{"id":3561,"date":"2025-04-08T11:41:34","date_gmt":"2025-04-08T08:41:34","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3561"},"modified":"2025-04-08T11:41:36","modified_gmt":"2025-04-08T08:41:36","slug":"the-immune-system-may-be-decreasing-the-effectiveness-of-gene-therapy-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/the-immune-system-may-be-decreasing-the-effectiveness-of-gene-therapy-for-duchenne-muscular-dystrophy\/","title":{"rendered":"\u514d\u75ab\u7cfb\u7edf\u53ef\u80fd\u4f1a\u964d\u4f4e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u7684\u6709\u6548\u6027"},"content":{"rendered":"

\u7531\u4e8e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u8005\u7f3a\u4e4f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u901a\u8fc7\u57fa\u56e0\u7597\u6cd5\u91cd\u65b0\u5f15\u5165\u8be5\u86cb\u767d\u53ef\u80fd\u4f1a\u5bfc\u81f4\u514d\u75ab\u7cfb\u7edf\u5c06\u5176\u8bef\u8ba4\u4e3a\u6297\u539f\u6216\u5916\u6765\u86cb\u767d\u8d28\uff0c\u4ece\u800c\u5bfc\u81f4\u7834\u574f\u4ea7\u751f\u8be5\u86cb\u767d\u7684\u808c\u8089\u7684\u53cd\u5e94\u5e76\u5371\u53ca\u6cbb\u7597\u7684\u6548\u679c\u3002<\/p>\n\n\n\n

\u514d\u75ab\u7cfb\u7edf\u53ef\u80fd\u6291\u5236\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f<\/h2>\n\n\n\n

\u7814\u7a76\u7ed3\u679c\u8868\u660e\uff0c\u65e8\u5728\u6062\u590d\u7f3a\u5931\u86cb\u767d\u8d28\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u57fa\u56e0\u7597\u6cd5\uff08\u4f8b\u5982 Elevidys\uff08delandistrogene moxeparvovec\uff09\uff09\u53ef\u80fd\u4f1a\u53d7\u5230\u4eba\u4f53\u514d\u75ab\u7cfb\u7edf\u7684\u963b\u788d\uff0c\u4ece\u800c\u53ef\u80fd\u963b\u6b62\u5176\u53d1\u6325\u4efb\u4f55\u6cbb\u7597\u6548\u679c\u3002\uff08\u5173\u4e8e Elevidys \u7684\u5e38\u89c1\u95ee\u9898\u89e3\u7b54<\/a>)<\/p>\n\n\n\n

DMD \u662f\u4e00\u79cd\u4f7f\u4eba\u8870\u5f31\u7684\u9057\u4f20\u6027\u75be\u75c5\uff0c\u4e3b\u8981\u5f71\u54cd\u7537\u5b69\u3002\u8be5\u75c5\u4f1a\u5bfc\u81f4\u8fc7\u65e9\u6b7b\u4ea1\u3001\u9752\u6625\u671f\u65e9\u671f\u8fd0\u52a8\u80fd\u529b\u4e27\u5931\u4ee5\u53ca\u6301\u7eed\u7684\u808c\u8089\u840e\u7f29\u3002\u7531\u4e8e\u76ee\u524d\u5c1a\u65e0\u6cbb\u7597\u65b9\u6cd5\uff0c\u57fa\u56e0\u7597\u6cd5\u88ab\u89c6\u4e3a\u6700\u6709\u5e0c\u671b\u53d6\u5f97\u8fdb\u5c55\u7684\u9014\u5f84\u4e4b\u4e00\u3002<\/p>\n\n\n\n

Elevidys \u7597\u6cd5\u53ef\u6062\u590d\u4e00\u79cd\u540d\u4e3a\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u91cd\u8981\u808c\u8089\u86cb\u767d\uff0c\u4eba\u4eec\u5bf9\u5176\u5c06\u6539\u53d8\u60a3\u8005\u7684\u751f\u6d3b\u5bc4\u4e88\u539a\u671b\uff0c\u8be5\u7597\u6cd5\u4e8e 2024 \u5e74\u83b7\u5f97\u4e86\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\u7684\u6269\u5927\u6279\u51c6\u3002\u800c\u7b2c\u4e09\u9636\u6bb5 EMBARK \u8bd5\u9a8c\u7684\u6700\u65b0\u7ed3\u679c\u5219\u63cf\u7ed8\u4e86\u4e00\u5e45\u66f4\u4e3a\u4e25\u5cfb\u7684\u56fe\u666f\u3002\uff08EMBARK Elevidys \u7814\u7a76<\/a>)<\/p>\n\n\n\n

DMD \u57fa\u56e0\u6cbb\u7597\u4e2d\u7684\u514d\u75ab\u7cfb\u7edf\u56f0\u5883<\/h2>\n\n\n\n

\u7ed3\u679c\u5e76\u4e0d\u4ee4\u4eba\u6ee1\u610f\u3002\u636e\u6734\u8328\u8305\u65af\u5927\u5b66\u533b\u5b66\u3001\u836f\u5b66\u548c\u751f\u7269\u79d1\u5b66\u5b66\u9662 GT \u8bba\u6587\u7684\u8d44\u6df1\u4f5c\u8005 Darek Gorecki \u6559\u6388\u79f0\uff0c\u514d\u75ab\u7cfb\u7edf\u5bf9\u91cd\u65b0\u5f15\u5165\u7684\u86cb\u767d\u8d28\u7684\u53cd\u5e94\u53ef\u80fd\u662f\u6cbb\u7597\u672a\u80fd\u660e\u663e\u6539\u5584\u808c\u8089\u529f\u80fd\u7684\u539f\u56e0\u3002<\/p>\n\n\n\n

\u8fd9\u79cd\u53cd\u5e94\u5728\u67d0\u4e9b\u60a3\u8005\u4e2d\u53ef\u80fd\u7279\u522b\u5e38\u89c1\u3002\u6162\u6027\u808c\u8089\u708e\u75c7\u3001\u901a\u8fc7\u6240\u8c13\u7684\u201c\u56de\u590d\u7ea4\u7ef4\u201d\u63a5\u89e6\u5fae\u91cf\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u6216\u5b58\u5728\u5728\u65e9\u671f\u53d1\u80b2\u8fc7\u7a0b\u4e2d\u901a\u5e38\u4e0d\u4f1a\u88ab\u5220\u9664\u7684\u53cd\u5e94\u6027\u514d\u75ab\u7ec6\u80de\u90fd\u662f\u98ce\u9669\u56e0\u7d20\u3002<\/p>\n\n\n\n

\u8fd9\u9879\u7814\u7a76\u8fdb\u4e00\u6b65\u5f3a\u8c03\u4e86 DMD \u7684\u590d\u6742\u6027\u3002\u4efb\u4f55\u6709\u6548\u7684\u6cbb\u7597\u65b9\u6cd5\u90fd\u53ef\u80fd\u9700\u8981\u8003\u8651\u5230\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u5168\u8eab\u5f71\u54cd\uff0c\u8fd9\u79cd\u5f71\u54cd\u4e0d\u4ec5\u9650\u4e8e\u808c\u8089\uff0c\u8fd8\u5305\u62ec\u5927\u8111\u3001\u9aa8\u9abc\u548c\u5176\u4ed6\u7ec4\u7ec7\u3002<\/p>\n\n\n\n

\u5c3d\u7ba1\u5982\u6b64\uff0c\u79d1\u5b66\u5bb6\u4eec\u8fd8\u662f\u62b1\u6709\u5e0c\u671b\u3002\u6208\u96f7\u8328\u57fa\u6559\u6388\u7ee7\u7eed\u8bf4\u9053\uff1a\u201cDMD \u7684\u57fa\u56e0\u6cbb\u7597\u5c1a\u672a\u5b8c\u6210\u3002\u201d\u201c\u8fd9\u63d0\u9192\u6211\u4eec\u5fc5\u987b\u66f4\u8fdb\u4e00\u6b65\uff0c\u4e3a\u6bcf\u4f4d\u60a3\u8005\u91cf\u8eab\u5b9a\u5236\u6cbb\u7597\u65b9\u6848\uff0c\u5e76\u76f4\u63a5\u89e3\u51b3\u514d\u75ab\u539f\u6027\u95ee\u9898\u3002\u201d<\/p>\n\n\n\n

\u8fd9\u9879\u5de5\u4f5c\u4f7f\u4eba\u4eec\u8d8a\u6765\u8d8a\u8ba4\u8bc6\u5230\uff0c\u4ece\u5b9e\u9a8c\u5ba4\u5230\u6539\u53d8\u751f\u6d3b\u7684\u836f\u7269\u7684\u65c5\u7a0b\u7edd\u975e\u7b80\u5355\uff0c\u5373\u4f7f\u662f\u6700\u5148\u8fdb\u7684\u57fa\u56e0\u7597\u6cd5\u4e5f\u9700\u8981\u5b9a\u5236\u7684\u3001\u591a\u5b66\u79d1\u7684\u65b9\u6cd5\u3002<\/p>\n\n\n\n

\u8003\u8651\u5230\u8fd9\u9879\u7814\u7a76\u548c\u8bba\u6587\uff0c\u6211\u4eec\u96be\u9053\u4e0d\u5e94\u8be5\u95ee\u4e3a\u4ec0\u4e48Elevidys\u57fa\u56e0\u7597\u6cd5\u7684\u552e\u4ef7\u4e3a$3\u767e\u4e07\u7f8e\u5143\u5417\uff1f<\/strong><\/p>\n\n\n\n

\u9605\u8bfb\u66f4\u591a\uff1a<\/strong> Elevidys \u57fa\u56e0\u6cbb\u7597\u4e34\u5e8a\u8bd5\u9a8c\u8bc4\u8bba\uff1aElevidys \u503c\u5f97\u82b1\u8d39\u5417\uff1f<\/a><\/p>","protected":false},"excerpt":{"rendered":"

Since patients with Duchenne Muscular Dystrophy (DMD) lack dystrophin, reintroducing it through gene therapy may cause the immune system to mistake it for an antigen, or foreign protein, which could lead to a reaction that destroys the muscle that produces it and jeopardize the effectiveness of the treatment. The Immune System May Inhibit Dystrophin Production […]<\/p>\n","protected":false},"author":1,"featured_media":3567,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[33,61,349,203,65],"class_list":["post-3561","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-elevidys","tag-gene-therapy","tag-immune-system","tag-roche","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3561","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=3561"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3561\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3567"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3561"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3561"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3561"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}