{"id":2723,"date":"2025-02-01T09:45:45","date_gmt":"2025-02-01T06:45:45","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=2723"},"modified":"2025-02-01T14:34:43","modified_gmt":"2025-02-01T11:34:43","slug":"a-novel-mini-crispr-could-be-more-successful-at-editing-the-dystrophin-gene","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/a-novel-mini-crispr-could-be-more-successful-at-editing-the-dystrophin-gene\/","title":{"rendered":"\u4e00\u79cd\u65b0\u578b\u201cMini-CRISPR\u201d\u53ef\u4ee5\u66f4\u6210\u529f\u5730\u7f16\u8f91\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u4e00\u4e9b\u7814\u7a76\u5c0f\u7ec4\u4e00\u76f4\u5728\u5c1d\u8bd5\u521b\u5efa\u7d27\u51d1\u578b CRISPR \u7cfb\u7edf\uff0c\u8be5\u7cfb\u7edf\u53ef\u4ee5\u88c5\u5165\u5355\u4e2a AAV \u8f7d\u4f53\uff0c\u540c\u65f6\u4fdd\u6301\u7f16\u8f91\u529f\u80fd\u3002Mammoth Biosciences \u7684\u56e2\u961f\u5c31\u662f\u5176\u4e2d\u4e4b\u4e00\uff1b\u4ed6\u4eec\u53ef\u80fd\u5df2\u7ecf\u53d6\u5f97\u4e86\u6210\u529f\u3002\u8be5\u56e2\u961f\u7684\u7814\u7a76\u4eba\u5458\u9996\u5148\u5bf9\u5fae\u751f\u7269\u4e2d\u53d1\u73b0\u7684\u51e0\u79cd CRISPR \u7c7b\u578b\u7684\u57fa\u56e0\u5e8f\u5217\u4fe1\u606f\u8fdb\u884c\u5206\u7c7b\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u516c\u53f8\u7684\u9996\u5e2d\u79d1\u5b66\u5b98\u517c\u8054\u5408\u521b\u59cb\u4eba\u3001\u675c\u5fb7\u7eb3\u7684\u524d\u5b66\u751f\u5362\u5361\u65af\u00b7\u54c8\u7075\u987f (Lucas Harrington) \u58f0\u79f0\uff0c\u5728\u5bf9\u6700\u521d\u7684 176 \u540d\u7533\u8bf7\u8005\u8fdb\u884c\u201c\u4e00\u7cfb\u5217\u6d4b\u8bd5\u201d\u540e\uff0c\u4ed6\u4eec\u53d1\u73b0\u4e86\u201c\u5927\u6d77\u635e\u9488\u201d\u3002\u8fd9\u4e2a\u6700\u4f73\u9009\u62e9\u88ab\u547d\u540d\u4e3a NanoCas\uff1b\u5b83\u6709 425 \u4e2a\u6c28\u57fa\u9178\uff0c\u6bd4 Cas9 \u957f\u7ea6\u4e09\u5206\u4e4b\u4e00\u3002\u5b83\u7531\u54c8\u7075\u987f\u548c\u4ed6\u7684\u540c\u4e8b\u8bbe\u8ba1\uff0c\u7528\u4e8e\u9ad8\u6548\u5207\u5272\u54fa\u4e73\u52a8\u7269 DNA\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">NanoCas \u7814\u7a76\u7684\u4e3b\u8981\u7ed3\u8bba\u5305\u62ec\uff1a<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u7f16\u8f91\u6548\u7387\u4e0e\u7b2c\u4e00\u4ee3 CRISPR \u7cfb\u7edf\u76f8\u5f53\uff1a\u5728\u4f53\u5185\u9776\u5411\u5c0f\u9f20\u809d\u810f\u4e2d\u7684 PCSK9 \u57fa\u56e0\u65f6\uff0cNanoCas \u663e\u793a\u51fa\u7ea6 60% \u7684\u9971\u548c\u7f16\u8f91\u6548\u7387\uff0c\u4e0e\u5927\u5c0f\u7ea6\u4e3a\u5176\u4e09\u500d\u7684 SaCas9 \u76f8\u5f53\u3002\u4e24\u79cd CRISPR \u7cfb\u7edf\u5747\u5c06\u8840\u6e05 PCSK9 \u86cb\u767d\u964d\u4f4e\u81f3\u4e0d\u53ef\u68c0\u6d4b\u7684\u6c34\u5e73\u3002<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5728\u591a\u79cd\u808c\u8089\u7ec4\u7ec7\u4e2d\u8fdb\u884c\u5f3a\u5927\u7684\u5355\u4e2a AAV \u7f16\u8f91\uff1a\u5f53\u901a\u8fc7\u5355\u4e2a AAV \u8f7d\u4f53\u9012\u9001\u65f6\uff0cNanoCas \u53ef\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u4eba\u6e90\u5316\u5c0f\u9f20\u6a21\u578b\u4e2d\u7684\u80a1\u56db\u5934\u808c\u3001\u5c0f\u817f\u548c\u5fc3\u808c\u4e2d\u5bf9\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u8fdb\u884c 10% \u81f3 40% \u7684\u7f16\u8f91\u3002<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u9996\u6b21\u5728\u975e\u4eba\u7c7b\u7075\u957f\u7c7b\u52a8\u7269\u4e2d\u6f14\u793a\u5355\u4e2a AAV \u808c\u8089\u7f16\u8f91\uff1aNanoCas \u5728\u9776\u5411\u98df\u87f9\u7334\u9aa8\u9abc\u808c\u4e2d\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u65f6\u5b9e\u73b0\u4e86\u9ad8\u8fbe 30% \u7684\u4f53\u5185\u7f16\u8f91\u6548\u7387\u3002NanoCas \u8fd8\u663e\u793a\u5fc3\u810f\u4e2d 15% \u7684\u7f16\u8f91\uff0c\u800c SaCas9 \u4e3a 10%\u3002\u5bf9\u809d\u810f\u7ec4\u7ec7\u7684\u5206\u6790\u663e\u793a\u8131\u9776\u7f16\u8f91\u6781\u5c11\u3002<\/li>\n<\/ul>","protected":false},"excerpt":{"rendered":"<p>Several research groups have been attempting to create compact CRISPR systems that can fit into a single AAV vector while maintaining editing capabilities. The group at Mammoth Biosciences is one of these; they might have found success. Its researchers started by sorting through genetic sequence information from several CRISPR types that are found in microorganisms. [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":2724,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[66,226,72,227,225,228],"class_list":["post-2723","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-duchenne-muscular-dystrophy","tag-dystrophin-gene","tag-gene-editing","tag-mammoth-biosciences","tag-mini-crispr","tag-nanocas"],"subtitle":"Mammoth Biosciences \u7684\u4e00\u9879\u7814\u7a76\u8868\u660e\uff0cmini-CRISPR \u57fa\u56e0\u7f16\u8f91\u65b9\u6cd5\uff08\u4e5f\u79f0\u4e3a NanoCas\uff09\u5728\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u65b9\u9762\u66f4\u6709\u6548\u3002","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/2723","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=2723"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/2723\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/2724"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=2723"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=2723"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=2723"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}