{"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\/ar\/gen6050x-exon-50-skipping-base-editing-drug-one-year-results\/","title":{"rendered":"\u062f\u0648\u0627\u0621 GEN6050X \u0644\u062a\u0639\u062f\u064a\u0644 \u0642\u0627\u0639\u062f\u0629 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 50: \u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0633\u0646\u0629 \u0627\u0644\u0623\u0648\u0644\u0649 \u062a\u064f\u0638\u0647\u0631 \u062a\u0642\u062f\u0645\u0627\u064b \u0645\u0634\u062c\u0639\u0627\u064b \u0641\u064a \u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>\u064a\u062d\u0638\u0649 \u062f\u0648\u0627\u0621 GEN6050X \u0644\u062a\u0639\u062f\u064a\u0644 \u0627\u0644\u0642\u0627\u0639\u062f\u0629 \u0628\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 50\u060c \u0627\u0644\u0630\u064a \u0637\u0648\u0631\u062a\u0647 \u0634\u0631\u0643\u0629 GenAssist Therapeutics\u060c \u0628\u0627\u0647\u062a\u0645\u0627\u0645 \u0645\u062a\u0632\u0627\u064a\u062f \u0628\u0627\u0639\u062a\u0628\u0627\u0631\u0647 \u0623\u062d\u062f \u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u0623\u0648\u0644\u0649 \u0644\u062a\u0639\u062f\u064a\u0644 \u0627\u0644\u0642\u0627\u0639\u062f\u0629 \u0627\u0644\u062a\u064a \u064a\u062a\u0645 \u062a\u0642\u064a\u064a\u0645\u0647\u0627 \u0644\u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a (DMD).<\/strong> \u0639\u0644\u0649 \u0639\u0643\u0633 \u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u062c\u064a\u0646\u064a\u0629 \u0627\u0644\u062a\u0642\u0644\u064a\u062f\u064a\u0629 \u0627\u0644\u062a\u064a \u062a\u064f\u0648\u0635\u0644 \u0646\u0633\u062e\u0629 \u0645\u062e\u062a\u0635\u0631\u0629 \u0645\u0646 \u062c\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646\u060c \u0635\u064f\u0645\u0645 \u0639\u0644\u0627\u062c GEN6050X \u0644\u062a\u0639\u062f\u064a\u0644 \u0627\u0644\u062d\u0645\u0636 \u0627\u0644\u0646\u0648\u0648\u064a \u0628\u062f\u0642\u0629 \u0628\u062d\u064a\u062b \u064a\u062a\u0645 \u062d\u0630\u0641 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 50\u060c \u0645\u0645\u0627 \u0642\u062f \u064a\u064f\u0639\u064a\u062f \u0625\u0637\u0627\u0631 \u0627\u0644\u0642\u0631\u0627\u0621\u0629 \u0625\u0644\u0649 \u0648\u0636\u0639\u0647 \u0627\u0644\u0637\u0628\u064a\u0639\u064a \u0648\u064a\u064f\u0645\u0643\u0651\u0646 \u0627\u0644\u062c\u0633\u0645 \u0645\u0646 \u0625\u0646\u062a\u0627\u062c \u0634\u0643\u0644 \u0648\u0638\u064a\u0641\u064a \u0645\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646. \u0648\u0628\u064a\u0646\u0645\u0627 \u0644\u0627 \u064a\u0632\u0627\u0644 \u0647\u0630\u0627 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062a\u062c\u0631\u064a\u0628\u064a \u0641\u064a \u0627\u0644\u0645\u0631\u0627\u062d\u0644 \u0627\u0644\u0623\u0648\u0644\u0649 \u0645\u0646 \u0627\u0644\u062a\u0637\u0648\u064a\u0631 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u060c \u062a\u0634\u064a\u0631 \u0627\u0644\u0628\u064a\u0627\u0646\u0627\u062a \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0627\u0644\u062d\u062f\u064a\u062b\u0629 \u0627\u0644\u062a\u064a \u0646\u064f\u0634\u0631\u062a \u0628\u0639\u062f \u0639\u0627\u0645 \u0648\u0627\u062d\u062f \u0625\u0644\u0649 \u0623\u0646\u0647 \u0642\u062f \u064a\u064f\u0642\u062f\u0645 \u0641\u0648\u0627\u0626\u062f \u0645\u0644\u0645\u0648\u0633\u0629 \u0648\u0645\u0633\u062a\u062f\u0627\u0645\u0629 \u0644\u0628\u0639\u0636 \u0627\u0644\u0645\u0631\u0636\u0649.<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u062c\u062f\u0648\u0644 \u0627\u0644\u0645\u062d\u062a\u0648\u064a\u0627\u062a<\/h2><nav><ul><li><a href=\"#what-is-the-gen-6050-x-exon-50-skipping-base-editing-drug\">\u0645\u0627 \u0647\u0648 \u062f\u0648\u0627\u0621 GEN6050X \u0644\u062a\u0639\u062f\u064a\u0644 \u0627\u0644\u0642\u0648\u0627\u0639\u062f \u0628\u062a\u062c\u0627\u0648\u0632 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 50\u061f<\/a><\/li><li><a href=\"#one-year-clinical-results-of-gen-6050-x\">\u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u0645\u062f\u0629 \u0639\u0627\u0645 \u0648\u0627\u062d\u062f \u0644\u0640 GEN6050X<\/a><ul><li><a href=\"#motor-function-outcomes\">\u0646\u062a\u0627\u0626\u062c \u0648\u0638\u0627\u0626\u0641 \u0627\u0644\u062d\u0631\u0643\u0629<\/a><\/li><li><a href=\"#cardiac-function-results\">\u0646\u062a\u0627\u0626\u062c \u0648\u0638\u0627\u0626\u0641 \u0627\u0644\u0642\u0644\u0628<\/a><\/li><li><a href=\"#pulmonary-function-results\">\u0646\u062a\u0627\u0626\u062c \u0648\u0638\u0627\u0626\u0641 \u0627\u0644\u0631\u0626\u0629<\/a><\/li><\/ul><\/li><li><a href=\"#safety-profile-of-the-gen-6050-x-exon-50-skipping-base-editing-drug\">\u0627\u0644\u0645\u0644\u0641 \u0627\u0644\u062a\u0639\u0631\u064a\u0641\u064a \u0644\u0644\u0633\u0644\u0627\u0645\u0629 \u0644\u062f\u0648\u0627\u0621 GEN6050X \u0644\u062a\u0639\u062f\u064a\u0644 \u0642\u0627\u0639\u062f\u0629 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 50<\/a><\/li><li><a href=\"#limitations-of-the-current-clinical-study\">\u0645\u062d\u062f\u062f\u0627\u062a \u0627\u0644\u062f\u0631\u0627\u0633\u0629 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0627\u0644\u062d\u0627\u0644\u064a\u0629<\/a><\/li><li><a href=\"#future-outlook-for-gen-6050-x\">\u0627\u0644\u062a\u0648\u0642\u0639\u0627\u062a \u0627\u0644\u0645\u0633\u062a\u0642\u0628\u0644\u064a\u0629 \u0644\u0640 GEN6050X<\/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\">\u0645\u0627 \u0647\u0648 \u062f\u0648\u0627\u0621 GEN6050X \u0644\u062a\u0639\u062f\u064a\u0644 \u0627\u0644\u0642\u0648\u0627\u0639\u062f \u0628\u062a\u062c\u0627\u0648\u0632 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 50\u061f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u064a\u062a\u0645 \u062d\u0627\u0644\u064a\u064b\u0627 \u062f\u0631\u0627\u0633\u0629 GEN6050X \u0641\u064a \u062a\u062c\u0631\u0628\u0629 \u0633\u0631\u064a\u0631\u064a\u0629 \u0628\u062f\u0623\u0647\u0627 \u0627\u0644\u0628\u0627\u062d\u062b\u0648\u0646 (NCT06392724) \u0623\u062c\u0631\u064a\u062a \u0641\u064a \u0645\u0633\u062a\u0634\u0641\u0649 \u0643\u0644\u064a\u0629 \u0627\u0644\u0637\u0628 \u0628\u062c\u0627\u0645\u0639\u0629 \u0628\u0643\u064a\u0646 \u0627\u0644\u0627\u062a\u062d\u0627\u062f\u064a\u0629 \u0641\u064a \u0627\u0644\u0635\u064a\u0646.<\/strong> \u0634\u0645\u0644\u062a \u0627\u0644\u062f\u0631\u0627\u0633\u0629 \u0627\u0644\u0623\u0648\u0644\u064a\u0629 \u062b\u0644\u0627\u062b\u0629 \u0623\u0648\u0644\u0627\u062f \u0642\u0627\u062f\u0631\u064a\u0646 \u0639\u0644\u0649 \u0627\u0644\u0645\u0634\u064a \u0645\u0635\u0627\u0628\u064a\u0646 \u0628\u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a\u060c \u062a\u062a\u0631\u0627\u0648\u062d \u0623\u0639\u0645\u0627\u0631\u0647\u0645 \u0628\u064a\u0646 6.5 \u064810 \u0633\u0646\u0648\u0627\u062a. \u062a\u0644\u0642\u0649 \u0643\u0644 \u0645\u0634\u0627\u0631\u0643 \u062c\u0631\u0639\u0629 \u0648\u0627\u062d\u062f\u0629 \u0639\u0646 \u0637\u0631\u064a\u0642 \u0627\u0644\u062d\u0642\u0646 \u0627\u0644\u0648\u0631\u064a\u062f\u064a \u0645\u0646 GEN6050X \u0628\u062c\u0631\u0639\u0629 5 \u00d7 10\u00b9\u00b3 \u0648\u062d\u062f\u0629 \u062c\u064a\u0646\u0648\u0645\u064a\u0629\/\u0643\u062c\u0645. \u0641\u064a \u0622\u062e\u0631 \u0645\u062a\u0627\u0628\u0639\u0629\u060c \u0623\u0643\u0645\u0644 \u0627\u0644\u0645\u0634\u0627\u0631\u0643\u0648\u0646 \u0627\u0644\u062b\u0644\u0627\u062b\u0629 \u0639\u0627\u0645\u064b\u0627 \u0645\u0646 \u0627\u0644\u0645\u0631\u0627\u0642\u0628\u0629\u060c \u0628\u064a\u0646\u0645\u0627 \u0648\u0635\u0644 \u0627\u062b\u0646\u0627\u0646 \u0645\u0646\u0647\u0645 \u0625\u0644\u0649 18 \u0634\u0647\u0631\u064b\u0627 \u0628\u0639\u062f \u0627\u0644\u0639\u0644\u0627\u062c. \u0644\u0644\u0645\u0632\u064a\u062f \u0645\u0646 \u0627\u0644\u0645\u0639\u0644\u0648\u0645\u0627\u062a: <a href=\"https:\/\/dmdwarrior.com\/ar\/mutations-amenable-to-exon-50-skipping\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0627\u0644\u0637\u0641\u0631\u0627\u062a \u0627\u0644\u0642\u0627\u0628\u0644\u0629 \u0644\u0644\u0639\u0644\u0627\u062c \u0628\u062a\u062c\u0627\u0648\u0632 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 50 \u0641\u064a \u062f\u0648\u0634\u064a\u0646<\/a><\/p>\n\n\n\n<h2 id=\"one-year-clinical-results-of-gen-6050-x\" class=\"wp-block-heading\">\u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u0645\u062f\u0629 \u0639\u0627\u0645 \u0648\u0627\u062d\u062f \u0644\u0640 GEN6050X<\/h2>\n\n\n\n<h3 id=\"motor-function-outcomes\" class=\"wp-block-heading\">\u0646\u062a\u0627\u0626\u062c \u0648\u0638\u0627\u0626\u0641 \u0627\u0644\u062d\u0631\u0643\u0629<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0645\u0646 \u0623\u0643\u062b\u0631 \u0627\u0644\u062c\u0648\u0627\u0646\u0628 \u0627\u0644\u0645\u0634\u062c\u0639\u0629 \u0641\u064a \u0627\u0644\u062f\u0631\u0627\u0633\u0629 \u0627\u0633\u062a\u0645\u0631\u0627\u0631 \u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629. \u0641\u0641\u064a \u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a\u060c \u062a\u062a\u0631\u0627\u062c\u0639 \u0627\u0644\u0648\u0638\u0627\u0626\u0641 \u0627\u0644\u062d\u0631\u0643\u064a\u0629 \u0639\u0645\u0648\u0645\u0627\u064b \u0628\u0645\u0631\u0648\u0631 \u0627\u0644\u0648\u0642\u062a.<\/strong> \u0645\u0639 \u0630\u0644\u0643\u060c \u0648\u0628\u0639\u062f \u0645\u0631\u0648\u0631 \u0639\u0627\u0645\u060c \u0623\u0641\u0627\u062f \u0627\u0644\u0628\u0627\u062d\u062b\u0648\u0646 \u0628\u0623\u0646 \u0646\u062a\u0627\u0626\u062c \u062a\u0642\u064a\u064a\u0645 \u0627\u0644\u0645\u0634\u064a \u0641\u064a \u0646\u0648\u0631\u062b \u0633\u062a\u0627\u0631 (NSAA) \u0648\u0646\u062a\u0627\u0626\u062c \u062a\u0642\u064a\u064a\u0645 \u0623\u062f\u0627\u0621 \u0627\u0644\u0637\u0631\u0641 \u0627\u0644\u0639\u0644\u0648\u064a 2.0 (PUL 2.0) \u0638\u0644\u062a \u0645\u0633\u062a\u0642\u0631\u0629 \u0623\u0648 \u0634\u0647\u062f\u062a \u062a\u062d\u0633\u0646\u0627\u064b \u0637\u0641\u064a\u0641\u0627\u064b. \u0648\u0631\u063a\u0645 \u0635\u063a\u0631 \u0639\u062f\u062f \u0627\u0644\u0645\u0634\u0627\u0631\u0643\u064a\u0646\u060c \u064a\u064f\u0639\u062f \u0627\u0644\u062d\u0641\u0627\u0638 \u0639\u0644\u0649 \u0627\u0644\u0648\u0638\u064a\u0641\u0629 \u0627\u0644\u062d\u0631\u0643\u064a\u0629 \u0644\u0641\u062a\u0631\u0629 \u0637\u0648\u064a\u0644\u0629 \u0646\u062a\u064a\u062c\u0629\u064b \u0645\u0634\u062c\u0639\u0629 \u0641\u064a \u0645\u0631\u0636 \u0645\u062a\u0641\u0627\u0642\u0645 \u0645\u062b\u0644 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a.<\/p>\n\n\n\n<h3 id=\"cardiac-function-results\" class=\"wp-block-heading\">\u0646\u062a\u0627\u0626\u062c \u0648\u0638\u0627\u0626\u0641 \u0627\u0644\u0642\u0644\u0628<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u0644\u0645 \u062a\u0642\u062a\u0635\u0631 \u0627\u0644\u0641\u0648\u0627\u0626\u062f \u0627\u0644\u0645\u0630\u0643\u0648\u0631\u0629 \u0639\u0644\u0649 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u0647\u064a\u0643\u0644\u064a\u0629 \u0641\u0642\u0637. <strong>\u0644\u0627\u062d\u0638 \u0627\u0644\u0628\u0627\u062d\u062b\u0648\u0646 \u0623\u064a\u0636\u064b\u0627 \u0627\u062a\u062c\u0627\u0647\u0627\u062a \u0625\u064a\u062c\u0627\u0628\u064a\u0629 \u0641\u064a \u0648\u0638\u0627\u0626\u0641 \u0627\u0644\u0642\u0644\u0628\u060c \u0648\u0647\u0648 \u0623\u0645\u0631 \u0645\u0647\u0645 \u0628\u0634\u0643\u0644 \u062e\u0627\u0635 \u0644\u0623\u0646 \u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0642\u0644\u0628 \u0647\u064a \u0633\u0628\u0628 \u0631\u0626\u064a\u0633\u064a \u0644\u0644\u0645\u0636\u0627\u0639\u0641\u0627\u062a \u0641\u064a \u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a.<\/strong> \u0623\u0638\u0647\u0631\u062a \u0627\u0644\u062f\u0631\u0627\u0633\u0629 \u062a\u062d\u0633\u0646\u0627\u064b \u0645\u0637\u0644\u0642\u0627\u064b \u0645\u062a\u0648\u0633\u0637\u0627\u064b \u0628\u0646\u0633\u0628\u0629 5.32% \u0641\u064a \u0646\u0633\u0628\u0629 \u0642\u0630\u0641 \u0627\u0644\u0628\u0637\u064a\u0646 \u0627\u0644\u0623\u064a\u0633\u0631 (LVEF) \u0645\u0642\u0627\u0631\u0646\u0629\u064b \u0628\u0627\u0644\u0642\u064a\u0627\u0633\u0627\u062a \u0627\u0644\u0623\u0633\u0627\u0633\u064a\u0629 \u0644\u0643\u0644 \u0645\u0631\u064a\u0636. \u0648\u064a\u0645\u0643\u0646 \u0623\u0646 \u062a\u064f\u0633\u0647\u0645 \u0647\u0630\u0647 \u0627\u0644\u062a\u062d\u0633\u064a\u0646\u0627\u062a \u0641\u064a \u0648\u0638\u0627\u0626\u0641 \u0627\u0644\u0642\u0644\u0628 \u0641\u064a \u062a\u062d\u0633\u064a\u0646 \u0627\u0644\u0635\u062d\u0629 \u0639\u0644\u0649 \u0627\u0644\u0645\u062f\u0649 \u0627\u0644\u0637\u0648\u064a\u0644 \u0625\u0630\u0627 \u0645\u0627 \u062a\u0623\u0643\u062f\u062a \u0641\u064a \u062a\u062c\u0627\u0631\u0628 \u0633\u0631\u064a\u0631\u064a\u0629 \u0623\u0648\u0633\u0639 \u0646\u0637\u0627\u0642\u0627\u064b.<\/p>\n\n\n\n<h3 id=\"pulmonary-function-results\" class=\"wp-block-heading\">\u0646\u062a\u0627\u0626\u062c \u0648\u0638\u0627\u0626\u0641 \u0627\u0644\u0631\u0626\u0629<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0643\u0645\u0627 \u0644\u0648\u062d\u0638 \u062a\u062d\u0633\u0646 \u0641\u064a \u0648\u0638\u0627\u0626\u0641 \u0627\u0644\u0631\u0626\u0629 \u0628\u0639\u062f \u0627\u0644\u0639\u0644\u0627\u062c. \u0641\u0645\u0642\u0627\u0631\u0646\u0629\u064b \u0628\u0627\u0644\u0648\u0636\u0639 \u0642\u0628\u0644 \u0627\u0644\u0639\u0644\u0627\u062c\u060c \u0623\u0638\u0647\u0631 \u0627\u0644\u0645\u0631\u0636\u0649 \u062a\u062d\u0633\u0646\u0627\u064b \u0645\u062a\u0648\u0633\u0637\u0627\u064b \u0628\u0646\u0633\u0628\u0629 11.72% \u0641\u064a \u0627\u0644\u0633\u0639\u0629 \u0627\u0644\u062d\u064a\u0648\u064a\u0629 \u0627\u0644\u0642\u0633\u0631\u064a\u0629 (FVC) \u0648\u062a\u062d\u0633\u0646\u0627\u064b \u0628\u0646\u0633\u0628\u0629 25.29% \u0641\u064a \u0630\u0631\u0648\u0629 \u062a\u062f\u0641\u0642 \u0627\u0644\u0632\u0641\u064a\u0631 (PEF).<\/strong> \u062a\u0642\u064a\u0651\u0645 \u0647\u0630\u0647 \u0627\u0644\u0642\u064a\u0627\u0633\u0627\u062a \u0642\u0648\u0629 \u0627\u0644\u0631\u0626\u062a\u064a\u0646 \u0648\u0633\u0639\u0629 \u0627\u0644\u062a\u0646\u0641\u0633\u060c \u0648\u0643\u0644\u0627\u0647\u0645\u0627 \u064a\u062a\u0631\u0627\u062c\u0639 \u062a\u062f\u0631\u064a\u062c\u064a\u064b\u0627 \u0645\u0639 \u062a\u0642\u062f\u0645 \u0645\u0631\u0636 \u062f\u0648\u0634\u064a\u0646. \u0648\u0642\u062f \u0623\u0634\u0627\u0631 \u0627\u0644\u0628\u0627\u062d\u062b\u0648\u0646 \u0625\u0644\u0649 \u0623\u0646 \u0647\u0630\u0647 \u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0642\u062f \u062a\u062f\u0644 \u0639\u0644\u0649 \u0623\u0646 \u062f\u0648\u0627\u0621 GEN6050X \u064a\u0648\u0641\u0631 \u0641\u0648\u0627\u0626\u062f \u062a\u062a\u062c\u0627\u0648\u0632 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u0647\u064a\u0643\u0644\u064a\u0629\u060c \u0639\u0644\u0649 \u0627\u0644\u0631\u063a\u0645 \u0645\u0646 \u0627\u0644\u062d\u0627\u062c\u0629 \u0625\u0644\u0649 \u062f\u0631\u0627\u0633\u0627\u062a \u0623\u0648\u0633\u0639 \u0646\u0637\u0627\u0642\u064b\u0627 \u0644\u0644\u062a\u062d\u0642\u0642 \u0645\u0646 \u0647\u0630\u0647 \u0627\u0644\u0645\u0644\u0627\u062d\u0638\u0627\u062a \u0627\u0644\u0623\u0648\u0644\u064a\u0629.<\/p>\n\n\n\n<h2 id=\"safety-profile-of-the-gen-6050-x-exon-50-skipping-base-editing-drug\" class=\"wp-block-heading\">\u0627\u0644\u0645\u0644\u0641 \u0627\u0644\u062a\u0639\u0631\u064a\u0641\u064a \u0644\u0644\u0633\u0644\u0627\u0645\u0629 \u0644\u062f\u0648\u0627\u0621 GEN6050X \u0644\u062a\u0639\u062f\u064a\u0644 \u0642\u0627\u0639\u062f\u0629 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 50<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0644\u0627 \u062a\u0632\u0627\u0644 \u0627\u0644\u0633\u0644\u0627\u0645\u0629 \u0648\u0627\u062d\u062f\u0629 \u0645\u0646 \u0623\u0647\u0645 \u0627\u0644\u0623\u0633\u0626\u0644\u0629 \u0644\u0623\u064a \u0639\u0644\u0627\u062c \u062c\u064a\u0646\u064a \u062a\u062c\u0631\u064a\u0628\u064a. <strong>\u0628\u062d\u0633\u0628 \u0627\u0644\u0645\u062a\u0627\u0628\u0639\u0629 \u0627\u0644\u062a\u064a \u0627\u0633\u062a\u0645\u0631\u062a \u0639\u0627\u0645\u064b\u0627 \u0648\u0627\u062d\u062f\u064b\u0627\u060c \u0643\u0627\u0646 \u062f\u0648\u0627\u0621 GEN6050X \u062c\u064a\u062f \u0627\u0644\u062a\u062d\u0645\u0644 \u0628\u0634\u0643\u0644 \u0639\u0627\u0645. \u0639\u0627\u0646\u0649 \u0628\u0639\u0636 \u0627\u0644\u0645\u0631\u0636\u0649 \u0645\u0646 \u0622\u062b\u0627\u0631 \u062c\u0627\u0646\u0628\u064a\u0629 \u062e\u0637\u064a\u0631\u0629 \u0628\u0639\u062f \u0641\u062a\u0631\u0629 \u0648\u062c\u064a\u0632\u0629 \u0645\u0646 \u062a\u0646\u0627\u0648\u0644 \u0627\u0644\u062c\u0631\u0639\u0629\u060c \u0644\u0643\u0646 \u0627\u0644\u0628\u0627\u062d\u062b\u064a\u0646 \u0623\u0641\u0627\u062f\u0648\u0627 \u0628\u0623\u0646 \u0647\u0630\u0647 \u0627\u0644\u0622\u062b\u0627\u0631 \u0643\u0627\u0646\u062a \u0639\u0627\u0628\u0631\u0629\u060c \u0648\u064a\u0645\u0643\u0646 \u0627\u0644\u0633\u064a\u0637\u0631\u0629 \u0639\u0644\u064a\u0647\u0627\u060c \u0648\u0627\u062e\u062a\u0641\u062a \u062a\u0645\u0627\u0645\u064b\u0627 \u062f\u0648\u0646 \u0623\u064a \u0639\u0648\u0627\u0642\u0628 \u0637\u0648\u064a\u0644\u0629 \u0627\u0644\u0623\u0645\u062f.<\/strong> \u062e\u0644\u0627\u0644 \u0641\u062a\u0631\u0629 \u0627\u0644\u0645\u062a\u0627\u0628\u0639\u0629 \u0627\u0644\u0645\u0645\u062a\u062f\u0629\u060c \u0644\u0645 \u064a\u062d\u062f\u062f \u0627\u0644\u0628\u0627\u062d\u062b\u0648\u0646 \u0623\u064a \u0623\u0639\u0631\u0627\u0636 \u062c\u062f\u064a\u062f\u0629 \u0630\u0627\u062a \u0623\u0647\u0645\u064a\u0629 \u0633\u0631\u064a\u0631\u064a\u0629 \u0645\u0631\u062a\u0628\u0637\u0629 \u0628\u0627\u0644\u0639\u0644\u0627\u062c \u0623\u0648 \u0623\u064a \u062a\u0634\u0648\u0647\u0627\u062a \u0645\u062e\u062a\u0628\u0631\u064a\u0629 \u0628\u064a\u0646 \u0627\u0644\u0645\u0634\u0627\u0631\u0643\u064a\u0646 \u0627\u0644\u0630\u064a\u0646 \u062e\u0636\u0639\u0648\u0627 \u0644\u0644\u0639\u0644\u0627\u062c.<\/p>\n\n\n\n<h2 id=\"limitations-of-the-current-clinical-study\" class=\"wp-block-heading\">\u0645\u062d\u062f\u062f\u0627\u062a \u0627\u0644\u062f\u0631\u0627\u0633\u0629 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0627\u0644\u062d\u0627\u0644\u064a\u0629<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0639\u0644\u0649 \u0627\u0644\u0631\u063a\u0645 \u0645\u0646 \u0623\u0646 \u0647\u0630\u0647 \u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0648\u0627\u0639\u062f\u0629\u060c \u0625\u0644\u0627 \u0623\u0646\u0647 \u0645\u0646 \u0627\u0644\u0636\u0631\u0648\u0631\u064a \u062a\u0641\u0633\u064a\u0631\u0647\u0627 \u0628\u0639\u0646\u0627\u064a\u0629. \u0641\u0627\u0644\u062f\u0631\u0627\u0633\u0629 \u0627\u0644\u062d\u0627\u0644\u064a\u0629 \u0644\u0627 \u062a\u0634\u0645\u0644 \u0633\u0648\u0649 \u062b\u0644\u0627\u062b\u0629 \u0645\u0631\u0636\u0649\u060c \u0645\u0645\u0627 \u064a\u062c\u0639\u0644 \u0645\u0646 \u0627\u0644\u0645\u0633\u062a\u062d\u064a\u0644 \u0627\u0633\u062a\u062e\u0644\u0627\u0635 \u0627\u0633\u062a\u0646\u062a\u0627\u062c\u0627\u062a \u0642\u0627\u0637\u0639\u0629 \u062d\u0648\u0644 \u0627\u0644\u0641\u0639\u0627\u0644\u064a\u0629 \u0623\u0648 \u0627\u0644\u0633\u0644\u0627\u0645\u0629 \u0639\u0644\u0649 \u0627\u0644\u0645\u062f\u0649 \u0627\u0644\u0637\u0648\u064a\u0644. \u0644\u0630\u0627\u060c \u0633\u062a\u0643\u0648\u0646 \u0647\u0646\u0627\u0643 \u062d\u0627\u062c\u0629 \u0625\u0644\u0649 \u062a\u062c\u0627\u0631\u0628 \u0633\u0631\u064a\u0631\u064a\u0629 \u0623\u0648\u0633\u0639 \u0646\u0637\u0627\u0642\u064b\u0627 \u062a\u0634\u0645\u0644 \u0639\u062f\u062f\u064b\u0627 \u0623\u0643\u0628\u0631 \u0645\u0646 \u0627\u0644\u0645\u0634\u0627\u0631\u0643\u064a\u0646 \u0648\u0641\u062a\u0631\u0627\u062a \u0645\u0631\u0627\u0642\u0628\u0629 \u0623\u0637\u0648\u0644 \u0642\u0628\u0644 \u0627\u0644\u0646\u0638\u0631 \u0641\u064a \u0627\u0633\u062a\u062e\u062f\u0627\u0645 \u062f\u0648\u0627\u0621 GEN6050X\u060c \u0627\u0644\u0630\u064a \u064a\u064f\u0639\u0627\u0644\u062c \u062d\u0630\u0641 \u0627\u0644\u0642\u0627\u0639\u062f\u0629 \u0641\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 50\u060c \u0639\u0644\u0649 \u0646\u0637\u0627\u0642 \u0633\u0631\u064a\u0631\u064a \u0623\u0648\u0633\u0639.<\/p>\n\n\n\n<h2 id=\"future-outlook-for-gen-6050-x\" class=\"wp-block-heading\">\u0627\u0644\u062a\u0648\u0642\u0639\u0627\u062a \u0627\u0644\u0645\u0633\u062a\u0642\u0628\u0644\u064a\u0629 \u0644\u0640 GEN6050X<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0625\u0630\u0627 \u0623\u0643\u062f\u062a \u0627\u0644\u062f\u0631\u0627\u0633\u0627\u062a \u0627\u0644\u0645\u0633\u062a\u0642\u0628\u0644\u064a\u0629 \u0647\u0630\u0647 \u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0645\u0628\u0643\u0631\u0629\u060c \u0641\u0625\u0646 \u062f\u0648\u0627\u0621 GEN6050X \u0644\u062a\u0639\u062f\u064a\u0644 \u0627\u0644\u0642\u0627\u0639\u062f\u0629 \u0628\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 50 \u0642\u062f \u064a\u0645\u062b\u0644 \u062a\u0642\u062f\u0645\u0627\u064b \u0645\u0647\u0645\u0627\u064b \u0641\u064a \u0627\u0644\u0637\u0628 \u0627\u0644\u062f\u0642\u064a\u0642 \u0644\u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a.<\/strong> \u0645\u0646 \u062e\u0644\u0627\u0644 \u062a\u0635\u062d\u064a\u062d \u0627\u0644\u0637\u0641\u0631\u0629 \u0627\u0644\u062c\u064a\u0646\u064a\u0629 \u0627\u0644\u0623\u0633\u0627\u0633\u064a\u0629 \u0628\u0634\u0643\u0644 \u0645\u0628\u0627\u0634\u0631 \u0645\u0646 \u062e\u0644\u0627\u0644 \u062a\u062d\u0631\u064a\u0631 \u0627\u0644\u0642\u0627\u0639\u062f\u0629 \u0628\u062f\u0644\u0627\u064b \u0645\u0646 \u0645\u062c\u0631\u062f \u0627\u0633\u062a\u0628\u062f\u0627\u0644 \u062c\u0632\u0621 \u0645\u0646 \u0627\u0644\u062c\u064a\u0646\u060c \u0642\u062f \u064a\u0641\u062a\u062d \u0647\u0630\u0627 \u0627\u0644\u0646\u0647\u062c \u0625\u0645\u0643\u0627\u0646\u064a\u0627\u062a \u062c\u062f\u064a\u062f\u0629 \u0644\u0639\u0644\u0627\u062c \u0637\u0641\u0631\u0627\u062a DMD \u0627\u0644\u0645\u0646\u0627\u0633\u0628\u0629 \u0644\u062a\u062c\u0627\u0648\u0632 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 50. <strong>\u0639\u0644\u0649 \u0627\u0644\u0631\u063a\u0645 \u0645\u0646 \u0623\u0646 \u0627\u0644\u0639\u0644\u0627\u062c \u0644\u0627 \u064a\u0632\u0627\u0644 \u0642\u064a\u062f \u0627\u0644\u0628\u062d\u062b\u060c \u0625\u0644\u0627 \u0623\u0646 \u0627\u0644\u0628\u064a\u0627\u0646\u0627\u062a \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0627\u0644\u062a\u064a \u062a\u0645 \u062c\u0645\u0639\u0647\u0627 \u0639\u0644\u0649 \u0645\u062f\u0627\u0631 \u0639\u0627\u0645 \u0648\u0627\u062d\u062f \u062a\u0648\u0641\u0631 \u062a\u0641\u0627\u0624\u0644\u0627\u064b \u062d\u0630\u0631\u0627\u064b \u0628\u0623\u0646 \u062a\u0639\u062f\u064a\u0644 \u0627\u0644\u0642\u0648\u0627\u0639\u062f \u0642\u062f \u064a\u0635\u0628\u062d \u0625\u0636\u0627\u0641\u0629 \u0645\u0647\u0645\u0629 \u0625\u0644\u0649 \u0627\u0644\u0645\u0634\u0647\u062f \u0627\u0644\u0645\u062a\u0646\u0627\u0645\u064a \u0644\u0639\u0644\u0627\u062c\u0627\u062a \u062f\u0648\u0634\u064a\u0646.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0627\u0633\u062a\u0643\u0634\u0641 \u0627\u0644\u0622\u0646<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0645\u0631\u0643\u0632 \u0627\u0644\u062a\u062c\u0627\u0631\u0628 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 DMDWarrioR<\/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\/ar\/wp-json\/wp\/v2\/posts\/8291","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/comments?post=8291"}],"version-history":[{"count":2,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/8291\/revisions"}],"predecessor-version":[{"id":8294,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/8291\/revisions\/8294"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media\/8293"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media?parent=8291"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/categories?post=8291"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/tags?post=8291"}],"curies":[{"name":"\u0648\u0648\u0631\u062f\u0628\u0631\u064a\u0633","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}