{"id":3807,"date":"2025-05-10T21:42:26","date_gmt":"2025-05-10T18:42:26","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3807"},"modified":"2025-05-10T23:36:44","modified_gmt":"2025-05-10T20:36:44","slug":"micro-dystrophin","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/micro-dystrophin\/","title":{"rendered":"\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff1a\u6bcf\u4e2a\u4eba\u90fd\u5728\u601d\u8003\u7684 5 \u4e2a\u5173\u952e\u95ee\u9898"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u548c\u5168\u957f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u5728\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u4e2d\u53d1\u6325\u7740\u81f3\u5173\u91cd\u8981\u7684\u4f5c\u7528\u3002\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u662f\u4e00\u79cd\u5bfc\u81f4\u8fdb\u884c\u6027\u808c\u65e0\u529b\u7684\u9057\u4f20\u6027\u75be\u75c5\u3002\u867d\u7136\u5168\u957f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u662f DMD \u60a3\u8005\u6240\u7f3a\u5931\u7684\u86cb\u767d\u8d28\uff0c\u4f46\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7597\u6cd5\u6b63\u9010\u6e10\u6210\u4e3a\u4e00\u79cd\u6709\u524d\u666f\u7684\u66ff\u4ee3\u7597\u6cd5\uff0c\u4e3a DMD \u60a3\u8005\u5e26\u6765\u6f5c\u5728\u76ca\u5904\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u62e5\u670979\u4e2a\u5916\u663e\u5b50\uff08\u5373\u4e3a\u7ec6\u80de\u63d0\u4f9b\u5236\u9020\u86cb\u767d\u8d28\u6307\u4ee4\u7684DNA\u7247\u6bb5\uff09\uff0c\u662f\u4eba\u4f53\u4e2d\u6700\u5927\u7684\u57fa\u56e0\u3002\u76ee\u524d\uff0c\u7531\u4e8e\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7684\u5927\u5c0f\u4ee5\u53ca\u7528\u4e8e\u5c06\u8f6c\u57fa\u56e0\u8f6c\u79fb\u5230\u7ec6\u80de\u4e2d\u7684\u8f7d\u4f53\u7684\u5c3a\u5bf8\u8f83\u5c0f\uff0c\u56e0\u6b64\u65e0\u6cd5\u5c06\u5b8c\u6574\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u9012\u9001\u5230\u7ec6\u80de\u4e2d\u3002\u4e3a\u4e86\u6784\u5efa\u529f\u80fd\u66f4\u77ed\u7684\u86cb\u767d\u8d28\uff0c\u7814\u7a76\u4eba\u5458\u6784\u5efa\u4e86\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8f6c\u57fa\u56e0\uff0c\u4ee5\u4fdd\u7559\u5fc5\u8981\u7684\u9057\u4f20\u4fe1\u606f\u3002\u2014\u2014\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/dystrophin-gene\/\" target=\"_blank\" data-type=\"post\" data-id=\"3785\" rel=\"noreferrer noopener\">\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\uff1a\u529f\u80fd\u3001\u75be\u75c5\u548c\u6cbb\u7597\u8fdb\u5c55<\/a><\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u76ee\u5f55<\/h2><nav><ul><li><a href=\"#mechanism-of-micro-dystrophin\">\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u673a\u5236<\/a><\/li><li><a href=\"#micro-dystrophin-transgenes\">\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8f6c\u57fa\u56e0<\/a><\/li><li><a href=\"#micro-dystrophin-in-becker-muscular-dystrophy\">\u8d1d\u514b\u5c14\u578b\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d\u7684\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d<\/a><\/li><li><a href=\"#challenges-of-micro-dystrophin\">\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u6311\u6218<\/a><\/li><li><a href=\"#unknown-answers-about-micro-dystrophin\">\u5173\u4e8e\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u672a\u77e5\u7b54\u6848<\/a><\/li><li><a href=\"#the-problem-of-reaching-all-cells\">\u8986\u76d6\u6240\u6709\u7ec6\u80de\u7684\u95ee\u9898<\/a><\/li><li><a href=\"#relationship-between-micro-dystrophin-and-ck-ast-and-alt\">\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4e0eCK\u3001AST\u3001ALT\u7684\u5173\u7cfb<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"mechanism-of-micro-dystrophin\">\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u673a\u5236<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5bfc\u81f4\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7a81\u53d8\u4f1a\u5bfc\u81f4\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u5408\u6210\u51e0\u4e4e\u6216\u5b8c\u5168\u505c\u6b62\u3002\u5c06\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7684\u529f\u80fd\u6027\u62f7\u8d1d\u63d0\u4f9b\u7ed9\u808c\u8089\u7ec6\u80de\uff0c\u4f7f\u5176\u80fd\u591f\u5f00\u59cb\u4ea7\u751f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u662f\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4e00\u79cd\u53ef\u80fd\u6cbb\u7597\u65b9\u6cd5\u3002\u8fd9\u6b63\u662f\u4f7f\u7528\u57fa\u56e0\u6cbb\u7597\u4ea7\u54c1\u66ff\u4ee3\u7ec6\u80de\u7684\u7406\u5ff5\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u901a\u8fc7\u5f15\u5165\u8f6c\u57fa\u56e0\uff08\u5373DNA\u6307\u4ee4\uff0c\u6307\u5bfc\u8eab\u4f53\u5236\u9020\u86cb\u767d\u8d28\uff09\u53ef\u4ee5\u542f\u52a8\u7f3a\u5931\u86cb\u767d\u8d28\u7684\u751f\u6210\u3002\u60a3\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e14\u643a\u5e26\u591a\u79cd\u57fa\u56e0\u53d8\u5f02\u7684\u60a3\u8005\u6709\u671b\u9009\u62e9\u4f7f\u7528\u5fae\u6297\u808c\u840e\u7f29\u86cb\u767d\u8fdb\u884c\u57fa\u56e0\u6cbb\u7597\uff0c\u56e0\u4e3a\u5b83\u53d6\u4ee3\u7684\u662f\u57fa\u56e0\uff0c\u800c\u4e0d\u662f\u7ea0\u6b63\u57fa\u56e0\u7f3a\u9677\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"micro-dystrophin-transgenes\">\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8f6c\u57fa\u56e0<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5c06\u8f6c\u57fa\u56e0\u9012\u9001\u5230\u7ec6\u80de\u4e2d\u6240\u9700\u7684\u8f7d\u4f53\u6216\u8f6c\u8fd0\u86cb\u767d\u592a\u5c0f\uff0c\u65e0\u6cd5\u5305\u88c5\u5b8c\u6574\u7684\u6297\u808c\u840e\u7f29\u86cb\u767d\u57fa\u56e0\uff08\u800c\u4ea7\u751f\u5168\u957f\u6297\u808c\u840e\u7f29\u86cb\u767d\u662f\u5fc5\u9700\u7684\uff09\uff0c\u8fd9\u5bf9\u73b0\u6709\u7684\u57fa\u56e0\u66ff\u6362\u6280\u672f\u662f\u4e00\u4e2a\u91cd\u5927\u969c\u788d\u3002\u56e0\u6b64\uff0c\u7814\u7a76\u4eba\u5458\u5f00\u53d1\u4e86\u6240\u8c13\u7684\u201c\u5fae\u6297\u808c\u840e\u7f29\u86cb\u767d\u8f6c\u57fa\u56e0\u201d\u3002\u8fd9\u4e9b\u201c\u5fae\u6297\u808c\u840e\u7f29\u86cb\u767d\u8f6c\u57fa\u56e0\u201d\u53ef\u4ee5\u6307\u793a\u7ec6\u80de\u4ea7\u751f\u622a\u77ed\u4f46\u6709\u529f\u80fd\u7684\u86cb\u767d\u8d28\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"micro-dystrophin-in-becker-muscular-dystrophy\">\u8d1d\u514b\u5c14\u578b\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d\u7684\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u67d0\u4e9b\u8d1d\u514b\u578b\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u4e2d\u89c2\u5bdf\u5230\u7684\u529f\u80fd\u6027\u4f46\u622a\u77ed\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u542f\u53d1\u4e86\u8fd9\u4e9b\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u53d1\u660e\u3002\u5c3d\u7ba1\u8fd9\u4e9b\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4e0e\u67d0\u4e9b\u8d1d\u514b\u578b\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u4f53\u5185\u4ea7\u751f\u7684\u86cb\u767d\u8d28\u76f8\u4f3c\uff0c\u4f46\u5f88\u96be\u5c06\u63a5\u53d7\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u66ff\u6362\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u4e0e\u8d1d\u514b\u578b\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u8fdb\u884c\u6bd4\u8f83\u3002\u8fd9\u662f\u56e0\u4e3a\u8fd9\u4e9b\u60a3\u8005\u81ea\u51fa\u751f\u4ee5\u6765\uff0c\u6240\u6709\u808c\u8089\u7ec6\u80de\u4e2d\u90fd\u5728\u4ea7\u751f\u8fd9\u4e9b\u86cb\u767d\u8d28\u3002\u8d1d\u514b\u578b\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u4e0d\u592a\u53ef\u80fd\u4ece\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u57fa\u56e0\u6cbb\u7597\u4e2d\u83b7\u76ca\uff0c\u4e5f\u4e0d\u9002\u5408\u63a5\u53d7\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u57fa\u56e0\u6cbb\u7597\uff0c\u56e0\u4e3a\u4ed6\u4eec\u901a\u5e38\u4f1a\u81ea\u884c\u4ea7\u751f\u4e00\u79cd\u5fae\u5c0f\u7684\u3001\u534a\u529f\u80fd\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002\u2014\u2014\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/differences-between-dmd-and-bmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD \u548c BMD \u4e4b\u95f4\u6709\u4ec0\u4e48\u533a\u522b\uff1f<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"challenges-of-micro-dystrophin\">\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u6311\u6218<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u86cb\u767d\u8d28\u7684\u7f29\u77ed\u5e26\u6765\u4e86\u65b0\u7684\u96be\u9898\u3002\u4f8b\u5982\uff0c\u8fd9\u4e9b\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u9700\u8981\u6392\u9664\u6784\u6210\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7684\u4f17\u591a\u5916\u663e\u5b50\u3002\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u5728\u4f53\u5185\u7684\u4f17\u591a\u91cd\u8981\u529f\u80fd\u4e4b\u4e00\u662f\u7a33\u5b9a\u808c\u8089\u7ec6\u80de\u819c\u3002\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4e0e\u808c\u8089\u7ec6\u80de\u819c\u4e0a\u8bb8\u591a\u5176\u4ed6\u6709\u76ca\u86cb\u767d\u4e4b\u95f4\u7684\u76f8\u4e92\u4f5c\u7528\uff0c\u4f7f\u7ec6\u80de\u819c\u7a33\u5b9a\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5c3d\u7ba1\u7814\u7a76\u4eba\u5458\u7aed\u5c3d\u5168\u529b\u8c28\u614e\u9009\u62e9\u54ea\u4e9b\u7247\u6bb5\u88ab\u7eb3\u5165\uff0c\u4f46\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u4e2d\u67d0\u4e9b\u4e0e\u5176\u4ed6\u86cb\u767d\u8d28\u76f8\u4e92\u4f5c\u7528\u7684\u7247\u6bb5\u6700\u7ec8\u4f1a\u5728\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8f6c\u57fa\u56e0\u4e2d\u7f3a\u5931\u3002\u56e0\u6b64\uff0c\u62e5\u6709\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u76ca\u5904\u4e0d\u5982\u62e5\u6709\u5168\u957f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u4f46\u8fd9\u53ef\u80fd\u4ecd\u7136\u6bd4\u6ca1\u6709\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8981\u597d\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"unknown-answers-about-micro-dystrophin\">\u5173\u4e8e\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u672a\u77e5\u7b54\u6848<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u808c\u8089\u7ec6\u80de\u80fd\u591f\u6301\u7eed\u4ea7\u751f\u5fae\u6297\u808c\u840e\u7f29\u86cb\u767d\u591a\u4e45\uff0c\u6216\u8005\u8f6c\u57fa\u56e0\u7684\u6301\u4e45\u529b\u5982\u4f55\uff0c\u662f\u5fae\u6297\u808c\u840e\u7f29\u86cb\u767d\u57fa\u56e0\u66ff\u6362\u8fc7\u7a0b\u4e2d\u7684\u53e6\u4e00\u4e2a\u672a\u77e5\u6570\u3002\u4e2a\u4f53\u7684DNA\u4e0d\u4f1a\u6574\u5408\u8f6c\u57fa\u56e0\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u52a8\u7269\u7814\u7a76\u5df2\u7ecf\u5b8c\u6210\uff0c\u4f46\u4e3a\u4e86\u786e\u5b9a\u8f6c\u57fa\u56e0\u5728\u7ec6\u80de\u4e2d\u505c\u7559\u591a\u957f\u65f6\u95f4\uff0c\u6211\u4eec\u5fc5\u987b\u8bc4\u4f30\u5176\u5728\u4eba\u7c7b\u4f53\u5185\u7684\u6301\u4e45\u6027\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6709\u51e0\u4e2a\u56e0\u7d20\u53ef\u80fd\u4f1a\u5f71\u54cd\u8010\u4e45\u6027\u3002\u4f8b\u5982\uff0c\u7a00\u91ca\u53ef\u80fd\u5bfc\u81f4\u8f6c\u57fa\u56e0\u4e22\u5931\u3002\u968f\u7740\u5e74\u9f84\u7684\u589e\u957f\uff0c\u4eba\u4f53\u4f1a\u6bd4\u5e74\u8f7b\u65f6\u589e\u957f\u66f4\u591a\u7684\u808c\u8089\u3002\u5fae\u6297\u808c\u840e\u7f29\u86cb\u767d\u8f6c\u57fa\u56e0\u6216\u86cb\u767d\u8d28\u53ef\u80fd\u4e0d\u5b58\u5728\u4e8e\u65b0\u5f62\u6210\u7684\u808c\u8089\u7ec6\u80de\u4e2d\uff0c\u8fd9\u53ef\u80fd\u5bfc\u81f4\u5b83\u4eec\u8868\u73b0\u5f97\u50cf\u6b63\u5e38\u7684\u8425\u517b\u4e0d\u826f\u808c\u8089\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u808c\u8089\u635f\u4f24\u53ef\u80fd\u662f\u5f71\u54cd\u836f\u7269\u6301\u4e45\u6027\u7684\u53e6\u4e00\u4e2a\u56e0\u7d20\u3002\u643a\u5e26\u5fae\u6297\u808c\u840e\u7f29\u86cb\u767d\u8f6c\u57fa\u56e0\u7684\u7ec6\u80de\u53ef\u80fd\u4f1a\u56e0\u6d88\u8017\u6216\u635f\u4f24\u808c\u8089\u7684\u4f53\u529b\u6d3b\u52a8\u800c\u4e22\u5931\u3002\u4e86\u89e3\u836f\u7269\u7684\u6301\u4e45\u6027\u4ee5\u53ca\u786e\u5b9a\u662f\u5426\u9700\u8981\u4ee5\u53ca\u4f55\u65f6\u9700\u8981\u91cd\u65b0\u7ed9\u836f\uff0c\u5c06\u53d6\u51b3\u4e8e\u5bf9\u63a5\u53d7\u5fae\u6297\u808c\u840e\u7f29\u86cb\u767d\u57fa\u56e0\u66ff\u4ee3\u6cbb\u7597\u7684\u60a3\u8005\u8fdb\u884c\u76d1\u6d4b\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"the-problem-of-reaching-all-cells\">\u8986\u76d6\u6240\u6709\u7ec6\u80de\u7684\u95ee\u9898<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u540c\u6837\u91cd\u8981\u7684\u662f\u8981\u8bb0\u4f4f\uff0c\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u53ea\u80fd\u7531\u643a\u5e26\u8f6c\u57fa\u56e0\u7684\u6d3b\u808c\u7ec6\u80de\u4ea7\u751f\u3002\u8fd9\u610f\u5473\u7740\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8f6c\u57fa\u56e0\u5bf9\u4efb\u4f55\u5df2\u88ab\u7ea4\u7ef4\u5316\u6216\u8102\u80aa\u53d6\u4ee3\u7684\u808c\u8089\u7ec4\u7ec7\u65e0\u6548\u3002\u968f\u7740\u8d8a\u6765\u8d8a\u591a\u5904\u4e8e\u4e0d\u540c\u75be\u75c5\u9636\u6bb5\u7684\u60a3\u8005\u63a5\u53d7\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u6cbb\u7597\uff0c\u4eba\u4eec\u5c06\u53d1\u73b0\u662f\u5426\u6709\u6700\u4f73\u65f6\u673a\u5e94\u7528\u8fd9\u79cd\u7279\u5b9a\u7684\u57fa\u56e0\u66ff\u4ee3\u6280\u672f\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"relationship-between-micro-dystrophin-and-ck-ast-and-alt\">\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4e0eCK\u3001AST\u3001ALT\u7684\u5173\u7cfb<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u6cbb\u7597\u662f\u5426\u4f1a\u5c06 CK\u3001AST \u548c ALT \u964d\u4f4e\u81f3\u6b63\u5e38\u6c34\u5e73\uff1f<\/strong> EMBARK \u4e09\u671f\u968f\u673a\u8bd5\u9a8c\u7ed3\u679c\u663e\u793a\uff0cdelandistrogene moxeparvovec (Elevidys) \u7684 CK\u3001ALT \u548c AST \u672a\u964d\u81f3\u53ef\u63a5\u53d7\u7684\u6b63\u5e38\u6c34\u5e73\u3002\u2014\u2014 \u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/www.nature.com\/articles\/s41591-024-03304-z\" target=\"_blank\" rel=\"noreferrer noopener\">EMBARK 3\u671f\u968f\u673a\u8bd5\u9a8c<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>Micro dystrophin and full-length dystrophin play crucial roles in treating Duchenne muscular dystrophy (DMD), a genetic disorder that causes progressive muscle weakness. While full-length dystrophin is the protein missing in individuals with DMD, micro-dystrophin therapy is emerging as a promising alternative, offering potential benefits for those with the condition. With 79 exons\u2014the sections of DNA [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3820,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[66,61],"class_list":["post-3807","post","type-post","status-publish","format-standard","has-post-thumbnail","category-dmd","tag-duchenne-muscular-dystrophy","tag-gene-therapy"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3807","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/comments?post=3807"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3807\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3820"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3807"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3807"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3807"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}