{"id":4151,"date":"2025-07-18T12:17:47","date_gmt":"2025-07-18T09:17:47","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4151"},"modified":"2025-07-18T12:17:49","modified_gmt":"2025-07-18T09:17:49","slug":"uw-medicine-announces-new-gene-therapy-research-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/uw-medicine-announces-new-gene-therapy-research-for-duchenne-muscular-dystrophy\/","title":{"rendered":"UW Medicine\u5ba3\u5e03\u5f00\u5c55\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u65b0\u57fa\u56e0\u6cbb\u7597\u7814\u7a76"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u5bf9\u4e8e\u60a3\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u4eba\u6765\u8bf4\uff0c\u4e00\u79cd\u65b0\u578b\u57fa\u56e0\u7597\u6cd5\u6709\u671b\u963b\u6b62\u808c\u8089\u6d41\u5931\uff0c\u5e76\u53ef\u80fd\u5728\u672a\u6765\u4fee\u590d\u53d7\u635f\u7684\u808c\u8089\u3002 <strong>UW Medicine \u8868\u793a\uff0c\u8be5\u7597\u6cd5\u7684\u4e0b\u4e00\u6b65\u5c06\u8fdb\u884c\u4eba\u4f53\u8bd5\u9a8c\uff0c\u9884\u8ba1\u5c06\u5728\u5927\u7ea6\u4e24\u5e74\u540e\u5f00\u59cb\u3002<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u9879\u7531UW Medicine\u57fa\u56e0\u7f16\u8f91\u7684\u7814\u7a76\u65e8\u5728\u901a\u8fc7\u5728\u7a7f\u68ad\u8f7d\u4f53\u4e2d\u5f15\u5165\u4e00\u7cfb\u5217\u86cb\u767d\u8d28\u5305\u6765\u66ff\u6362\u808c\u8089\u4e2d\u53d7\u635f\u7684DMD\u57fa\u56e0\u3002\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u4f53\u5185\u7f3a\u4e4f\u7684\u6297\u808c\u840e\u7f29\u86cb\u767d\uff08dystrophin\uff09\u5c06\u7531\u989d\u5916\u7684\u9057\u4f20\u5bc6\u7801\u4ea7\u751f\u3002<\/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=\"#currently-no-cure-for-dmd\">\u76ee\u524d\uff0cDMD \u5c1a\u65e0\u6cbb\u6108\u65b9\u6cd5<\/a><\/li><li><a href=\"#who-is-dr-jeffrey-chamberlain\">\u6770\u5f17\u91cc\u00b7\u5f20\u4f2f\u4f26\u535a\u58eb\u662f\u8c01\uff1f<\/a><\/li><li><a href=\"#what-is-uw-medicines-new-gene-study\">UW Medicine \u7684\u65b0\u57fa\u56e0\u7814\u7a76\u662f\u4ec0\u4e48\uff1f<\/a><\/li><li><a href=\"#human-trials-should-begin-in-approximately-two-years\">\u4eba\u4f53\u8bd5\u9a8c\u9884\u8ba1\u5c06\u5728\u5927\u7ea6\u4e24\u5e74\u5185\u5f00\u59cb<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"currently-no-cure-for-dmd\">\u76ee\u524d\uff0cDMD \u5c1a\u65e0\u6cbb\u6108\u65b9\u6cd5<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u76ee\u524d\uff0c\u8fd9\u79cd\u75be\u75c5\u5c1a\u65e0\u5df2\u77e5\u7684\u6cbb\u6108\u65b9\u6cd5\uff0c\u73b0\u6709\u7684\u836f\u7269\u548c\u7597\u6cd5\u53ea\u80fd\u51cf\u7f13\u5176\u53d1\u5c55\u3002\u7531\u4e8e\u7f3a\u9677\u57fa\u56e0\u4f4d\u4e8eX\u67d3\u8272\u4f53\u4e0a\uff0c\u56e0\u6b64\u672c\u7814\u7a76\u7684\u5bf9\u8c61\u2014\u2014\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u5168\u90e8\u4e3a\u7537\u6027\u3002\u4ed6\u4eec\u4ece\u56db\u5c81\u5f00\u59cb\u51fa\u73b0\u75c7\u72b6\uff0c\u901a\u5e38\u5728\u4e8c\u4e09\u5341\u5c81\u65f6\u53bb\u4e16\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5173\u4e8e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u65b0\u57fa\u56e0\u7597\u6cd5\u7684\u7814\u7a76\u7ed3\u679c\u53d1\u8868\u5728\u300a\u81ea\u7136\u300b\u6742\u5fd7\u4e0a\u3002 <a href=\"https:\/\/www.nature.com\/articles\/s41586-024-07710-8\" target=\"_blank\" rel=\"noreferrer noopener\">\u9605\u8bfb\u66f4\u591a<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"who-is-dr-jeffrey-chamberlain\">\u6770\u5f17\u91cc\u00b7\u5f20\u4f2f\u4f26\u535a\u58eb\u662f\u8c01\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u7814\u7a76\u7684\u4e3b\u8981\u4f5c\u8005\u662fUW Medicine\u7684\u9057\u4f20\u5b66\u5bb6\u548c\u795e\u7ecf\u5b66\u5bb6\u6770\u5f17\u91cc\u00b7\u5f20\u4f2f\u4f26\u535a\u58eb\uff0c\u4ed6\u6bd5\u751f\u81f4\u529b\u4e8e\u5bfb\u627e\u6cbb\u7597\u6216\u6cbb\u6108\u808c\u8089\u840e\u7f29\u75c7\u7684\u65b9\u6cd5\u3002\u4ed6\u62c5\u4efb\u534e\u76db\u987f\u5927\u5b66\u533b\u5b66\u9662\u9ea6\u8003\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6350\u8d60\u57fa\u91d1\u4e3b\u5e2d\uff0c\u5e76\u62c5\u4efb\u97e6\u5c14\u65af\u901a\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7814\u7a76\u4e2d\u5fc3\u4e3b\u4efb\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5f20\u4f2f\u4f26\u5b9e\u9a8c\u5ba4\u7684\u5206\u5b50\u751f\u7269\u5b66\u5bb6 Hichem Tasfaout \u535a\u58eb\u8d1f\u8d23\u6307\u5bfc\u8fd9\u9879\u7814\u7a76\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4ed6\u58f0\u79f0\uff0c\u9700\u8981\u4fee\u590d\u7684\u57fa\u56e0\u201c\u662f\u81ea\u7136\u754c\u4e2d\u6700\u5927\u7684\u57fa\u56e0\u201d\uff0c\u8fd9\u4e00\u4e8b\u5b9e\u8fc4\u4eca\u4e3a\u6b62\u963b\u788d\u4e86\u7814\u7a76\u4eba\u5458\u53d6\u5f97\u8fdb\u5c55\u3002\u8fc4\u4eca\u4e3a\u6b62\uff0c\u5c06\u5fc5\u8981\u7684\u86cb\u767d\u8d28\u4fee\u590d\u9012\u9001\u5230\u808c\u8089\u53ca\u5176\u57fa\u56e0\u4e2d\u662f\u4e0d\u53ef\u80fd\u7684\u3002<\/p>\n\n\n\n<figure class=\"wp-block-embed is-type-video is-provider-youtube wp-block-embed-youtube wp-embed-aspect-16-9 wp-has-aspect-ratio\"><div class=\"wp-block-embed__wrapper\">\n<div class=\"youtube-embed\" data-video_id=\"0Shrd9SW6O0\"><iframe loading=\"lazy\" title=\"\u808c\u8089\u840e\u7f29\u75c7\u7684\u65b0\u57fa\u56e0\u7597\u6cd5\" width=\"696\" height=\"392\" src=\"https:\/\/www.youtube.com\/embed\/0Shrd9SW6O0?feature=oembed&#038;enablejsapi=1\" frameborder=\"0\" allow=\"accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture; web-share\" referrerpolicy=\"strict-origin-when-cross-origin\" allowfullscreen><\/iframe><\/div>\n<\/div><\/figure>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-uw-medicines-new-gene-study\">UW Medicine \u7684\u65b0\u57fa\u56e0\u7814\u7a76\u662f\u4ec0\u4e48\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u79cd\u65b0\u65b9\u6cd5\u5229\u7528\u591a\u79cd\u817a\u76f8\u5173\u75c5\u6bd2\u8f7d\u4f53\uff08AAV\uff0c\u7531\u75c5\u6bd2\u4ea7\u751f\u7684\u5fae\u578b\u7a7f\u68ad\u673a\uff0c\u7528\u4e8e\u5c06\u57fa\u56e0\u6cbb\u7597\u8fd0\u9001\u5230\u4eba\u4f53\u7ec6\u80de\u4e2d\uff09\uff0c\u5728\u5c0f\u9f20\u6a21\u578b\u4e2d\u5df2\u663e\u793a\u51fa\u826f\u597d\u7684\u524d\u666f\u3002 <strong>\u8fd9\u79cd\u57fa\u56e0\u7597\u6cd5\u91c7\u7528\u7684\u662f\u4e00\u7cfb\u5217 AAV\uff0c\u800c\u4e0d\u662f\u5355\u4e2a AAV\uff0c\u5b83\u4ece\u808c\u8089\u4e2d\u63d0\u53d6\u6cbb\u7597\u6027\u86cb\u767d\u8d28\u7684\u7247\u6bb5\uff0c\u5e76\u5d4c\u5165\u6307\u4ee4\uff0c\u5f00\u59cb\u5728\u4f53\u5185\u7ec4\u5408\u6240\u9700\u7684\u57fa\u56e0\u4fee\u590d\u3002<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"human-trials-should-begin-in-approximately-two-years\">\u4eba\u4f53\u8bd5\u9a8c\u9884\u8ba1\u5c06\u5728\u5927\u7ea6\u4e24\u5e74\u5185\u5f00\u59cb<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5f20\u4f2f\u4f26\u8868\u793a\uff0c\u4eba\u4f53\u8bd5\u9a8c\u662f\u8be5\u7597\u6cd5\u7684\u4e0b\u4e00\u9636\u6bb5\uff0c\u9884\u8ba1\u5c06\u5728\u4e24\u5e74\u5de6\u53f3\u5f00\u59cb\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u79cd\u65b9\u6cd5\u5728\u5b9e\u9a8c\u5ba4\u4e2d\u7ea0\u6b63\u4e86\u4e0e\u8425\u517b\u4e0d\u826f\u76f8\u5173\u7684\u5927\u90e8\u5206\u75c5\u7406\uff0c\u5e76\u963b\u6b62\u4e86\u75c5\u60c5\u7684\u8fdb\u4e00\u6b65\u53d1\u5c55\u3002\u5f20\u4f2f\u4f26\u548c\u5854\u65af\u6cd5\u7279\u5e0c\u671b\u8fd9\u79cd\u65b9\u6cd5\u6700\u7ec8\u80fd\u591f\u9006\u8f6c\u808c\u8089\u840e\u7f29\uff0c\u6062\u590d\u808c\u8089\u7ec4\u7ec7\u7684\u6b63\u5e38\u5065\u5eb7\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u636e\u5f20\u4f2f\u4f26\u4ecb\u7ecd\uff0c\u6700\u65b0\u7684\u65b9\u6cd5\u8fd8\u5229\u7528\u4e86\u4e00\u79cd\u65b0\u578b AAV \u8f7d\u4f53\u7c7b\u578b\uff0c\u5141\u8bb8\u4f7f\u7528\u8f83\u4f4e\u7684\u5242\u91cf\uff0c\u4ece\u800c\u51cf\u8f7b\u6216\u6d88\u9664\u4e86\u65e9\u671f\u65b9\u6cd5\u7684\u8bb8\u591a\u8d1f\u9762\u540e\u679c\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/new-gene-therapies-for-duchenne-muscular-dystrophy\/\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u6f5c\u5728\u65b0\u57fa\u56e0\u7597\u6cd5<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>For those who suffer from Duchenne muscular dystrophy (DMD), a novel gene therapy holds promise for both stopping muscle loss and maybe restoring the damaged muscle in the future. The next step for the treatment will be human trials, which are expected to begin in about two years, UW Medicine said. The goal of the [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4158,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[61,396],"class_list":["post-4151","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-gene-therapy","tag-uw-medicine"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4151","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=4151"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4151\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/4158"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4151"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4151"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4151"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}