{"id":8567,"date":"2026-08-24T13:49:06","date_gmt":"2026-08-24T10:49:06","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8567"},"modified":"2026-08-24T16:14:23","modified_gmt":"2026-08-24T13:14:23","slug":"pbgene-dmd-first-patient-dosed-gene-editing-study","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/pbgene-dmd-first-patient-dosed-gene-editing-study\/","title":{"rendered":"PBGENE-DMD\u8fdb\u5165\u4e34\u5e8a\u8bd5\u9a8c\uff1a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u7f16\u8f91\u7814\u7a76\u4e2d\u9996\u4f8b\u60a3\u8005\u63a5\u53d7\u7ed9\u836f"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Precision BioSciences \u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u57fa\u56e0\u7f16\u8f91\u65b9\u9762\u53d6\u5f97\u4e86\u91cd\u8981\u91cc\u7a0b\u7891\u5f0f\u7684\u8fdb\u5c55\u3002. <strong>\u8be5\u516c\u53f8\u4e8e 2026 \u5e74 8 \u6708 24 \u65e5\u5ba3\u5e03\uff0c\u9996\u4f4d\u60a3\u8005\u5df2\u5728 1\/2 \u671f FUNCTION-DMD \u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u63a5\u53d7\u7ed9\u836f\uff0c\u8be5\u8bd5\u9a8c\u65e8\u5728\u8bc4\u4f30 PBGENE-DMD\uff0c\u4e00\u79cd\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u5728\u7814\u4f53\u5185\u57fa\u56e0\u7f16\u8f91\u7597\u6cd5\u3002.<\/strong> \u7b2c\u4e00\u5242\u75ab\u82d7\u662f\u5728\u963f\u80af\u8272\u513f\u7ae5\u533b\u9662\uff08\u4e00\u5bb6\u4e13\u95e8\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4e2d\u5fc3\uff09\u63a5\u79cd\u7684\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=\"#what-is-pbgene-dmd\">PBGENE-DMD\u662f\u4ec0\u4e48\uff1f<\/a><\/li><li><a href=\"#why-exons-45-55-matter-in-duchenne\">\u4e3a\u4ec0\u4e48\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d\u7684\u7b2c 45-55 \u5916\u663e\u5b50\u5982\u6b64\u91cd\u8981<\/a><\/li><li><a href=\"#function-dmd-phase-1-2-trial-begins\">FUNCTION-DMD \u4e00\/\u4e8c\u671f\u4e34\u5e8a\u8bd5\u9a8c\u5f00\u59cb<\/a><\/li><li><a href=\"#how-pbgene-dmd-differs-from-microdystrophin\">PBGENE-DMD \u4e0e\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6709\u4f55\u4e0d\u540c<\/a><\/li><li><a href=\"#what-should-dmd-families-watch-next\">DMD\u60a3\u8005\u5bb6\u5ead\u63a5\u4e0b\u6765\u5e94\u8be5\u770b\u4ec0\u4e48\uff1f<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 id=\"what-is-pbgene-dmd\" class=\"wp-block-heading\">PBGENE-DMD\u662f\u4ec0\u4e48\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">PBGENE-DMD \u7684\u8bbe\u8ba1\u76ee\u6807\u662f\u89e3\u51b3\u6839\u672c\u7684\u57fa\u56e0\u7f3a\u9677\uff0c\u800c\u975e\u4ec5\u4ec5\u9012\u9001\u7f29\u77ed\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002\u8be5\u7597\u6cd5\u4f7f\u7528\u4e24\u79cd\u4e92\u8865\u7684 ARCUS \u6838\u9178\u9176\uff0c\u901a\u8fc7\u5355\u4e2a AAV \u8f7d\u4f53\u9012\u9001\uff0c\u4ece DMD \u57fa\u56e0\u4e2d\u5207\u9664\u5916\u663e\u5b50 45-55\u3002\u5176\u76ee\u6807\u662f\u6062\u590d\u9605\u8bfb\u6846\uff0c\u5e76\u4f7f\u63a5\u8fd1\u5168\u957f\u7684\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u5f97\u4ee5\u4ea7\u751f\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u9879\u7b56\u7565\u5177\u6709\u91cd\u8981\u7684\u79d1\u5b66\u610f\u4e49\uff0c\u56e0\u4e3a\u57fa\u56e0\u7f16\u8f91\u6b64\u524d\u5df2\u88ab\u7814\u7a76\u7528\u4e8e\u53bb\u9664\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7684\u7834\u574f\u6027\u533a\u57df\u5e76\u6062\u590d\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u8868\u8fbe\u3002\u5b9e\u9a8c\u7814\u7a76\u5df2\u8bc1\u5b9e\uff0c\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u6a21\u578b\u4e2d\uff0c\u5220\u9664\u5916\u663e\u5b50 45-55 \u53ef\u4ee5\u6062\u590d\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u8868\u8fbe\u3002\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/precision-biosciences-pbgene-dmd-restores-production-of-nearly-full-length-dystrophin-protein\/\" target=\"_blank\" data-type=\"post\" data-id=\"3860\" rel=\"noreferrer noopener\">PBGENE-DMD<\/a><\/p>\n\n\n\n<h2 id=\"why-exons-45-55-matter-in-duchenne\" class=\"wp-block-heading\">\u4e3a\u4ec0\u4e48\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d\u7684\u7b2c 45-55 \u5916\u663e\u5b50\u5982\u6b64\u91cd\u8981<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5916\u663e\u5b50 45-55 \u533a\u57df\u662f DMD \u7684\u4e3b\u8981\u7a81\u53d8\u70ed\u70b9\u533a\u57df\u3002. <strong>Precision BioSciences \u4f30\u8ba1\uff0c\u8be5\u533a\u57df\u7684\u7a81\u53d8\u53ef\u80fd\u4f7f\u5927\u7ea6 60% \u7684 DMD \u7537\u5b69\u6709\u53ef\u80fd\u901a\u8fc7 PBGENE-DMD \u8fdb\u884c\u6cbb\u7597\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7136\u800c\uff0c\u57fa\u56e0\u68c0\u6d4b\u7ed3\u679c\u662f\u5426\u7b26\u5408\u5165\u7ec4\u6807\u51c6\u4e0d\u5e94\u4e0e\u4e34\u5e8a\u7597\u6548\u6df7\u6dc6\u3002\u5373\u4f7f\u60a3\u8005\u9776\u533a\u57df\u5185\u5b58\u5728\u7a81\u53d8\uff0c\u4ecd\u9700\u8fdb\u884c\u8fdb\u4e00\u6b65\u8bc4\u4f30\uff0c\u624d\u80fd\u786e\u5b9a\u5176\u662f\u5426\u9002\u5408\u53c2\u52a0\u4e34\u5e8a\u8bd5\u9a8c\u6216\u63a5\u53d7\u540e\u7eed\u6cbb\u7597\u3002.<\/p>\n\n\n\n<h2 id=\"function-dmd-phase-1-2-trial-begins\" class=\"wp-block-heading\">FUNCTION-DMD \u4e00\/\u4e8c\u671f\u4e34\u5e8a\u8bd5\u9a8c\u5f00\u59cb<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>FUNCTION-DMD \u7814\u7a76\u76ee\u524d\u6b63\u5728\u62db\u52df 2-7 \u5c81\u3001\u80fd\u591f\u884c\u8d70\u7684 DMD \u60a3\u8005\uff0c\u8fd9\u4e9b\u60a3\u8005\u7684\u57fa\u56e0\u7a81\u53d8\u4f4d\u4e8e\u5916\u663e\u5b50 45 \u548c 55 \u4e4b\u95f4\u3002.<\/strong> \u591a\u4e2a\u7f8e\u56fd\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e13\u79d1\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u5fc3\u53c2\u4e0e\u5176\u4e2d\uff0c\u9884\u8ba1\u5c06\u4e8e2026\u5e74\u5e95\u516c\u5e03\u521d\u6b65\u5b89\u5168\u6027\u6570\u636e\u3002\u7acb\u5373\u4e86\u89e3\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/duchenne-exon-deletion-tool\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u675c\u6c0f\u5916\u663e\u5b50\u5220\u9664\u5de5\u5177<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u9996\u8981\u4efb\u52a1\u662f\u5b89\u5168\u6027\u3002\u7531\u4e8ePBGENE-DMD\u4f1a\u6c38\u4e45\u6027\u5730\u6539\u53d8DNA\uff0c\u4e34\u5e8a\u8bd5\u9a8c\u4e0d\u4ec5\u8981\u786e\u5b9aDNA\u7f16\u8f91\u662f\u5426\u53d1\u751f\uff0c\u8fd8\u8981\u786e\u5b9a\u8be5\u7597\u6cd5\u662f\u5426\u5177\u6709\u53ef\u63a5\u53d7\u7684\u5b89\u5168\u6027\u4ee5\u53ca\u662f\u5426\u5177\u6709\u663e\u8457\u7684\u751f\u7269\u5b66\u548c\u529f\u80fd\u6027\u6548\u5e94\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u4e00\u533a\u522b\u81f3\u5173\u91cd\u8981\u3002\u4e34\u5e8a\u524d\u7814\u7a76\u7684\u6210\u529f\u5e76\u4e0d\u7b49\u540c\u4e8e\u4e34\u5e8a\u7597\u6548\u3002FDA DMD\u836f\u7269\u7814\u53d1\u6307\u5357\u5f3a\u8c03\u4e86\u5177\u6709\u4e34\u5e8a\u610f\u4e49\u7684\u7ed3\u5c40\u6307\u6807\u7684\u91cd\u8981\u6027\uff0c\u5e76\u9f13\u52b1\u5728\u53ef\u884c\u7684\u60c5\u51b5\u4e0b\u5bf9\u9aa8\u9abc\u3001\u547c\u5438\u548c\u5fc3\u810f\u8868\u73b0\u8fdb\u884c\u8bc4\u4f30\u3002\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/pbgene-dmd-phase-1-2a-clinical-trial-for-duchenne-function-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">PBGENE-DMD 1\/2a\u671f\u4e34\u5e8a\u8bd5\u9a8c<\/a><\/p>\n\n\n\n<h2 id=\"how-pbgene-dmd-differs-from-microdystrophin\" class=\"wp-block-heading\">PBGENE-DMD \u4e0e\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6709\u4f55\u4e0d\u540c<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5176\u4e2d\u4e00\u4e2a\u5173\u952e\u533a\u522b\u5728\u4e8e\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u6765\u6e90\u3002PBGENE-DMD\u65e8\u5728\u7f16\u8f91\u60a3\u8005\u81ea\u8eab\u7684DMD\u57fa\u56e0\uff0c\u4ece\u800c\u6709\u53ef\u80fd\u5b9e\u73b0\u5185\u6e90\u6027\u4ea7\u751f\u63a5\u8fd1\u5168\u957f\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002\u8fd9\u4e0e\u5f15\u5165\u77ed\u94fe\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u65b9\u6cd5\u4e0d\u540c\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Precision BioSciences\u5df2\u516c\u5e03\u4e86\u4e00\u4e9b\u4ee4\u4eba\u9f13\u821e\u7684\u4e34\u5e8a\u524d\u7814\u7a76\u7ed3\u679c\uff0c\u5305\u62ec\u52a8\u7269\u6a21\u578b\u4e2d\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u6062\u590d\u548c\u529f\u80fd\u6539\u5584\u3002\u4f46\u8fd9\u4e9b\u7ed3\u679c\u4ecd\u5904\u4e8e\u4e34\u5e8a\u524d\u9636\u6bb5\uff0c\u56e0\u6b64\u65e0\u6cd5\u786e\u5b9a\u5728\u4eba\u4f53\u4e2d\u662f\u5426\u4f1a\u4ea7\u751f\u7c7b\u4f3c\u7684\u76ca\u5904\u3002.<\/p>\n\n\n\n<h2 id=\"what-should-dmd-families-watch-next\" class=\"wp-block-heading\">DMD\u60a3\u8005\u5bb6\u5ead\u63a5\u4e0b\u6765\u5e94\u8be5\u770b\u4ec0\u4e48\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u6700\u91cd\u8981\u7684\u91cc\u7a0b\u7891\u5c06\u662f\u4e34\u5e8a\u5b89\u5168\u6027\u6570\u636e\u3001\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6062\u590d\u60c5\u51b5\u3001\u529f\u80fd\u6027\u7ed3\u679c\u548c\u7597\u6548\u6301\u4e45\u6027\u3002\u60a3\u8005\u5bb6\u5c5e\u5e94\u4ed4\u7ec6\u533a\u5206\u516c\u53f8\u516c\u5e03\u7684\u4e34\u5e8a\u524d\u7ed3\u679c\u548c\u7ecf\u540c\u884c\u8bc4\u5ba1\u7684\u4eba\u4f53\u4e34\u5e8a\u8bc1\u636e\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">DMD \u662f\u4e00\u79cd\u7d2f\u53ca\u9aa8\u9abc\u808c\u3001\u5fc3\u808c\u548c\u547c\u5438\u808c\u7684\u591a\u7cfb\u7edf\u75be\u75c5\uff0c\u56e0\u6b64\u8fdb\u884c\u5168\u9762\u8bc4\u4f30\u81f3\u5173\u91cd\u8981\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u56e0\u6b64\uff0c\u9996\u6b21\u60a3\u8005\u7ed9\u836f\u6807\u5fd7\u7740\u4e00\u4e2a\u5f00\u59cb\uff0c\u800c\u975e\u7ed3\u675f\u3002PBGENE-DMD\u73b0\u5df2\u4ece\u5b9e\u9a8c\u5ba4\u7814\u53d1\u8fdb\u5165\u4eba\u4f53\u4e34\u5e8a\u8bd5\u9a8c\u9636\u6bb5\u3002\u4e0b\u4e00\u4e2a\u95ee\u9898\u662f\uff0c\u8fd9\u79cd\u57fa\u56e0\u7f16\u8f91\u7b56\u7565\u80fd\u5426\u5b89\u5168\u5730\u5c06\u5176\u751f\u7269\u5b66\u524d\u666f\u8f6c\u5316\u4e3a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u6301\u4e45\u800c\u6709\u610f\u4e49\u7684\u83b7\u76ca\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u53d1\u73b0\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/clinical-trials\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u63a2\u7d22\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e34\u5e8a\u8bd5\u9a8c <\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>Precision BioSciences has reached an important milestone in Duchenne muscular dystrophy (DMD) gene editing. The company announced on August 24, 2026, that the first patient has been dosed in the Phase 1\/2 FUNCTION-DMD clinical trial evaluating PBGENE-DMD, an investigational in vivo gene-editing therapy for Duchenne. The first dose was administered at Arkansas Children\u2019s Hospital, a [&hellip;]<\/p>\n","protected":false},"author":5,"featured_media":8568,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[726,242,359,72,725,314,724,313],"class_list":["post-8567","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-arcus-gene-editing","tag-clinical-trials","tag-full-length-dystrophin","tag-gene-editing","tag-gene-editing-therapy","tag-pbgene-dmd","tag-pbgene-dmd-therapy","tag-precision-biosciences"],"subtitle":"PBGENE-DMD \u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7814\u7a76\u4e2d\u53d6\u5f97\u4e86\u91cd\u5927\u91cc\u7a0b\u7891\u5f0f\u7684\u8fdb\u5c55\u3002\u9996\u4f8b\u60a3\u8005\u5df2\u5728 1\/2 \u671f FUNCTION-DMD \u8bd5\u9a8c\u4e2d\u63a5\u53d7\u7ed9\u836f\u3002\u57fa\u56e0\u7f16\u8f91\u80fd\u5426\u4e3a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u6cbb\u7597\u5e26\u6765\u65b0\u7eaa\u5143\uff1f","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8567","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\/5"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/comments?post=8567"}],"version-history":[{"count":5,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8567\/revisions"}],"predecessor-version":[{"id":8574,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8567\/revisions\/8574"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/8568"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=8567"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=8567"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=8567"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}