{"id":3860,"date":"2025-05-15T13:01:14","date_gmt":"2025-05-15T10:01:14","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3860"},"modified":"2025-05-15T13:01:16","modified_gmt":"2025-05-15T10:01:16","slug":"precision-biosciences-pbgene-dmd-restores-production-of-nearly-full-length-dystrophin-protein","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/precision-biosciences-pbgene-dmd-restores-production-of-nearly-full-length-dystrophin-protein\/","title":{"rendered":"Precision BioSciences PBGENE-DMD \u6062\u590d\u8fd1\u5168\u957f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Precision BioSciences \u4eca\u5929\u5ba3\u5e03\u4e86 PBGENE-DMD \u7684\u6218\u7565\u4f18\u5148\u548c\u52a0\u901f\uff0cPBGENE-DMD \u662f\u8be5\u516c\u53f8\u9488\u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u9996\u521b\u4f53\u5185\u57fa\u56e0\u7f16\u8f91\u65b9\u6cd5\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\">\u4ec0\u4e48\u662f PBGENE-DMD\uff1f<\/a><\/li><li><a href=\"#how-the-pbgene-dmd-approach-works\">PBGENE-DMD \u65b9\u6cd5\u5982\u4f55\u5de5\u4f5c\uff1f<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-pbgene-dmd\">\u4ec0\u4e48\u662f PBGENE-DMD\uff1f<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>PBGENE-DMD \u662f\u4e00\u79cd\u4e00\u6d41\u7684\u4f53\u5185\u57fa\u56e0\u7f16\u8f91\u65b9\u6cd5\uff0c\u9002\u7528\u4e8e\u5927\u591a\u6570\u53d7\u5230\u5916\u663e\u5b50 45-55 \u4e4b\u95f4\u6700\u5e38\u89c1\u201c\u70ed\u70b9\u201d\u533a\u57df\u4e2d\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7a81\u53d8\u5f71\u54cd\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u3002<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u6700\u7ec8\u4e34\u5e8a\u5019\u9009\u836f\u7269 PBGENE-DMD \u5c55\u793a\u4e86\u4ee4\u4eba\u4fe1\u670d\u7684\u4e34\u5e8a\u524d\u6570\u636e\uff0c\u53ef\u4ee5\u968f\u7740\u65f6\u95f4\u7684\u63a8\u79fb\u6301\u4e45\u6539\u5584\u529f\u80fd\u6548\u76ca\u3002<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u7cbe\u51c6\u5b9a\u4f4d\uff0c\u4e8e 2025 \u5e74\u63d0\u4ea4 PBGENE-DMD \u7684\u65b0\u836f\u4e34\u5e8a\u8bd5\u9a8c\u7533\u8bf7 (IND) \u548c\/\u6216\u4e34\u5e8a\u8bd5\u9a8c\u7533\u8bf7 (CTA)\uff0c\u9884\u8ba1 2026 \u5e74\u83b7\u5f97\u4e34\u5e8a\u6570\u636e\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/precision-biosciences-presents-preclinical-efficacy-and-durability-data-on-pbgene-dmd-for-the-treatment-of-duchenne-muscular-dystrophy-dmd-at-the-2025-muscular-dystrophy-association-mda-clinical\/\" target=\"_blank\" rel=\"noreferrer noopener\">PBGENE-DMD\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-the-pbgene-dmd-approach-works\">PBGENE-DMD \u65b9\u6cd5\u5982\u4f55\u5de5\u4f5c\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u76ee\u524d\u5c1a\u65e0\u83b7\u6279\u7684\u7597\u6cd5\u80fd\u591f\u663e\u8457\u4e14\u6301\u4e45\u5730\u6539\u5584\u808c\u8089\u529f\u80fd\u3002PBGENE-DMD \u5229\u7528\u5355\u4e2a AAV \u9012\u9001\u4e24\u79cd\u4e92\u8865\u7684 ARCUS \u6838\u9178\u9176\uff0c\u5207\u9664\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7684 45-55 \u4e2a\u5916\u663e\u5b50\uff0c\u65e8\u5728\u6062\u590d\u4eba\u4f53\u81ea\u7136\u4ea7\u751f\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u80fd\u529b\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728ASGCT\u5927\u4f1a\u4e0a\u516c\u5e03\u7684\u4e34\u5e8a\u524d\u6570\u636e\u663e\u793a\uff0cPBGENE-DMD\u5728\u4eba\u6e90\u5316\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u5c0f\u9f20\u6a21\u578b\u4e2d\u8868\u73b0\u51fa\u663e\u8457\u4e14\u6301\u4e45\u7684\u529f\u80fd\u6539\u5584\u3002AAV\u9012\u9001\u540e\uff0cPBGENE-DMD\u6062\u590d\u4e86\u5c0f\u9f20\u5728\u591a\u79cd\u808c\u8089\uff08\u5305\u62ec\u5fc3\u808c\u548c\u9aa8\u9abc\u808c\uff09\u4e2d\u4ea7\u751f\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u80fd\u529b\u3002\u57289\u4e2a\u6708\u7684\u6cbb\u7597\u8fc7\u7a0b\u4e2d\uff0c\u63a5\u53d7PBGENE-DMD\u6cbb\u7597\u7684\u5c0f\u9f20\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u589e\u52a0\uff0c\u4ece\u800c\u83b7\u5f97\u4e86\u663e\u8457\u4e14\u6301\u7eed\u7684\u808c\u8089\u529f\u80fd\u6539\u5584\u3002\u6b64\u5916\uff0cPBGENE-DMD\u7f16\u8f91\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767dmRNA\u8f6c\u5f55\u672c\u5728\u808c\u8089\u536b\u661f\u5e72\u7ec6\u80de\uff08\u5373\u65b0\u751f\u808c\u8089\u7ec6\u80de\u7684\u7956\u7ec6\u80de\uff09\u4e2d\u8868\u8fbe\uff0c\u8fd9\u652f\u6301\u4e86\u5176\u957f\u671f\u6301\u4e45\u6027\u7684\u6f5c\u529b\u3002<\/p>","protected":false},"excerpt":{"rendered":"<p>Precision BioSciences, today announced the strategic prioritization and acceleration of PBGENE-DMD, the Company\u2019s first-in-class in vivo gene editing approach for Duchenne Muscular Dystrophy (DMD). What is PBGENE-DMD? Read more: PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy (DMD) How the PBGENE-DMD Approach Works? There are currently no approved therapies that can drive significant and durable [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3863,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,72,314,313],"class_list":["post-3860","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-full-length-dystrophin","tag-gene-editing","tag-pbgene-dmd","tag-precision-biosciences"],"subtitle":"PBGENE-DMD \u6709\u671b\u63d0\u4f9b\u4e00\u6b21\u6027\u3001\u6301\u4e45\u7684\u5e72\u9884\uff0c\u4f7f\u808c\u8089\u518d\u751f\u548c\u529f\u80fd\u53d7\u76ca\u7ec8\u8eab\u3002\u4e34\u5e8a\u524d\u6a21\u578b\u8868\u660e\uff0cPBGENE-DMD \u901a\u8fc7\u6062\u590d\u4eba\u4f53\u529f\u80fd\u6027\u3001\u63a5\u8fd1\u5168\u957f\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u751f\u6210\uff0c\u663e\u8457\u4e14\u6301\u7eed\u5730\u63d0\u9ad8\u6700\u5927\u529b\u91cf\u8f93\u51fa\u3002","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3860","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=3860"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3860\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3863"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3860"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3860"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3860"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}