{"id":3288,"date":"2025-03-19T16:03:20","date_gmt":"2025-03-19T13:03:20","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3288"},"modified":"2025-05-15T14:31:04","modified_gmt":"2025-05-15T11:31:04","slug":"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","status":"publish","type":"post","link":"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\/","title":{"rendered":"Precision BioSciences \u5728 2025 \u5e74\u808c\u8089\u840e\u7f29\u75c7\u534f\u4f1a (MDA) \u4e34\u5e8a\u4e0e\u79d1\u5b66\u4f1a\u8bae\u4e0a\u5c55\u793a\u4e86 PBGENE-DMD \u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u4e34\u5e8a\u524d\u7597\u6548\u548c\u8010\u4e45\u6027\u6570\u636e"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u8fd9\u79cd\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u6821\u6b63\u65b9\u6cd5\u6d89\u53ca\u7f16\u8f91\u808c\u8089\u536b\u661f\u5e72\u7ec6\u80de\uff0c\u4e0e\u5408\u6210\u65b9\u6cd5\u76f8\u6bd4\uff0c\u5b83\u6709\u53ef\u80fd\u63d0\u9ad8\u8010\u4e45\u6027\u548c\u529f\u80fd\u7ed3\u679c\u3002\u7531\u4e8e\u591a\u8fbe 60% \u7684 DMD \u75c5\u4f8b\u662f\u7531\u5916\u663e\u5b50 45 \u548c 55 \u4e4b\u95f4\u7684\u7f3a\u9677\u5f15\u8d77\u7684\uff0c\u56e0\u6b64\u8fd9\u79cd\u65b9\u6cd5\u6bd4\u5916\u663e\u5b50\u8df3\u8dc3\u8005\u66f4\u5e7f\u6cdb\u5730\u9002\u7528\u4e8e\u5927\u591a\u6570 DMD \u60a3\u8005\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u8be5\u7814\u7a76\u7684\u4e3b\u8981\u53d1\u73b0\u5305\u62ec\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\uff1a<\/strong> PBGENE-DMD \u6062\u590d\u4e86\u4eba\u4f53\u5728\u591a\u79cd\u808c\u8089\uff08\u5305\u62ec\u5fc3\u810f\u3001\u6a2a\u8188\u819c\u548c\u9aa8\u9abc\u808c\uff09\u4e2d\u4ea7\u751f\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u80fd\u529b\uff0c\u5176\u6c34\u5e73\u9884\u8ba1\u53ef\u4ee5\u63d0\u4f9b\u6cbb\u7597\u76ca\u5904\u3002<\/li>\n\n\n\n<li><strong>\u589e\u5f3a\u808c\u8089\u5f39\u6027\uff1a<\/strong> \u4e0e\u672a\u7ecf\u6cbb\u7597\u7684\u60a3\u75c5\u5c0f\u9f20\u76f8\u6bd4\uff0c\u63a5\u53d7\u6cbb\u7597\u7684\u5c0f\u9f20\u5bf9\u79bb\u5fc3\u635f\u4f24\u7684\u62b5\u6297\u529b\u63d0\u9ad8\u4e86 66%\uff0c\u8fd9\u662f\u808c\u8089\u5f39\u6027\u589e\u5f3a\u7684\u6307\u6807\u3002<\/li>\n\n\n\n<li><strong>\u957f\u671f\u529f\u80fd\u6539\u5584\uff1a<\/strong> \u5728\u7528 PBGENE-DMD \u6cbb\u7597\u7684\u5c0f\u9f20\u4e2d\uff0c\u6700\u5927\u529b\u8f93\u51fa (MFO)\uff08\u4e00\u79cd\u5173\u952e\u7684\u529f\u80fd\u6307\u6807\uff09\u5728\u5065\u5eb7\u5bf9\u7167\u5c0f\u9f20\u4e2d\u8fbe\u5230 MFO \u7684 93%\uff0c\u5e76\u4e14\u5728 3 \u81f3 6 \u4e2a\u6708\u671f\u95f4\u5728\u7528 PBGENE-DMD \u6cbb\u7597\u7684\u5c0f\u9f20\u4e2d\u89c2\u5bdf\u5230\u6539\u5584\u3002<\/li>\n\n\n\n<li><strong>\u6301\u4e45\u6210\u679c\uff1a<\/strong> \u5728 PAX7+ \u7ec6\u80de\uff08\u808c\u8089\u536b\u661f\u5e72\u7ec6\u80de\u7684\u6807\u5fd7\u7269\uff09\u4e2d\u68c0\u6d4b\u5230\u4e86 PBGENE-DMD \u7f16\u8f91\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d mRNA \u8f6c\u5f55\u672c\uff0c\u8868\u660e\u4e0e\u6807\u51c6\u57fa\u56e0\u6cbb\u7597\u65b9\u6cd5\u76f8\u6bd4\uff0c\u5177\u6709\u6301\u4e45\u6cbb\u7597\u6548\u679c\u7684\u6f5c\u529b\u3002<\/li>\n<\/ul>","protected":false},"excerpt":{"rendered":"<p>This dystrophin gene correction approach which involves editing muscle satellite stem cells potentially enhances durability and functional outcomes compared to synthetic approaches. Since up to 60% of DMD cases are caused by defects between exons 45 and 55, this approach is more broadly applicable for the majority of DMD patients than exon skippers. Key findings [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3289,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,72,297,314,313],"class_list":["post-3288","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-full-length-dystrophin","tag-gene-editing","tag-muscular-dystrophy-association","tag-pbgene-dmd","tag-precision-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3288","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=3288"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3288\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3289"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3288"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3288"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3288"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}