<\/a><ul><\/ul><\/li><li><a href=\"#future-directions-and-innovations\">\u092d\u0935\u093f\u0937\u094d\u092f \u0915\u0940 \u0926\u093f\u0936\u093e\u090f\u0901 \u0914\u0930 \u0928\u0935\u093e\u091a\u093e\u0930<\/a><\/li><li><a href=\"#conclusion\">\u0928\u093f\u0937\u094d\u0915\u0930\u094d\u0937<\/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=\"the-scientific-foundation-behind-gene-therapy-in-dmd\">\u0921\u0940\u090f\u092e\u0921\u0940 \u092e\u0947\u0902 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u092a\u0940\u091b\u0947 \u0915\u093e \u0935\u0948\u091c\u094d\u091e\u093e\u0928\u093f\u0915 \u0906\u0927\u093e\u0930<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0921\u0940\u090f\u092e\u0921\u0940 \u0915\u0947 \u0932\u093f\u090f \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u092e\u0941\u0916\u094d\u092f \u0930\u0942\u092a \u0938\u0947 \u090f\u0921\u0947\u0928\u094b-\u090f\u0938\u094b\u0938\u093f\u090f\u091f\u0947\u0921 \u0935\u093e\u092f\u0930\u0938 (\u090f\u090f\u0935\u0940) \u0935\u0948\u0915\u094d\u091f\u0930 \u092a\u0930 \u0928\u093f\u0930\u094d\u092d\u0930 \u0915\u0930\u0924\u0940 \u0939\u0948, \u091c\u094b \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u091c\u0940\u0928 \u0915\u0947 \u090f\u0915 \u091b\u094b\u091f\u0947 \u0932\u0947\u0915\u093f\u0928 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0938\u0902\u0938\u094d\u0915\u0930\u0923 \u0915\u094b \u0935\u093f\u0924\u0930\u093f\u0924 \u0915\u0930\u0924\u0947 \u0939\u0948\u0902, \u091c\u093f\u0938\u0947 \u0906\u092e\u0924\u094c\u0930 \u092a\u0930 \u092e\u093e\u0907\u0915\u094d\u0930\u094b-\u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0915\u0939\u093e \u091c\u093e\u0924\u093e \u0939\u0948\u0964.<\/strong> \u0915\u094d\u092f\u094b\u0902\u0915\u093f \u0938\u0902\u092a\u0942\u0930\u094d\u0923 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u091c\u0940\u0928 \u0935\u093e\u092f\u0930\u0932 \u0935\u0948\u0915\u094d\u091f\u0930 \u092e\u0947\u0902 \u0938\u092e\u093e\u0939\u093f\u0924 \u0939\u094b\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u092c\u0939\u0941\u0924 \u092c\u0921\u093c\u093e \u0939\u0948, \u0907\u0938\u0932\u093f\u090f \u0936\u094b\u0927\u0915\u0930\u094d\u0924\u093e\u0913\u0902 \u0928\u0947 \u0910\u0938\u0947 \u0938\u0902\u0915\u094d\u0937\u093f\u092a\u094d\u0924 \u0938\u0902\u0938\u094d\u0915\u0930\u0923 \u0924\u0948\u092f\u093e\u0930 \u0915\u093f\u090f \u0939\u0948\u0902 \u091c\u094b \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0921\u094b\u092e\u0947\u0928 \u0915\u094b \u092c\u0930\u0915\u0930\u093e\u0930 \u0930\u0916\u0924\u0947 \u0939\u0948\u0902\u0964.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u092a\u094d\u0930\u092e\u0941\u0916 \u0924\u0902\u0924\u094d\u0930\u094b\u0902 \u092e\u0947\u0902 \u0928\u093f\u092e\u094d\u0928\u0932\u093f\u0916\u093f\u0924 \u0936\u093e\u092e\u093f\u0932 \u0939\u0948\u0902:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u092e\u093e\u0907\u0915\u094d\u0930\u094b-\u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u091c\u0940\u0928 \u0915\u094b \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0915\u094b\u0936\u093f\u0915\u093e\u0913\u0902 \u092e\u0947\u0902 \u092a\u0939\u0941\u0902\u091a\u093e\u0928\u093e<\/li>\n\n\n\n<li>\u0938\u093e\u0930\u094d\u0915\u094b\u0932\u0947\u092e\u0932 \u0938\u094d\u0925\u093f\u0930\u0924\u093e \u0915\u0940 \u092c\u0939\u093e\u0932\u0940<\/li>\n\n\n\n<li>\u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0915\u0947 \u0915\u094d\u0937\u0930\u0923 \u0914\u0930 \u0938\u0942\u091c\u0928 \u092e\u0947\u0902 \u0915\u092e\u0940<\/li>\n\n\n\n<li>\u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0915\u0940 \u0924\u093e\u0915\u0924 \u0914\u0930 \u0915\u093e\u0930\u094d\u092f\u0915\u094d\u0937\u092e\u0924\u093e \u092e\u0947\u0902 \u0938\u0941\u0927\u093e\u0930<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u0914\u0930 \u0905\u0927\u093f\u0915 \u091c\u093e\u0928\u0947\u0902: <a href=\"https:\/\/dmdwarrior.com\/hi\/micro-dystrophin\/\" target=\"_blank\" data-type=\"post\" data-id=\"3807\" rel=\"noreferrer noopener\">\u092e\u093e\u0907\u0915\u094d\u0930\u094b-\u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0915\u094d\u092f\u093e \u0939\u0948?<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0939\u093e\u0932\u093e\u0902\u0915\u093f \u0915\u0941\u091b \u091a\u0941\u0928\u094c\u0924\u093f\u092f\u093e\u0902 \u0905\u092d\u0940 \u092d\u0940 \u092e\u094c\u091c\u0942\u0926 \u0939\u0948\u0902\u2014\u091c\u0948\u0938\u0947 \u0915\u093f \u092a\u094d\u0930\u0924\u093f\u0930\u0915\u094d\u0937\u093e \u092a\u094d\u0930\u0924\u093f\u0915\u094d\u0930\u093f\u092f\u093e, \u0935\u0947\u0915\u094d\u091f\u0930 \u0935\u093f\u0924\u0930\u0923 \u0926\u0915\u094d\u0937\u0924\u093e \u0914\u0930 \u0905\u092d\u093f\u0935\u094d\u092f\u0915\u094d\u0924\u093f \u0915\u0940 \u0938\u094d\u0925\u093e\u092f\u093f\u0924\u094d\u0935\u2014\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\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u092e\u0947\u0902 \u092a\u094d\u0930\u0917\u0924\u093f \u0924\u0947\u091c\u0940 \u0938\u0947 \u0939\u094b \u0930\u0939\u0940 \u0939\u0948\u0964 \u0914\u0930 \u092a\u0922\u093c\u0947\u0902: <a href=\"https:\/\/dmdwarrior.com\/hi\/biomarkers-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\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 \u092c\u093e\u092f\u094b\u092e\u093e\u0930\u094d\u0915\u0930<\/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=\"solid-biosciences-sgt-003\">1\u091f\u0940\u092a\u09401\u091f\u0940<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"overview-of-sgt-003\">\u090f\u0938\u091c\u0940\u091f\u0940-003 \u0915\u093e \u0905\u0935\u0932\u094b\u0915\u0928<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Solid Biosciences&#039; SGT-003 \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\u092c\u0938\u0947 \u0909\u0928\u094d\u0928\u0924 \u0938\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u092e\u0947\u0902 \u0938\u0947 \u090f\u0915 \u0939\u0948, \u091c\u093f\u0938\u0947 \u0905\u0917\u0932\u0940 \u092a\u0940\u0922\u093c\u0940 \u0915\u0940 AAV-\u0906\u0927\u093e\u0930\u093f\u0924 \u092e\u093e\u0907\u0915\u094d\u0930\u094b-\u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u0930\u0942\u092a \u092e\u0947\u0902 \u0921\u093f\u091c\u093c\u093e\u0907\u0928 \u0915\u093f\u092f\u093e \u0917\u092f\u093e \u0939\u0948\u0964 \u0907\u0938\u092e\u0947\u0902 \u092a\u0939\u0932\u0947 \u0915\u0947 \u0915\u0902\u0938\u094d\u091f\u094d\u0930\u0915\u094d\u091f\u094d\u0938 \u0915\u0940 \u0924\u0941\u0932\u0928\u093e \u092e\u0947\u0902 \u0915\u0908 \u0938\u0941\u0927\u093e\u0930 \u0936\u093e\u092e\u093f\u0932 \u0939\u0948\u0902, \u091c\u093f\u0928\u092e\u0947\u0902 \u092c\u0947\u0939\u0924\u0930 \u0915\u093e\u0930\u094d\u0921\u093f\u092f\u0915 \u091f\u093e\u0930\u0917\u0947\u091f\u093f\u0902\u0917 \u0914\u0930 \u0905\u0928\u0941\u0915\u0942\u0932\u093f\u0924 \u090f\u0915\u094d\u0938\u092a\u094d\u0930\u0947\u0936\u0928 \u0936\u093e\u092e\u093f\u0932 \u0939\u0948\u0902\u0964.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"clinical-development-and-findings\">\u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0935\u093f\u0915\u093e\u0938 \u0914\u0930 \u0928\u093f\u0937\u094d\u0915\u0930\u094d\u0937<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">SGT-003 \u092a\u0930\u0940\u0915\u094d\u0937\u0923\u094b\u0902 \u0938\u0947 \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0921\u0947\u091f\u093e \u0938\u0947 \u0928\u093f\u092e\u094d\u0928\u0932\u093f\u0916\u093f\u0924 \u0938\u0902\u0915\u0947\u0924 \u092e\u093f\u0932\u0924\u0947 \u0939\u0948\u0902:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0915\u0902\u0915\u093e\u0932 \u0914\u0930 \u0939\u0943\u0926\u092f \u0915\u0940 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u092e\u093e\u0907\u0915\u094d\u0930\u094b-\u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0915\u0940 \u092a\u094d\u0930\u092c\u0932 \u0905\u092d\u093f\u0935\u094d\u092f\u0915\u094d\u0924\u093f<\/li>\n\n\n\n<li>\u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0915\u0940 \u0905\u0916\u0902\u0921\u0924\u093e \u0938\u0947 \u091c\u0941\u0921\u093c\u0947 \u092c\u0947\u0939\u0924\u0930 \u092c\u093e\u092f\u094b\u092e\u093e\u0930\u094d\u0915\u0930<\/li>\n\n\n\n<li>\u092a\u0939\u0932\u0947 \u0915\u0947 \u0909\u092e\u094d\u092e\u0940\u0926\u0935\u093e\u0930\u094b\u0902 \u0915\u0940 \u0924\u0941\u0932\u0928\u093e \u092e\u0947\u0902 \u092c\u0947\u0939\u0924\u0930 \u0938\u0941\u0930\u0915\u094d\u0937\u093e \u092a\u094d\u0930\u094b\u092b\u093c\u093e\u0907\u0932<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u0917\u094c\u0930\u0924\u0932\u092c \u0939\u0948 \u0915\u093f \u090f\u0938\u091c\u0940\u091f\u0940-003 \u090f\u0915 \u0928\u090f \u0915\u0948\u092a\u094d\u0938\u093f\u0921 \u0915\u093e \u0909\u092a\u092f\u094b\u0917 \u0915\u0930\u0924\u093e \u0939\u0948 \u091c\u093f\u0938\u0947 \u0921\u093f\u0932\u0940\u0935\u0930\u0940 \u0926\u0915\u094d\u0937\u0924\u093e \u092e\u0947\u0902 \u0938\u0941\u0927\u093e\u0930 \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, \u0935\u093f\u0936\u0947\u0937 \u0930\u0942\u092a \u0938\u0947 \u0939\u0943\u0926\u092f \u0915\u0947 \u090a\u0924\u0915\u094b\u0902 \u0924\u0915 - \u092f\u0939 \u090f\u0915 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0915\u093e\u0930\u0915 \u0939\u0948 \u0915\u094d\u092f\u094b\u0902\u0915\u093f \u0915\u093e\u0930\u094d\u0921\u093f\u092f\u094b\u092e\u093e\u092f\u094b\u092a\u0948\u0925\u0940 \u0921\u0940\u090f\u092e\u0921\u0940 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u092e\u0943\u0924\u094d\u092f\u0941 \u0915\u093e \u090f\u0915 \u092a\u094d\u0930\u092e\u0941\u0916 \u0915\u093e\u0930\u0923 \u0939\u0948\u0964.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0914\u0930 \u0905\u0927\u093f\u0915 \u091c\u093e\u0928\u0947\u0902: <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT06138639\" target=\"_blank\" rel=\"noopener\">\u090f\u0938\u091c\u0940\u091f\u0940-003 \u0915\u0947 \u092c\u093e\u0930\u0947 \u092e\u0947\u0902<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"strategic-importance\">\u0930\u0923\u0928\u0940\u0924\u093f\u0915 \u092e\u0939\u0924\u094d\u0935<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\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\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u0935\u094d\u092f\u093e\u092a\u0915 \u0915\u094d\u0937\u0947\u0924\u094d\u0930 \u092e\u0947\u0902 \u090f\u0938\u091c\u0940\u091f\u0940-003 \u0915\u093e \u092e\u0939\u0924\u094d\u0935 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u092e\u0947\u0902 \u092a\u0939\u0932\u0947 \u0915\u0940 \u0938\u0940\u092e\u093e\u0913\u0902 \u0915\u094b \u0926\u0942\u0930 \u0915\u0930\u0928\u0947 \u0915\u0947 \u0907\u0938\u0915\u0947 \u092a\u094d\u0930\u092f\u093e\u0938 \u092e\u0947\u0902 \u0928\u093f\u0939\u093f\u0924 \u0939\u0948, \u0935\u093f\u0936\u0947\u0937 \u0930\u0942\u092a \u0938\u0947:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0905\u092a\u0930\u094d\u092f\u093e\u092a\u094d\u0924 \u0939\u0943\u0926\u092f \u0935\u093f\u0924\u0930\u0923<\/li>\n\n\n\n<li>\u092a\u094d\u0930\u0924\u093f\u0930\u0915\u094d\u0937\u093e \u0938\u0902\u092c\u0902\u0927\u0940 \u092a\u094d\u0930\u0924\u093f\u0915\u0942\u0932 \u0918\u091f\u0928\u093e\u090f\u0902<\/li>\n\n\n\n<li>\u0905\u092d\u093f\u0935\u094d\u092f\u0915\u094d\u0924\u093f \u0938\u094d\u0924\u0930\u094b\u0902 \u092e\u0947\u0902 \u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928\u0936\u0940\u0932\u0924\u093e<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u092f\u0926\u093f \u092f\u0939 \u0938\u092b\u0932 \u0939\u094b\u0924\u093e \u0939\u0948, \u0924\u094b \u090f\u0938\u091c\u0940\u091f\u0940-003 \u0932\u0917\u093e\u0924\u093e\u0930 \u0914\u0930 \u091f\u093f\u0915\u093e\u090a \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u0940\u092f \u092a\u0930\u093f\u0923\u093e\u092e \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0915\u0930\u0928\u0947 \u0915\u0940 \u0926\u093f\u0936\u093e \u092e\u0947\u0902 \u090f\u0915 \u092c\u0921\u093c\u093e \u0915\u0926\u092e \u0938\u093e\u092c\u093f\u0924 \u0939\u094b \u0938\u0915\u0924\u093e \u0939\u0948\u0964.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0914\u0930 \u0905\u0927\u093f\u0915 \u091c\u093e\u0928\u0947\u0902:<\/strong>&nbsp;<a href=\"https:\/\/dmdwarrior.com\/hi\/inspire-duchenne-sgt-003-clinical-trial-update\/\" target=\"_blank\" rel=\"noreferrer noopener\">SGT-003 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940: INSPIRE DUCHENNE \u091a\u0930\u0923 3 \u0905\u0927\u094d\u092f\u092f\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=\"regenx-bio-rgx-202\">\u0930\u0947\u091c\u0947\u0928\u0915\u094d\u0938\u092c\u093e\u092f\u094b RGX-202<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"overview-of-rgx-202\">\u0906\u0930\u091c\u0940\u090f\u0915\u094d\u0938-202 \u0915\u093e \u0905\u0935\u0932\u094b\u0915\u0928<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0930\u0940\u091c\u0947\u0928\u0915\u094d\u0938\u092c\u093e\u092f\u094b \u0926\u094d\u0935\u093e\u0930\u093e \u0935\u093f\u0915\u0938\u093f\u0924 \u0906\u0930\u091c\u0940\u090f\u0915\u094d\u0938-202, \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\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u092e\u0947\u0902 \u090f\u0915 \u0914\u0930 \u0906\u0936\u093e\u091c\u0928\u0915 \u0909\u092e\u094d\u092e\u0940\u0926\u0935\u093e\u0930 \u0939\u0948\u0964.<\/strong> \u092f\u0939 \u0915\u0902\u092a\u0928\u0940 \u0915\u0947 \u0938\u094d\u0935\u093e\u092e\u093f\u0924\u094d\u0935 \u0935\u093e\u0932\u0947 NAV\u00ae AAV \u0935\u0947\u0915\u094d\u091f\u0930 \u092a\u094d\u0932\u0947\u091f\u092b\u0949\u0930\u094d\u092e \u0915\u093e \u0932\u093e\u092d \u0909\u0920\u093e\u0915\u0930 \u090f\u0915 \u0928\u0935\u0940\u0928 \u092e\u093e\u0907\u0915\u094d\u0930\u094b-\u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0938\u0902\u0930\u091a\u0928\u093e \u0915\u094b \u0935\u093f\u0924\u0930\u093f\u0924 \u0915\u0930\u0924\u093e \u0939\u0948\u0964.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"unique-features\">\u0905\u0928\u0928\u094d\u092f \u0935\u093f\u0936\u0947\u0937\u0924\u093e\u090f\u0902<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">RGX-202 \u0915\u0940 \u0916\u093e\u0938\u093f\u092f\u0924\u0947\u0902 \u0907\u0938 \u092a\u094d\u0930\u0915\u093e\u0930 \u0939\u0948\u0902:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0915\u0947 \u0938\u0940-\u091f\u0930\u094d\u092e\u093f\u0928\u0932 \u0921\u094b\u092e\u0947\u0928 \u0915\u094b \u0936\u093e\u092e\u093f\u0932 \u0915\u0930\u0928\u0947 \u0938\u0947 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0915\u0947 \u0915\u093e\u0930\u094d\u092f \u092e\u0947\u0902 \u0938\u0941\u0927\u093e\u0930 \u0939\u094b \u0938\u0915\u0924\u093e \u0939\u0948\u0964<\/li>\n\n\n\n<li>\u092c\u0947\u0939\u0924\u0930 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0938\u094d\u0925\u093e\u0928\u0940\u092f\u0915\u0930\u0923 \u0915\u0947 \u0932\u093f\u090f \u0905\u0928\u0941\u0915\u0942\u0932\u093f\u0924 \u091c\u0940\u0928 \u0938\u0902\u0930\u091a\u0928\u093e<\/li>\n\n\n\n<li>\u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u090a\u0924\u0915 \u092e\u0947\u0902 \u0909\u091a\u094d\u091a \u091f\u094d\u0930\u093e\u0902\u0938\u0921\u0915\u094d\u0936\u0928 \u0926\u0915\u094d\u0937\u0924\u093e<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u0907\u0928 \u0935\u093f\u0936\u0947\u0937\u0924\u093e\u0913\u0902 \u0915\u094b \u092a\u0939\u0932\u0947 \u0915\u0940 \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\u0940 \u0924\u0941\u0932\u0928\u093e \u092e\u0947\u0902 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0915\u093e \u0905\u0927\u093f\u0915 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0930\u0942\u092a \u0938\u0947 \u092a\u0942\u0930\u094d\u0923 \u0938\u0902\u0938\u094d\u0915\u0930\u0923 \u0924\u0948\u092f\u093e\u0930 \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\u0964.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0914\u0930 \u0905\u0927\u093f\u0915 \u091c\u093e\u0928\u0947\u0902: <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT05693142\" target=\"_blank\" rel=\"noreferrer noopener\">\u0906\u0930\u091c\u0940\u090f\u0915\u094d\u0938-202 \u0915\u0947 \u092c\u093e\u0930\u0947 \u092e\u0947\u0902<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"clinical-insights\">\u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0905\u0902\u0924\u0930\u094d\u0926\u0943\u0937\u094d\u091f\u093f<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u091a\u0932 \u0930\u0939\u0947 \u092a\u0930\u0940\u0915\u094d\u0937\u0923\u094b\u0902 \u0938\u0947 \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915 \u0906\u0902\u0915\u0921\u093c\u094b\u0902 \u0938\u0947 \u0938\u0902\u0915\u0947\u0924 \u092e\u093f\u0932\u0924\u093e \u0939\u0948:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0938\u092e\u0942\u0939\u094b\u0902 \u092e\u0947\u0902 \u091c\u0940\u0928 \u0905\u092d\u093f\u0935\u094d\u092f\u0915\u094d\u0924\u093f \u0915\u093e \u0909\u091a\u094d\u091a \u0938\u094d\u0924\u0930<\/li>\n\n\n\n<li>\u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915 \u091a\u0930\u0923 \u0915\u0947 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u092a\u0930\u093f\u0923\u093e\u092e\u094b\u0902 \u0915\u094b \u092a\u094d\u0930\u094b\u0924\u094d\u0938\u093e\u0939\u093f\u0924 \u0915\u0930\u0928\u093e<\/li>\n\n\n\n<li>\u092e\u093e\u0928\u0915 \u092a\u094d\u0930\u0924\u093f\u0930\u0915\u094d\u0937\u093e \u0926\u092e\u0928 \u092a\u094d\u0930\u094b\u091f\u094b\u0915\u0949\u0932 \u0915\u0947 \u0938\u093e\u0925 \u092a\u094d\u0930\u092c\u0902\u0927\u0928\u0940\u092f \u0938\u0941\u0930\u0915\u094d\u0937\u093e \u092a\u094d\u0930\u094b\u092b\u093c\u093e\u0907\u0932<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0906\u0930\u091c\u0940\u090f\u0915\u094d\u0938-202 \u0915\u094b \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0915\u0947 \u0932\u093f\u090f \u0938\u092c\u0938\u0947 \u0924\u0915\u0928\u0940\u0915\u0940 \u0930\u0942\u092a \u0938\u0947 \u092a\u0930\u093f\u0937\u094d\u0915\u0943\u0924 \u0938\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u092e\u0947\u0902 \u0938\u0947 \u090f\u0915 \u092e\u093e\u0928\u093e \u091c\u093e\u0924\u093e \u0939\u0948, \u091c\u093f\u0938\u0915\u0947 \u0921\u093f\u091c\u093e\u0907\u0928 \u0915\u093e \u0938\u092e\u0930\u094d\u0925\u0928 \u0915\u0930\u0928\u0947 \u0935\u093e\u0932\u093e \u092e\u091c\u092c\u0942\u0924 \u0935\u0948\u091c\u094d\u091e\u093e\u0928\u093f\u0915 \u0924\u0930\u094d\u0915 \u092e\u094c\u091c\u0942\u0926 \u0939\u0948\u0964.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0914\u0930 \u0905\u0927\u093f\u0915 \u091c\u093e\u0928\u0947\u0902: <a href=\"https:\/\/dmdwarrior.com\/hi\/regenxbio-phase-1-2-affinity-duchenne-trial-of-rgx-202\/\" target=\"_blank\" rel=\"noreferrer noopener\">Regenxbio \u0928\u0947 Rgx-202 \u0915\u0947 \u091a\u0930\u0923 1\/2 Affinity Duchenne \u092a\u0930\u0940\u0915\u094d\u0937\u0923 \u0938\u0947 \u0928\u090f \u0938\u0915\u093e\u0930\u093e\u0924\u094d\u092e\u0915 \u0905\u0902\u0924\u0930\u093f\u092e \u0921\u0947\u091f\u093e \u0915\u0940 \u0930\u093f\u092a\u094b\u0930\u094d\u091f \u0926\u0940 \u0939\u0948\u0964<\/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=\"genethon-gnt-0004\">\u091c\u0940\u0928\u0947\u0925\u0949\u0928 GNT-0004<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"overview-of-gnt-0004\">\u091c\u0940\u090f\u0928\u091f\u0940-0004 \u0915\u093e \u0905\u0935\u0932\u094b\u0915\u0928<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Genethon \u0915\u093e GNT-0004, \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\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u094b \u0906\u0917\u0947 \u092c\u0922\u093c\u093e\u0928\u0947 \u092e\u0947\u0902 \u092f\u0942\u0930\u094b\u092a\u0940\u092f \u0928\u0947\u0924\u0943\u0924\u094d\u0935 \u0935\u093e\u0932\u0947 \u092a\u094d\u0930\u092f\u093e\u0938 \u0915\u093e \u092a\u094d\u0930\u0924\u093f\u0928\u093f\u0927\u093f\u0924\u094d\u0935 \u0915\u0930\u0924\u093e \u0939\u0948\u0964.<\/strong> \u0935\u094d\u092f\u093e\u092a\u0915 \u092a\u0942\u0930\u094d\u0935-\u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0905\u0928\u0941\u0938\u0902\u0927\u093e\u0928 \u0915\u0947 \u092e\u093e\u0927\u094d\u092f\u092e \u0938\u0947 \u0935\u093f\u0915\u0938\u093f\u0924 \u0915\u0940 \u0917\u0908 \u0907\u0938 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u093e \u0915\u093e \u0909\u0926\u094d\u0926\u0947\u0936\u094d\u092f \u092c\u0947\u0939\u0924\u0930 \u0938\u0941\u0930\u0915\u094d\u0937\u093e \u0915\u0947 \u0938\u093e\u0925 \u092e\u093e\u0907\u0915\u094d\u0930\u094b-\u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0915\u0940 \u0928\u093f\u0930\u0902\u0924\u0930 \u0905\u092d\u093f\u0935\u094d\u092f\u0915\u094d\u0924\u093f \u092a\u094d\u0930\u0926\u093e\u0928 \u0915\u0930\u0928\u093e \u0939\u0948\u0964.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"clinical-development\">\u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0935\u093f\u0915\u093e\u0938<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">GNT-0004 \u0928\u0947 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092e\u0942\u0932\u094d\u092f\u093e\u0902\u0915\u0928 \u092e\u0947\u0902 \u092a\u094d\u0930\u0935\u0947\u0936 \u0915\u0930 \u0932\u093f\u092f\u093e \u0939\u0948 \u0914\u0930 \u0907\u0938\u0915\u0947 \u0936\u0941\u0930\u0941\u0906\u0924\u0940 \u0938\u0902\u0915\u0947\u0924 \u0906\u0936\u093e\u091c\u0928\u0915 \u0939\u0948\u0902:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0909\u092a\u091a\u093e\u0930\u093f\u0924 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u0938\u094d\u0925\u093f\u0930 \u092e\u093e\u0907\u0915\u094d\u0930\u094b-\u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0905\u092d\u093f\u0935\u094d\u092f\u0915\u094d\u0924\u093f<\/li>\n\n\n\n<li>\u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915 \u0938\u092e\u0942\u0939\u094b\u0902 \u092e\u0947\u0902 \u0915\u094b\u0908 \u0917\u0902\u092d\u0940\u0930 \u092a\u094d\u0930\u0924\u093f\u0915\u0942\u0932 \u0918\u091f\u0928\u093e \u0926\u0930\u094d\u091c \u0928\u0939\u0940\u0902 \u0915\u0940 \u0917\u0908, \u091c\u093f\u0938\u0938\u0947 \u0938\u0941\u0930\u0915\u094d\u0937\u093e \u0915\u093e \u0938\u0915\u093e\u0930\u093e\u0924\u094d\u092e\u0915 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923 \u0938\u093e\u092e\u0928\u0947 \u0906\u092f\u093e \u0939\u0948\u0964<\/li>\n\n\n\n<li>\u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0938\u0941\u0927\u093e\u0930 \u0915\u0947 \u0930\u0941\u091d\u093e\u0928\u094b\u0902 \u0915\u0947 \u092a\u094d\u0930\u092e\u093e\u0923<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u0914\u0930 \u0905\u0927\u093f\u0915 \u091c\u093e\u0928\u0947\u0902: <a href=\"https:\/\/dmdwarrior.com\/hi\/genethon-gnt0004-gene-therapy-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u091c\u0940\u090f\u0928\u091f\u0940-0004 \u0915\u0947 \u092c\u093e\u0930\u0947 \u092e\u0947\u0902<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"scientific-contribution\">\u0935\u0948\u091c\u094d\u091e\u093e\u0928\u093f\u0915 \u092f\u094b\u0917\u0926\u093e\u0928<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Genethon \u0915\u093e \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923 \u0907\u0938 \u092c\u093e\u0924 \u092a\u0930 \u091c\u094b\u0930 \u0926\u0947\u0924\u093e \u0939\u0948:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u092a\u0930\u093f\u0936\u0941\u0926\u094d\u0927 \u0935\u0947\u0915\u094d\u091f\u0930 \u0907\u0902\u091c\u0940\u0928\u093f\u092f\u0930\u093f\u0902\u0917<\/li>\n\n\n\n<li>\u0915\u092e \u092a\u094d\u0930\u0924\u093f\u0930\u0915\u094d\u0937\u093e\u091c\u0928\u0915\u0924\u093e<\/li>\n\n\n\n<li>\u091c\u0940\u0928 \u0905\u092d\u093f\u0935\u094d\u092f\u0915\u094d\u0924\u093f \u0915\u0940 \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0938\u094d\u0925\u093e\u092f\u093f\u0924\u094d\u0935<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u092f\u0939 \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\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u092a\u094d\u0930\u0924\u093f\u0938\u094d\u092a\u0930\u094d\u0927\u0940 \u0915\u094d\u0937\u0947\u0924\u094d\u0930 \u092e\u0947\u0902 \u091c\u0940\u090f\u0928\u091f\u0940-0004 \u0915\u094b \u090f\u0915 \u092e\u091c\u092c\u0942\u0924 \u0926\u093e\u0935\u0947\u0926\u093e\u0930 \u0915\u0947 \u0930\u0942\u092a \u092e\u0947\u0902 \u0938\u094d\u0925\u093e\u092a\u093f\u0924 \u0915\u0930\u0924\u093e \u0939\u0948\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=\"emerging-gene-therapy-research-in-china-and-russia\">\u091a\u0940\u0928 \u0914\u0930 \u0930\u0942\u0938 \u092e\u0947\u0902 \u0909\u092d\u0930\u0924\u0947 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0905\u0928\u0941\u0938\u0902\u0927\u093e\u0928<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u092a\u0936\u094d\u091a\u093f\u092e\u0940 \u091c\u0948\u0935 \u092a\u094d\u0930\u094c\u0926\u094d\u092f\u094b\u0917\u093f\u0915\u0940 \u0915\u0902\u092a\u0928\u093f\u092f\u094b\u0902 \u0915\u0947 \u0905\u0932\u093e\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\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u092a\u0930 \u091a\u0940\u0928 \u0914\u0930 \u0930\u0942\u0938 \u092e\u0947\u0902 \u092d\u0940 \u0938\u0915\u094d\u0930\u093f\u092f \u0930\u0942\u092a \u0938\u0947 \u0936\u094b\u0927 \u0915\u093f\u092f\u093e \u091c\u093e \u0930\u0939\u093e \u0939\u0948\u0964 \u092f\u0947 \u0915\u094d\u0937\u0947\u0924\u094d\u0930 \u0935\u0948\u0936\u094d\u0935\u093f\u0915 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0905\u0928\u0941\u0938\u0902\u0927\u093e\u0928 \u092a\u094d\u0930\u092f\u093e\u0938\u094b\u0902 \u092e\u0947\u0902 \u0924\u0947\u091c\u0940 \u0938\u0947 \u092f\u094b\u0917\u0926\u093e\u0928 \u0926\u0947 \u0930\u0939\u0947 \u0939\u0948\u0902\u0964.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"china\">\u091a\u0940\u0928<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u091a\u0940\u0928\u0940 \u0905\u0928\u0941\u0938\u0902\u0927\u093e\u0928 \u0938\u0902\u0938\u094d\u0925\u093e\u0928 \u0914\u0930 \u091c\u0948\u0935 \u092a\u094d\u0930\u094c\u0926\u094d\u092f\u094b\u0917\u093f\u0915\u0940 \u0915\u0902\u092a\u0928\u093f\u092f\u093e\u0902 \u0928\u093f\u092e\u094d\u0928\u0932\u093f\u0916\u093f\u0924 \u0939\u0948\u0902:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0928\u090f \u0915\u0948\u092a\u094d\u0938\u093f\u0921 \u0915\u0947 \u0938\u093e\u0925 \u090f\u090f\u0935\u0940-\u0906\u0927\u093e\u0930\u093f\u0924 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u094b \u0906\u0917\u0947 \u092c\u0922\u093c\u093e\u0928\u093e<\/li>\n\n\n\n<li>\u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915 \u091a\u0930\u0923 \u0915\u0947 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923\u094b\u0902 \u0915\u093e \u0938\u0902\u091a\u093e\u0932\u0928 \u0915\u0930\u0928\u093e<\/li>\n\n\n\n<li>\u0932\u093e\u0917\u0924-\u092a\u094d\u0930\u092d\u093e\u0935\u0940 \u0935\u093f\u0928\u093f\u0930\u094d\u092e\u093e\u0923 \u0930\u0923\u0928\u0940\u0924\u093f\u092f\u094b\u0902 \u0915\u0940 \u0916\u094b\u091c \u0915\u0930\u0928\u093e<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u092a\u094d\u0930\u093e\u0930\u0902\u092d\u093f\u0915 \u0930\u093f\u092a\u094b\u0930\u094d\u091f\u094b\u0902 \u0938\u0947 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0905\u092d\u093f\u0935\u094d\u092f\u0915\u094d\u0924\u093f \u0914\u0930 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0938\u0941\u0927\u093e\u0930 \u0915\u0947 \u0906\u0936\u093e\u091c\u0928\u0915 \u0938\u094d\u0924\u0930\u094b\u0902 \u0915\u093e \u0938\u0902\u0915\u0947\u0924 \u092e\u093f\u0932\u0924\u093e \u0939\u0948, \u0939\u093e\u0932\u093e\u0902\u0915\u093f \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0921\u0947\u091f\u093e \u0915\u0940 \u0905\u092d\u0940 \u092d\u0940 \u0906\u0935\u0936\u094d\u092f\u0915\u0924\u093e \u0939\u0948\u0964.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"russia\">\u0930\u0942\u0938<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u0930\u0942\u0938 \u092e\u0947\u0902 \u0905\u0915\u093e\u0926\u092e\u093f\u0915 \u0914\u0930 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u0905\u0928\u0941\u0938\u0902\u0927\u093e\u0928 \u0915\u0947\u0902\u0926\u094d\u0930 \u0928\u093f\u092e\u094d\u0928\u0932\u093f\u0916\u093f\u0924 \u0939\u0948\u0902:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0935\u0948\u0915\u0932\u094d\u092a\u093f\u0915 \u0935\u093e\u092f\u0930\u0932 \u0935\u093e\u0939\u0915\u094b\u0902 \u0914\u0930 \u0935\u093f\u0924\u0930\u0923 \u0935\u093f\u0927\u093f\u092f\u094b\u0902 \u0915\u0940 \u091c\u093e\u0902\u091a \u0915\u0930\u0928\u093e<\/li>\n\n\n\n<li>\u0918\u0930\u0947\u0932\u0942 \u0938\u094d\u0924\u0930 \u092a\u0930 \u0909\u0924\u094d\u092a\u093e\u0926\u093f\u0924 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u092a\u094d\u0932\u0947\u091f\u092b\u0949\u0930\u094d\u092e \u0935\u093f\u0915\u0938\u093f\u0924 \u0915\u0930\u0928\u093e<\/li>\n\n\n\n<li>\u092a\u094d\u0930\u093e\u092f\u094b\u0917\u093f\u0915 \u0909\u092a\u091a\u093e\u0930\u094b\u0902 \u0924\u0915 \u092a\u0939\u0941\u0902\u091a \u0915\u093e \u0935\u093f\u0938\u094d\u0924\u093e\u0930 \u0915\u0930\u0928\u093e<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u0939\u093e\u0932\u093e\u0902\u0915\u093f \u0921\u0947\u091f\u093e \u0915\u0940 \u092a\u093e\u0930\u0926\u0930\u094d\u0936\u093f\u0924\u093e \u0938\u0940\u092e\u093f\u0924 \u092c\u0928\u0940 \u0939\u0941\u0908 \u0939\u0948, \u0932\u0947\u0915\u093f\u0928 \u0936\u0941\u0930\u0941\u0906\u0924\u0940 \u0938\u0902\u0915\u0947\u0924 \u092c\u0924\u093e\u0924\u0947 \u0939\u0948\u0902 \u0915\u093f \u092f\u0947 \u0905\u0927\u094d\u092f\u092f\u0928 \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\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u0935\u0948\u0936\u094d\u0935\u093f\u0915 \u0935\u093f\u0915\u093e\u0938 \u092e\u0947\u0902 \u092e\u0942\u0932\u094d\u092f\u0935\u093e\u0928 \u0905\u0902\u0924\u0930\u094d\u0926\u0943\u0937\u094d\u091f\u093f \u092a\u094d\u0930\u0926\u093e\u0928 \u0915\u0930 \u0938\u0915\u0924\u0947 \u0939\u0948\u0902\u0964.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"a-hopeful-outlook\">\u090f\u0915 \u0906\u0936\u093e\u0935\u093e\u0926\u0940 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u092f\u0939 \u0909\u0924\u094d\u0938\u093e\u0939\u091c\u0928\u0915 \u0939\u0948 \u0915\u093f \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0905\u0928\u0941\u0938\u0902\u0927\u093e\u0928 \u0935\u0948\u0936\u094d\u0935\u093f\u0915 \u0938\u094d\u0924\u0930 \u092a\u0930 \u0935\u093f\u0938\u094d\u0924\u093e\u0930 \u0915\u0930 \u0930\u0939\u093e \u0939\u0948\u0964 \u0939\u092e \u0907\u0928 \u0918\u091f\u0928\u093e\u0915\u094d\u0930\u092e\u094b\u0902 \u092a\u0930 \u092c\u093e\u0930\u0940\u0915\u0940 \u0938\u0947 \u0928\u091c\u093c\u0930 \u0930\u0916 \u0930\u0939\u0947 \u0939\u0948\u0902, \u0914\u0930 \u0936\u0941\u0930\u0941\u0906\u0924\u0940 \u0938\u0902\u0915\u0947\u0924 \u092c\u0924\u093e\u0924\u0947 \u0939\u0948\u0902 \u0915\u093f \u091a\u0940\u0928 \u0914\u0930 \u0930\u0942\u0938 \u0926\u094b\u0928\u094b\u0902 \u092e\u0947\u0902 \u091a\u0932 \u0930\u0939\u0947 \u0905\u0927\u094d\u092f\u092f\u0928 \u0906\u0936\u093e\u091c\u0928\u0915 \u0939\u0948\u0902\u0964. <strong>\u091c\u0948\u0938\u0947-\u091c\u0948\u0938\u0947 \u0905\u0927\u093f\u0915 \u0921\u0947\u091f\u093e \u0909\u092a\u0932\u092c\u094d\u0927 \u0939\u094b\u0917\u093e, \u092f\u0947 \u092a\u094d\u0930\u092f\u093e\u0938 \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\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0940 \u0909\u092a\u0932\u092c\u094d\u0927\u0924\u093e \u0914\u0930 \u0938\u093e\u092e\u0930\u094d\u0925\u094d\u092f \u0915\u094b \u0935\u093f\u0936\u094d\u0935 \u0938\u094d\u0924\u0930 \u092a\u0930 \u0915\u093e\u092b\u0940 \u0939\u0926 \u0924\u0915 \u0924\u0947\u091c \u0915\u0930 \u0938\u0915\u0924\u0947 \u0939\u0948\u0902\u0964.<\/strong><\/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>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"challenges-and-limitations-of-gene-therapy-in-dmd\">\u092e\u0927\u0941\u092e\u0947\u0939 \u0930\u094b\u0917 \u092e\u0947\u0902 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0940 \u091a\u0941\u0928\u094c\u0924\u093f\u092f\u093e\u0901 \u0914\u0930 \u0938\u0940\u092e\u093e\u090f\u0901<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u092a\u094d\u0930\u0917\u0924\u093f \u0915\u0947 \u092c\u093e\u0935\u091c\u0942\u0926, \u0915\u0908 \u091a\u0941\u0928\u094c\u0924\u093f\u092f\u093e\u0901 \u0905\u092d\u0940 \u092d\u0940 \u092c\u0928\u0940 \u0939\u0941\u0908 \u0939\u0948\u0902:<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"immunogenicity\">\u092a\u094d\u0930\u0924\u093f\u0930\u0915\u094d\u0937\u093e\u091c\u0928\u0915\u0924\u093e<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">AAV \u0935\u0948\u0915\u094d\u091f\u0930 \u0915\u0947 \u0916\u093f\u0932\u093e\u092b \u092a\u0939\u0932\u0947 \u0938\u0947 \u092e\u094c\u091c\u0942\u0926 \u090f\u0902\u091f\u0940\u092c\u0949\u0921\u0940 \u0930\u094b\u0917\u0940 \u0915\u0940 \u092a\u093e\u0924\u094d\u0930\u0924\u093e \u0915\u094b \u0938\u0940\u092e\u093f\u0924 \u0915\u0930 \u0938\u0915\u0924\u0940 \u0939\u0948\u0902\u0964.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"durability\">\u0938\u0939\u0928\u0936\u0940\u0932\u0924\u093e<\/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 \u0915\u0940 \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0905\u092d\u093f\u0935\u094d\u092f\u0915\u094d\u0924\u093f \u0905\u0928\u093f\u0936\u094d\u091a\u093f\u0924 \u092c\u0928\u0940 \u0939\u0941\u0908 \u0939\u0948\u0964.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"manufacturing-constraints\">\u0935\u093f\u0928\u093f\u0930\u094d\u092e\u093e\u0923 \u0938\u0902\u092c\u0902\u0927\u0940 \u092c\u093e\u0927\u093e\u090f\u0901<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u0917\u0941\u0923\u0935\u0924\u094d\u0924\u093e \u092c\u0928\u093e\u090f \u0930\u0916\u0924\u0947 \u0939\u0941\u090f \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u092c\u0922\u093c\u093e\u0928\u093e \u091c\u091f\u093f\u0932 \u0914\u0930 \u092e\u0939\u0902\u0917\u093e \u0939\u0948\u0964.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"cost-and-accessibility\">\u0932\u093e\u0917\u0924 \u0914\u0930 \u092a\u0939\u0941\u0902\u091a<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u092c\u0947\u0939\u0926 \u092e\u0939\u0902\u0917\u0940 \u0939\u094b\u0928\u0947 \u0915\u0940 \u0906\u0936\u0902\u0915\u093e \u0939\u0948, \u091c\u093f\u0938\u0938\u0947 \u0935\u0948\u0936\u094d\u0935\u093f\u0915 \u0938\u094d\u0924\u0930 \u092a\u0930 \u0907\u0938\u0915\u0940 \u092a\u0939\u0941\u0902\u091a \u0915\u094b \u0932\u0947\u0915\u0930 \u091a\u093f\u0902\u0924\u093e\u090f\u0902 \u092c\u0922\u093c \u0930\u0939\u0940 \u0939\u0948\u0902\u0964.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0914\u0930 \u0905\u0927\u093f\u0915 \u091c\u093e\u0928\u0947\u0902: <a href=\"https:\/\/dmdwarrior.com\/hi\/duchenne-muscular-dystrophy-treatment-cost\/\" target=\"_blank\" rel=\"noreferrer noopener\">\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 \u0909\u092a\u091a\u093e\u0930 \u0915\u0940 \u0932\u093e\u0917\u0924 \u0928\u093f\u092f\u0902\u0924\u094d\u0930\u0923 \u0938\u0947 \u092c\u093e\u0939\u0930 \u0915\u094d\u092f\u094b\u0902 \u0939\u0948?<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\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\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u094b \u0935\u094d\u092f\u093e\u092a\u0915 \u0930\u0942\u092a \u0938\u0947 \u0905\u092a\u0928\u093e\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0907\u0928 \u091a\u0941\u0928\u094c\u0924\u093f\u092f\u094b\u0902 \u0915\u093e \u0938\u092e\u093e\u0927\u093e\u0928 \u0915\u0930\u0928\u093e \u0906\u0935\u0936\u094d\u092f\u0915 \u0939\u0948\u0964.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"future-directions-and-innovations\">\u092d\u0935\u093f\u0937\u094d\u092f \u0915\u0940 \u0926\u093f\u0936\u093e\u090f\u0901 \u0914\u0930 \u0928\u0935\u093e\u091a\u093e\u0930<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\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\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u092d\u0935\u093f\u0937\u094d\u092f \u092e\u0947\u0902 \u0928\u093f\u092e\u094d\u0928\u0932\u093f\u0916\u093f\u0924 \u0936\u093e\u092e\u093f\u0932 \u0939\u094b \u0938\u0915\u0924\u0947 \u0939\u0948\u0902:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u091c\u0940\u0928 \u0938\u0902\u092a\u093e\u0926\u0928 \u0924\u0915\u0928\u0940\u0915 (CRISPR\/Cas9) \u0915\u093e \u0909\u092a\u092f\u094b\u0917 \u0915\u0930\u0915\u0947 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928\u094b\u0902 \u0915\u094b \u0938\u0940\u0927\u0947 \u0920\u0940\u0915 \u0915\u093f\u092f\u093e \u091c\u093e \u0938\u0915\u0924\u093e \u0939\u0948\u0964<\/strong><\/li>\n\n\n\n<li>\u0915\u092e \u092a\u094d\u0930\u0924\u093f\u0930\u0915\u094d\u0937\u093e\u091c\u0928\u0915\u0924\u093e \u0935\u093e\u0932\u0947 \u0905\u0917\u0932\u0940 \u092a\u0940\u0922\u093c\u0940 \u0915\u0947 \u0935\u0947\u0915\u094d\u091f\u0930<\/li>\n\n\n\n<li><strong>\u092c\u093e\u0930-\u092c\u093e\u0930 \u0916\u0941\u0930\u093e\u0915 \u0926\u0947\u0928\u0947 \u0915\u0940 \u0930\u0923\u0928\u0940\u0924\u093f\u092f\u093e\u0901<\/strong><\/li>\n\n\n\n<li>\u090f\u0915\u094d\u0938\u0949\u0928 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0914\u0930 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u094b \u090f\u0915\u0940\u0915\u0943\u0924 \u0915\u0930\u0928\u0947 \u0935\u093e\u0932\u0940 \u0938\u0902\u092f\u094b\u091c\u0928 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u093e\u090f\u0901<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u092f\u0947 \u0928\u0935\u093e\u091a\u093e\u0930 \u092a\u094d\u0930\u092d\u093e\u0935\u0936\u0940\u0932\u0924\u093e \u0915\u094b \u0914\u0930 \u092c\u0922\u093c\u093e \u0938\u0915\u0924\u0947 \u0939\u0948\u0902 \u0914\u0930 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0940 \u092a\u093e\u0924\u094d\u0930\u0924\u093e \u0915\u093e \u0926\u093e\u092f\u0930\u093e \u092c\u0922\u093c\u093e \u0938\u0915\u0924\u0947 \u0939\u0948\u0902\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=\"conclusion\">\u0928\u093f\u0937\u094d\u0915\u0930\u094d\u0937<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\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\u0902\u092d\u093e\u0935\u093f\u0924 \u0928\u0908 \u091c\u0940\u0928 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u093e \u0935\u093f\u0915\u093e\u0938 \u0928\u094d\u092f\u0942\u0930\u094b\u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u092e\u0947\u0921\u093f\u0938\u093f\u0928 \u092e\u0947\u0902 \u0938\u092c\u0938\u0947 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u092a\u094d\u0930\u0917\u0924\u093f \u092e\u0947\u0902 \u0938\u0947 \u090f\u0915 \u0939\u0948\u0964.<\/strong> \u090f\u0938\u091c\u0940\u091f\u0940-003, \u0906\u0930\u091c\u0940\u090f\u0915\u094d\u0938-202 \u0914\u0930 \u091c\u0940\u090f\u0928\u091f\u0940-0004 \u091c\u0948\u0938\u0940 \u0925\u0947\u0930\u0947\u092a\u0940 \u091c\u0940\u0928 \u0921\u093f\u0932\u0940\u0935\u0930\u0940 \u092a\u094d\u0930\u094c\u0926\u094d\u092f\u094b\u0917\u093f\u0915\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u0928\u0935\u093e\u091a\u093e\u0930 \u0915\u0940 \u0924\u0940\u0935\u094d\u0930 \u0917\u0924\u093f \u0914\u0930 \u092c\u0922\u093c\u0924\u0940 \u092a\u0930\u093f\u0937\u094d\u0915\u093e\u0930\u093f\u0924\u093e \u0915\u094b \u0926\u0930\u094d\u0936\u093e\u0924\u0940 \u0939\u0948\u0902\u0964.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0938\u093e\u0925 \u0939\u0940, \u091a\u0940\u0928 \u0914\u0930 \u0930\u0942\u0938 \u092e\u0947\u0902 \u092c\u0922\u093c\u0924\u0947 \u0905\u0928\u0941\u0938\u0902\u0927\u093e\u0928 \u092a\u094d\u0930\u092f\u093e\u0938\u094b\u0902 \u0938\u0947 \u092f\u0939 \u0905\u0924\u093f\u0930\u093f\u0915\u094d\u0924 \u0909\u092e\u094d\u092e\u0940\u0926 \u091c\u0917\u0924\u0940 \u0939\u0948 \u0915\u093f \u0935\u0948\u0936\u094d\u0935\u093f\u0915 \u0938\u0939\u092f\u094b\u0917 \u0938\u0947 \u092a\u094d\u0930\u0917\u0924\u093f \u092e\u0947\u0902 \u0924\u0947\u091c\u0940 \u0906\u090f\u0917\u0940 \u0914\u0930 \u092a\u0939\u0941\u0902\u091a \u092e\u0947\u0902 \u0938\u0941\u0927\u093e\u0930 \u0939\u094b\u0917\u093e\u0964.<\/strong> \u0939\u093e\u0932\u093e\u0902\u0915\u093f \u091a\u0941\u0928\u094c\u0924\u093f\u092f\u093e\u0902 \u0905\u092d\u0940 \u092d\u0940 \u092c\u0928\u0940 \u0939\u0941\u0908 \u0939\u0948\u0902, \u0932\u0947\u0915\u093f\u0928 \u0907\u0928 \u0909\u092a\u091a\u093e\u0930\u094b\u0902 \u0915\u093e \u092d\u0935\u093f\u0937\u094d\u092f \u0928\u093f\u0938\u094d\u0938\u0902\u0926\u0947\u0939 \u0906\u0936\u093e\u091c\u0928\u0915 \u0939\u0948, \u091c\u094b \u0939\u092e\u0947\u0902 \u0910\u0938\u0947 \u092d\u0935\u093f\u0937\u094d\u092f \u0915\u0947 \u0915\u0930\u0940\u092c \u0932\u093e \u0930\u0939\u093e \u0939\u0948 \u091c\u0939\u093e\u0902 \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u093e \u0915\u0947 \u092e\u093e\u0927\u094d\u092f\u092e \u0938\u0947 \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\u094b \u092a\u094d\u0930\u092d\u093e\u0935\u0940 \u0922\u0902\u0917 \u0938\u0947 \u092a\u094d\u0930\u092c\u0902\u0927\u093f\u0924 \u0915\u093f\u092f\u093e \u091c\u093e \u0938\u0915\u0924\u093e \u0939\u0948 - \u092f\u093e \u092f\u0939\u093e\u0902 \u0924\u0915 \u0915\u093f \u092e\u094c\u0932\u093f\u0915 \u0930\u0942\u092a \u0938\u0947 \u092c\u0926\u0932\u093e \u092d\u0940 \u091c\u093e \u0938\u0915\u0924\u093e \u0939\u0948\u0964.<\/p>","protected":false},"excerpt":{"rendered":"<p>The landscape of potential new gene therapies for Duchenne muscular dystrophy (DMD) is rapidly evolving, offering renewed hope for patients and families affected by this devastating neuromuscular disorder. Among the most closely watched approaches are Solid Biosciences SGT-003, RegenxBio RGX-202, and Genethon GNT-0004, which represent the next wave of innovation in genetic medicine. These emerging [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":7484,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[33,72,61,105,106,107,108,65,110,109],"class_list":["post-2261","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-elevidys","tag-gene-editing","tag-gene-therapy","tag-genethon","tag-gnt-0004","tag-regenxbio","tag-rgx-202","tag-sarepta","tag-sgt-003","tag-solid-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/2261","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=2261"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/2261\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media\/7484"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media?parent=2261"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/categories?post=2261"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/tags?post=2261"}],"curies":[{"name":"\u0921\u092c\u094d\u0932\u094d\u092f\u0942\u092a\u0940","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}