{"id":112324,"date":"2019-05-22T00:00:50","date_gmt":"2019-05-22T07:42:24","guid":{"rendered":"http:\/\/www.05921.net\/index.php\/112324\/"},"modified":"2019-05-22T15:42:29","modified_gmt":"2019-05-22T07:42:29","slug":"67f051ae1c","status":"publish","type":"post","link":"https:\/\/www.05921.net\/index.php\/112324\/","title":{"rendered":"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f"},"content":{"rendered":"<div class=\"bpp-post-content\">\n<h1  style=\"margin-bottom: 0.8em;font-weight: inherit;font-size: 1.7em;white-space: normal;max-width: 100%;box-sizing: border-box;overflow-wrap: break-word;color: rgb(51, 51, 51);font-family: -apple-system-font, BlinkMacSystemFont, &quot;Helvetica Neue&quot;, &quot;PingFang SC&quot;, &quot;Hiragino Sans GB&quot;, &quot;Microsoft YaHei UI&quot;, &quot;Microsoft YaHei&quot;, Arial, sans-serif;letter-spacing: 0.544px;text-align: justify;background-color: rgb(255, 255, 255);line-height: 1.75em;\"><span style=\"font-size: 15px;\"><strong style=\"max-width: 100%;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;line-height: 32px;font-size: 20px;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><span style=\"max-width: 100%;font-family: SimSun;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;line-height: 32px;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><span style=\"max-width: 100%;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;line-height: 32px;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><span style=\"max-width: 100%;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;line-height: 32px;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><span style=\"max-width: 100%;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;line-height: 1.75em;font-family: \u5fae\u8f6f\u96c5\u9ed1, sans-serif;font-size: 15px;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><span style=\"max-width: 100%;color: rgb(0, 112, 192);font-family: \u6977\u4f53;box-sizing: border-box !important;overflow-wrap: break-word !important;\">\u258e\u836f\u660e<\/span><\/strong><strong style=\"max-width: 100%;line-height: 1.75em;font-family: \u5fae\u8f6f\u96c5\u9ed1, sans-serif;font-size: 15px;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><span style=\"max-width: 100%;color: rgb(0, 112, 192);font-family: \u6977\u4f53;box-sizing: border-box !important;overflow-wrap: break-word !important;\">\u5eb7\u5fb7\u7f16\u8bd1\u6574\u7406\uff08\u6765\u6e90\uff1a<em>Nature Reviews Drug Discovery<\/em>\uff09<\/span><\/strong><\/strong><\/span><\/strong><\/strong><\/span><\/strong><\/strong><\/strong><\/span><\/strong><\/strong><\/span><\/strong><\/strong><\/span><\/h1>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u8fd1\u4e24\u5e74\u6765\uff0c\u57fa\u56e0\u7597\u6cd5\u9886\u57df\u53d6\u5f97\u4e86\u91cd\u5927\u7a81\u7834\uff0c2017\u5e74\u5e95\uff0c\u660e\u661f\u57fa\u56e0\u7597\u6cd5\u516c\u53f8Spark Therapeutics\u5f00\u53d1\u7684<\/span><a href=\"https:\/\/mp.weixin.qq.com\/s?__biz=MzAwMDA5NTIxNQ==&amp;mid=2649968637&amp;idx=2&amp;sn=a2f1cac334ff011db18e656e1d685cd6&amp;scene=21#wechat_redirect\" target=\"_blank\" style=\"text-decoration: underline;font-size: 15px;color: rgb(0, 112, 192);\" ><span style=\"font-size: 15px;color: rgb(0, 112, 192);\">Luxturna\u83b7\u5f97FDA\u6279\u51c6\u4e0a\u5e02<\/span><\/a><span style=\"font-size: 15px;\">\uff0c\u6210\u4e3a\u9996\u6b3e\u5728\u7f8e\u56fd\u83b7\u6279\u7684\u201c\u4f53\u5185\u7ed9\u836f\u578b\u201d\u57fa\u56e0\u7597\u6cd5\u3002\u800c\u8bfa\u534e\u516c\u53f8\u5f00\u53d1\u7684<\/span><a href=\"https:\/\/mp.weixin.qq.com\/s?__biz=MzAwMDA5NTIxNQ==&amp;mid=2649983064&amp;idx=3&amp;sn=cbd967c924c70a30fc639416727bda6f&amp;scene=21#wechat_redirect\" target=\"_blank\" style=\"text-decoration: underline;font-size: 15px;color: rgb(0, 112, 192);\" ><span style=\"font-size: 15px;color: rgb(0, 112, 192);\">Zolgensma\u4e5f\u6709\u671b\u5728\u672c\u6708\u83b7\u5f97FDA\u6279\u51c6<\/span><\/a><span style=\"font-size: 15px;\">\uff0c\u6210\u4e3a\u7b2c\u4e8c\u6b3e\u201c\u4f53\u5185\u7ed9\u836f\u578b\u201d\u57fa\u56e0\u7597\u6cd5\u3002\u6839\u636e\u518d\u751f\u533b\u5b66\u8054\u76df\uff08ARM\uff09\u53d1\u5e03\u7684\u62a5\u544a\uff0c\u622a\u81f3\u52302019\u5e74\u7b2c\u4e00\u5b63\u5ea6\u672b\uff0c\u5168\u7403\u6709<strong>371<\/strong>\u9879\u57fa\u56e0\u7597\u6cd5\u5904\u4e8e\u4e34\u5e8a\u5f00\u53d1\u9636\u6bb5\uff0c\u4e0e2018\u5e74\u540c\u671f\u76f8\u6bd4\uff0c\u589e\u957f\u4e86<strong>17%<\/strong>\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u4f5c\u4e3a\u9012\u9001\u57fa\u56e0\u7597\u6cd5\u7684\u6709\u529b\u5de5\u5177\uff0c\u817a\u76f8\u5173\u75c5\u6bd2\uff08AAV\uff09\u7684\u5f00\u53d1\u548c\u5236\u9020\u4e5f\u6210\u4e3a\u4e1a\u754c\u7684\u5173\u6ce8\u7126\u70b9\u4e4b\u4e00\u3002\u8fd1\u65e5\u591a\u5bb6\u516c\u53f8\u901a\u8fc7\u5408\u4f5c\u548c\u5e76\u8d2d\uff0c\u6269\u5c55AAV\u7684\u8bbe\u8ba1\u548c\u5236\u9020\u80fd\u529b\u3002\u65e5\u524d\uff0c<span style=\"font-size: 15px;\"><em>Nature Reviews Drug Discovery<\/em>\u4e0a\u53d1\u8868<\/span><span style=\"font-size: 15px;\">\u4e00\u7bc7\u7efc\u8ff0\uff0c\u901a\u8baf\u4f5c\u8005\u662f<\/span>\u9a6c\u8428\u8bf8\u585e\u5927\u5b66\u533b\u5b66\u9662\uff08University of Massachusetts\uff09\u7684\u57fa\u56e0\u7597\u6cd5\u4e13\u5bb6\u9ad8\u5149\u576a\u6559\u6388\u3002\u8fd9\u7bc7\u7efc\u8ff0<\/span><span style=\"font-size: 15px;\">\u5bf9\u8fd9\u4e00\u57fa\u56e0\u7597\u6cd5\u9012\u9001\u5e73\u53f0\u8fdb\u884c\u4e86\u8be6\u7ec6\u4ecb\u7ecd\uff0c\u4eca\u5929\u7684\u5fae\u4fe1\u56e2\u961f\u5c06\u4e0e\u8bfb\u8005\u5206\u4eab\u8fd9\u7bc7\u6587\u7ae0\u7684\u7cbe\u5f69\u5185\u5bb9\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: center;\"><img class=\"rich_pages\"      style=\"\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2019\/05\/5-1558510944.jpg\" alt=\"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f\" title=\"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f\"  \/><\/p>\n<p style=\"line-height: 1.75em;\"><br  \/><\/p>\n<section style=\"max-width: 100%;box-sizing: border-box;font-family: -apple-system-font, BlinkMacSystemFont, &quot;Helvetica Neue&quot;, &quot;PingFang SC&quot;, &quot;Hiragino Sans GB&quot;, &quot;Microsoft YaHei UI&quot;, &quot;Microsoft YaHei&quot;, Arial, sans-serif;letter-spacing: 0.544px;text-align: justify;white-space: normal;color: rgb(62, 62, 62);font-size: 16px;background-color: rgb(255, 255, 255);overflow-wrap: break-word !important;\">\n<section class=\"\"  style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"margin-top: 10px;max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"padding-left: 5px;max-width: 100%;box-sizing: border-box;display: inline-block;width: 638.4px;border-width: 0px 0px 0px 10px;border-style: solid;border-left-color: rgb(0, 112, 192);border-right-color: rgb(0, 112, 192);overflow-wrap: break-word !important;\">\n<section class=\"\"  style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"max-width: 100%;box-sizing: border-box;color: rgb(0, 112, 192);overflow-wrap: break-word !important;\">\n<p style=\"max-width: 100%;box-sizing: border-box;min-height: 1em;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\"><span style=\"font-size: 15px;\">\u817a\u76f8\u5173\u75c5\u6bd2\u57fa\u672c\u6784\u9020\u548c\u4f5c\u4e3a\u57fa\u56e0\u7597\u6cd5\u8f7d\u4f53\u7684\u8fd0\u4f5c\u673a\u7406<\/span><\/strong><\/p>\n<\/section>\n<\/section>\n<\/section>\n<\/section>\n<\/section>\n<\/section>\n<section class=\"\"  style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"margin-bottom: 8px;max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"max-width: 100%;box-sizing: border-box;background-color: rgb(0, 112, 192);height: 1px;overflow-wrap: break-word !important;\"><\/section>\n<\/section>\n<\/section>\n<\/section>\n<p style=\"max-width: 100%;min-height: 1em;color: rgb(51, 51, 51);font-family: -apple-system-font, BlinkMacSystemFont, &quot;Helvetica Neue&quot;, &quot;PingFang SC&quot;, &quot;Hiragino Sans GB&quot;, &quot;Microsoft YaHei UI&quot;, &quot;Microsoft YaHei&quot;, Arial, sans-serif;font-size: 17px;letter-spacing: 0.544px;text-align: justify;white-space: normal;background-color: rgb(255, 255, 255);line-height: 1.75em;box-sizing: border-box !important;overflow-wrap: break-word !important;\"><br  \/><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u817a\u76f8\u5173\u75c5\u6bd2\u662f\u4e00\u79cd\u5355\u94feDNA\u75c5\u6bd2\uff0c\u76ee\u524d\u7684\u79d1\u5b66\u754c\u5171\u8bc6\u662f\u5b83\u4e0d\u4f1a\u5bfc\u81f4\u4efb\u4f55\u4eba\u7c7b\u75be\u75c5\u3002\u5b83\u7531\u86cb\u767d\u8863\u58f3\uff08capside\uff09\u548c\u957f\u5ea6\u4e3a4.7kb\u7684\u5355\u94feDNA\u57fa\u56e0\u7ec4\u6784\u6210\u3002\u86cb\u767d\u8863\u58f3\u7531\u4e09\u4e2a\u4e9a\u57fa\u7ec4\u6210\uff0c\u5206\u522b\u4e3aVP1\uff0cVP2\uff0c\u548cVP3\u3002AAV\u57fa\u56e0\u7ec4\u4e24\u7aef\u4e3a\u4e24\u4e2a\u201cT\u201d\u578b\u7684\u672b\u7aef\u53cd\u5411\u91cd\u590d\u5e8f\u5217\uff08inverted terminal repeat, ITR\uff09\u3002\u8fd9\u4e24\u4e2aITRs\u662f\u75c5\u6bd2DNA\u590d\u5236\u7684\u8d77\u70b9\u548c\u89e6\u53d1\u75c5\u6bd2\u5305\u88c5\u7684\u4fe1\u53f7\u3002AAV\u57fa\u56e0\u7ec4\u4e2d\u7684<em>rep<\/em>\u57fa\u56e0\u7f16\u78014\u4e2a\u4e0e\u75c5\u6bd2\u590d\u5236\u76f8\u5173\u7684\u86cb\u767d\uff0c\u5206\u522b\u4e3aRep78\u3001Rep68\u3001Rep52\u3001\u548cRep40\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u4f5c\u4e3a\u57fa\u56e0\u7597\u6cd5\u8f7d\u4f53\u7684\u91cd\u7ec4\u817a\u76f8\u5173\u75c5\u6bd2\uff08rAAV\uff09\u643a\u5e26\u7684\u86cb\u767d\u8863\u58f3\u4e0e\u91ce\u751f\u578bAAV\u51e0\u4e4e\u5b8c\u5168\u76f8\u540c\uff0c\u7136\u800c\u8863\u58f3\u5185\u7684\u57fa\u56e0\u7ec4\u4e2d\u7f16\u7801\u75c5\u6bd2\u86cb\u767d\u7684\u90e8\u5206\u5b8c\u5168\u88ab\u5220\u9664\uff0c\u53d6\u800c\u4ee3\u4e4b\u7684\u662f\u6cbb\u7597\u6027\u8f6c\u57fa\u56e0\uff08transgene\uff09\u3002AAV\u57fa\u56e0\u7ec4\u4e2d\u552f\u4e00\u88ab\u4fdd\u7559\u7684\u90e8\u5206\u662fITRs\uff0c\u5b83\u8d77\u5230\u6307\u5bfc\u57fa\u56e0\u7ec4\u7684\u590d\u5236\u548c\u75c5\u6bd2\u8f7d\u4f53\u7ec4\u88c5\u7684\u4f5c\u7528\u3002<strong>\u5c06\u7f16\u7801\u75c5\u6bd2\u86cb\u767d\u7684\u90e8\u5206\u5b8c\u5168\u5220\u9664\u7684\u4f18\u70b9\u662f\uff1a\u4e00\u65b9\u9762\u53ef\u4ee5\u6700\u5927\u5316\u91cd\u7ec4AAV\u643a\u5e26\u8f6c\u57fa\u56e0\u7684\u5bb9\u91cf\uff0c\u53e6\u4e00\u65b9\u9762\u51cf\u5c0f\u4f53\u5185\u9012\u9001\u8f6c\u57fa\u56e0\u65f6\u4ea7\u751f\u7684\u514d\u75ab\u539f\u6027\u548c\u7ec6\u80de\u6bd2\u6027\u3002<\/strong><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: center;\"><img class=\"rich_pages\"      style=\"\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2019\/05\/4-1558510945.jpg\" alt=\"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f\" title=\"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f\"  \/><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><\/span><\/p>\n<h1  style=\"white-space: normal;line-height: 1.75em;text-align: center;\"><strong><span style=\"font-size: 14px;color: rgb(0, 112, 192);\">\u25b2\u91cd\u7ec4AAV\u8f7d\u4f53\u4ecb\u5bfc\u8f6c\u57fa\u56e0\u8868\u8fbe\u7684\u8fc7\u7a0b\uff08\u56fe\u7247\u6765\u6e90\uff1a\u53c2\u8003\u8d44\u6599[1]\uff09<\/span><\/strong><\/h1>\n<p style=\"line-height: 1.75em;\"><br  \/><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u91cd\u7ec4AAV\u9897\u7c92\u901a\u8fc7\u4e0e\u5bbf\u4e3b\u7ec6\u80de\u8868\u9762\u8868\u8fbe\u7684\u7cd6\u5316\u53d7\u4f53\u76f8\u7ed3\u5408\uff0c\u901a\u8fc7\u7f51\u683c\u86cb\u767d\uff08clathrin\uff09\u4ecb\u5bfc\u7684\u5185\u541e\u4f5c\u7528\u8fdb\u5165\u7ec6\u80de\u3002\u5728\u5185\u541e\u5f62\u6210\u7684\u5185\u4f53\uff08endosome\uff09\u9178\u5316\u4e4b\u540e\uff0c\u75c5\u6bd2\u8863\u58f3\u7684VP1\/VP2\u90e8\u5206\u6784\u8c61\u53d1\u751f\u53d8\u5316\uff0c\u5bfc\u81f4\u75c5\u6bd2\u4ece\u5185\u4f53\u4e2d\u8131\u79bb\uff0c\u5e76\u4e14\u901a\u8fc7\u6838\u5b54\u8fdb\u5165\u7ec6\u80de\u6838\u3002\u8fdb\u5165\u7ec6\u80de\u6838\u540e\uff0c\u5355\u94feDNA\u4ece\u8863\u58f3\u4e2d\u91ca\u653e\u51fa\u6765\u3002\u8fd9\u65f6\u5355\u94feDNA\u8fd8\u4e0d\u80fd\u8fdb\u884c\u8f6c\u5f55\uff0c\u5b83\u4eec\u9700\u8981\u53d8\u6210\u53cc\u94feDNA\u3002\u5355\u94feDNA\u53ef\u4ee5\u5229\u7528\u5bbf\u4e3b\u7ec6\u80de\u7684DNA\u805a\u5408\u9176\u6765\u5408\u6210\u4e92\u8865\u94fe\uff0c\u6216\u8005\u4e24\u6761\u4ece\u4e0d\u540cAAV\u9897\u7c92\u4e2d\u91ca\u653e\u7684\u4e92\u8865\u94fe\u9000\u706b\uff08annealing\uff09\u5f62\u6210\u53cc\u94feDNA\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u6700\u65b0\u7684AAV\u57fa\u56e0\u7ec4\u8bbe\u8ba1\u80fd\u591f\u5c06\u8863\u58f3\u4e2d\u643a\u5e26\u7684\u5355\u94feDNA\u8bbe\u8ba1\u6210\u81ea\u6211\u4e92\u8865\uff08self-complementary\uff09\u7684\u5e8f\u5217\u3002<strong>\u8fd9\u79cd\u5e8f\u5217\u7684\u4f18\u70b9\u5728\u4e8e\u5b83\u4e0d\u9700\u8981\u5355\u94feDNA\u590d\u5236\u6210\u4e3a\u53cc\u94feDNA\u7684\u6b65\u9aa4\u5c31\u53ef\u4ee5\u8fdb\u884c\u8f6c\u5f55\uff0c\u4e0e\u4f20\u7edf\u7684\u5355\u94feAAV\u57fa\u56e0\u7ec4\u76f8\u6bd4\uff0c\u5b83\u7684\u57fa\u56e0\u8868\u8fbe\u66f4\u4e3a\u8fc5\u901f\uff0c\u800c\u4e14\u8868\u8fbe\u91cf\u66f4\u9ad8<\/strong>\u3002\u8bfa\u534e\u516c\u53f8\u5f00\u53d1\u7684\u6cbb\u7597\u810a\u9ad3\u6027\u808c\u8089\u840e\u7f29\u75c7\uff08SMA\uff09\u7684Zolgensma\u5c31\u4f7f\u7528\u4e86\u8fd9\u79cd\u57fa\u56e0\u7ec4\u8bbe\u8ba1\u3002\u7136\u800c\u8fd9\u79cd\u8bbe\u8ba1\u7684\u4e00\u4e2a\u7f3a\u70b9\u662fAAV\u643a\u5e26\u7684\u8f6c\u57fa\u56e0\u5bb9\u91cf\u51cf\u534a\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u53cc\u94fe\u5f62\u5f0f\u7684AAV\u57fa\u56e0\u7ec4\u7136\u540e\u5229\u7528ITRs\u8fdb\u884c\u5206\u5b50\u5185\u6216\u5206\u5b50\u95f4\u57fa\u56e0\u7ec4\u91cd\u7ec4\uff0c\u8fd9\u4e00\u8fc7\u7a0b\u8ba9AAV\u57fa\u56e0\u7ec4\u6210\u4e3a\u7a33\u5b9a\u7684\u6e38\u79bbDNA\uff08episomal DNA\uff09\uff0c\u5bfc\u81f4\u57fa\u56e0\u7ec4\u80fd\u591f\u5728\u4e0d\u518d\u8fdb\u884c\u6709\u4e1d\u5206\u88c2\u7684\u7ec6\u80de\u4e2d\u6301\u7eed\u8fdb\u884c\u57fa\u56e0\u8868\u8fbe\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<section style=\"white-space: normal;max-width: 100%;box-sizing: border-box;letter-spacing: 0.544px;color: rgb(62, 62, 62);font-size: 16px;background-color: rgb(255, 255, 255);overflow-wrap: break-word !important;\">\n<section class=\"\"  style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"margin-top: 10px;max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"padding-left: 5px;max-width: 100%;box-sizing: border-box;display: inline-block;width: 638.4px;border-width: 0px 0px 0px 10px;border-style: solid;border-left-color: rgb(0, 112, 192);border-right-color: rgb(0, 112, 192);overflow-wrap: break-word !important;\">\n<section class=\"\"  style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"max-width: 100%;box-sizing: border-box;color: rgb(0, 112, 192);overflow-wrap: break-word !important;\">\n<p style=\"max-width: 100%;box-sizing: border-box;min-height: 1em;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\"><span style=\"font-size: 15px;\"><span style=\"font-size: 15px;\">AAV\u8f7d\u4f53\u7684\u8f6c\u5316\u5e94\u7528<\/span><\/span><\/strong><\/p>\n<\/section>\n<\/section>\n<\/section>\n<\/section>\n<\/section>\n<\/section>\n<section class=\"\"  style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"margin-bottom: 8px;max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"max-width: 100%;box-sizing: border-box;background-color: rgb(0, 112, 192);height: 1px;overflow-wrap: break-word !important;\"><\/section>\n<\/section>\n<\/section>\n<\/section>\n<p style=\"line-height: 1.75em;\"><br  \/><span style=\"font-size: 15px;\"><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u622a\u81f32018\u5e7411\u670813\u65e5\uff0c\u5728ClinicalTrials.gov\u4e0a\u6709<strong>145<\/strong>\u9879\u5e72\u9884\u6027\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u4f7f\u7528\u91cd\u7ec4AAV\u8f7d\u4f53\u3002\u76ee\u524d\uff0c\u4e24\u6b3e\u91cd\u7ec4AAV\u8f7d\u4f53\u5df2\u7ecf\u83b7\u5f97\u6279\u51c6\u5728\u60a3\u8005\u8eab\u4e0a\u4f7f\u7528\uff0c\u5b83\u4eec\u662funiQure\u516c\u53f8\u5f00\u53d1\u7684Glybera\uff08AAV1\uff09\u548cSpark Therapeutics\u516c\u53f8\u5f00\u53d1\u7684Luxturna\uff08AAV2\uff09\u3002\u800c\u8bfa\u534e\u516c\u53f8\u5f00\u53d1\u7684Zolgensma\uff08AAV9\uff09\u53ef\u80fd\u5728\u672c\u6708\u52a0\u5165\u83b7\u6279\u7597\u6cd5\u7684\u884c\u5217\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u76ee\u524d\uff0c\u5927\u7ea6\u670910\u6765\u79cd\u4e0d\u540c\u8840\u6e05\u578b\u7684AAV\u5728\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u4f5c\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u8f7d\u4f53\uff0c\u57fa\u4e8eAAV2\u7684\u57fa\u56e0\u9012\u9001\u5e73\u53f0\u867d\u7136\u4ecd\u7136\u662f\u6700\u591a\u7684\uff0c\u4f46\u662f\u8bf8\u5982AAV8\uff0cAAV9\u548cAAVrh.10\u7b49\u65b0\u4e00\u4ee3\u8863\u58f3\u6b63\u5728\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u5f97\u5230\u66f4\u591a\u5e94\u7528\u3002\u4e0b\u9762\u6211\u4eec\u6765\u770b\u4e00\u770b\u8fd9\u4e9b\u4e34\u5e8a\u8bd5\u9a8c\u7684\u7279\u5f81\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><img class=\"rich_pages\"      style=\"text-align: center;white-space: normal;\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2019\/05\/10-1558510945.jpg\" alt=\"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f\" title=\"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f\"  \/><\/span><\/p>\n<h1  style=\"text-align: center;\"><strong><span style=\"font-size: 14px;color: rgb(0, 112, 192);\">\u25b2\u5e72\u9884\u6027AAV\u57fa\u56e0\u7597\u6cd5\u4e34\u5e8a\u8bd5\u9a8c\u7b80\u4ecb\uff08\u56fe\u7247\u6765\u6e90\uff1a\u53c2\u8003\u8d44\u6599[1]\uff09<\/span><\/strong><\/h1>\n<p style=\"line-height: 1.75em;\"><strong><span style=\"font-size: 15px;color: rgb(0, 112, 192);\"><br  \/><\/span><\/strong><\/p>\n<p style=\"line-height: 1.75em;\"><strong><span style=\"font-size: 15px;color: rgb(0, 112, 192);\">\u91cd\u7ec4AAV\u8f7d\u4f53\u9012\u9001\u57fa\u56e0\u7684\u9776\u5411\u7ec4\u7ec7<\/span><\/strong><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><strong><span style=\"font-size: 15px;\">\u5927\u90e8\u5206\u57fa\u56e0\u7597\u6cd5\u7814\u53d1\u9879\u76ee\u7684\u9776\u5411\u7ec4\u7ec7\u662f\u809d\u810f\u3001\u6a2a\u7eb9\u808c\u548c\u4e2d\u67a2\u795e\u7ecf\u7cfb\u7edf<\/span><\/strong><span style=\"font-size: 15px;\">\u3002\u51e0\u4e4e\u6240\u6709\u5929\u7136AAV\u8863\u58f3\u86cb\u767d\u80fd\u591f\u5728\u809d\u810f\u4e2d\u5f15\u53d1\u6709\u6548\u7684\u8f6c\u57fa\u56e0\u8868\u8fbe\uff0c\u56e0\u6b64\u9776\u5411\u809d\u810f\u7684\u91cd\u7ec4AAV\u4e3a\u6cbb\u7597\u4e00\u7cfb\u5217\u75be\u75c5\u63d0\u4f9b\u4e86\u4f18\u826f\u7684\u57fa\u56e0\u9012\u9001\u5e73\u53f0\u3002\u5b83\u4eec\u5305\u62ec<\/span><a href=\"https:\/\/mp.weixin.qq.com\/s?__biz=MzAwMDA5NTIxNQ==&amp;mid=2649982681&amp;idx=5&amp;sn=2e8ef1351b37f060bee281ad75cd802a&amp;scene=21#wechat_redirect\" target=\"_blank\" style=\"text-decoration: underline;font-size: 15px;color: rgb(0, 112, 192);\" ><span style=\"font-size: 15px;color: rgb(0, 112, 192);\">A\u578b<\/span><\/a><span style=\"font-size: 15px;color: rgb(0, 0, 0);\">\u548c<\/span><a href=\"https:\/\/mp.weixin.qq.com\/s?__biz=MzAwMDA5NTIxNQ==&amp;mid=2649981039&amp;idx=2&amp;sn=0f41477bbba64724cf2401e832489987&amp;scene=21#wechat_redirect\" target=\"_blank\" style=\"text-decoration: underline;font-size: 15px;color: rgb(0, 112, 192);\" ><span style=\"font-size: 15px;color: rgb(0, 112, 192);\">B\u578b<\/span><\/a><span style=\"font-size: 15px;\">\u8840\u53cb\u75c5\u3001\u5bb6\u65cf\u6027\u9ad8\u80c6\u56fa\u9187\u8840\u75c7\u7b49\u75be\u75c5\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">AAV8\u548cAAV9\u8863\u58f3\u86cb\u767d\u80fd\u591f\u9776\u5411\u8eab\u4f53\u4e2d\u7684\u591a\u79cd\u808c\u8089\u7c7b\u578b\uff0c\u8fd9\u8ba9AAV\u4ecb\u5bfc\u7684\u57fa\u56e0\u7597\u6cd5\u80fd\u591f\u7528\u4e8e\u6cbb\u7597\u591a\u79cd\u808c\u8089\u75be\u75c5\uff0c\u5176\u4e2d\u5305\u62ec<\/span><a href=\"https:\/\/mp.weixin.qq.com\/s?__biz=MzAwMDA5NTIxNQ==&amp;mid=2649977405&amp;idx=4&amp;sn=fae4839e991fb2ab5c112b79835a0bc8&amp;scene=21#wechat_redirect\" target=\"_blank\" style=\"text-decoration: underline;font-size: 15px;color: rgb(0, 112, 192);\" ><span style=\"font-size: 15px;color: rgb(0, 112, 192);\">\u675c\u5174\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09<\/span><\/a><span style=\"font-size: 15px;\">\u3002\u503c\u5f97\u4e00\u63d0\u7684\u662f\uff0c<strong>\u808c\u8089\u53ef\u4ee5\u4f5c\u4e3a\u751f\u6210\u6cbb\u7597\u6027\u5206\u5b50\u7684\u201c\u4f53\u5185\u5de5\u5382\u201d\uff0c\u56e0\u6b64\u9776\u5411\u808c\u8089\u7ec4\u7ec7\u7684\u57fa\u56e0\u7597\u6cd5\u53ef\u4ee5\u7528\u4e8e\u6cbb\u7597\u975e\u808c\u8089\u75be\u75c5<\/strong>\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u91cd\u7ec4AAV\u9012\u9001\u7684\u57fa\u56e0\u7597\u6cd5\u7684\u53e6\u4e00\u4e2a\u91cd\u8981\u65b9\u5411\u662fCNS\uff0c\u5305\u62ec\u773c\u775b\u548c\u5927\u8111\u3002<strong>\u773c\u775b\u662f\u4e00\u4e2a\u76f8\u5bf9\u9694\u79bb\u7684\u73af\u5883\uff0c\u76f4\u63a5\u8fdb\u884c\u773c\u5185\u6ce8\u5c04\u9012\u9001AAV\u57fa\u56e0\u7597\u6cd5\u80fd\u591f\u8fbe\u5230\u6cbb\u7597\u591a\u79cd\u9057\u4f20\u6027\u773c\u75c5\u7684\u6548\u679c<\/strong>\u3002Spark Therapeutics\u516c\u53f8\u5f00\u53d1\u7684\u83b7\u6279\u7597\u6cd5Luxturna\u5c31\u662f\u6cbb\u7597\u7531\u4e8e<em>RPE65<\/em>\u57fa\u56e0\u7a81\u53d8\u800c\u5bfc\u81f4\u5931\u660e\u7684\u60a3\u8005\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;color: rgb(0, 112, 192);\">\u76f8\u5173\u9605\u8bfb\uff1a<\/span><a href=\"https:\/\/mp.weixin.qq.com\/s?__biz=MzAwMDA5NTIxNQ==&amp;mid=2649980704&amp;idx=1&amp;sn=b573cb8315fead35440e96b64ab91c1d&amp;scene=21#wechat_redirect\" target=\"_blank\" style=\"text-decoration: underline;font-size: 15px;color: rgb(0, 112, 192);\" >Nature\u7efc\u8ff0\uff1a\u57fa\u56e0\u7597\u6cd5\u5728\u6cbb\u7597\u773c\u79d1\u75be\u75c5\u65b9\u9762\u53d6\u5f97\u4e86\u54ea\u4e9b\u8fdb\u5c55\uff1f<\/a><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u800c\u9759\u8109\u8f93\u5165\u7684AAV9\u548cAAVrh.10\u80fd\u591f\u7a7f\u8d8a\u8840\u8111\u5c4f\u969c\uff0c\u5728\u795e\u7ecf\u5143\u548c\u80f6\u8d28\u7ec6\u80de\u4e2d\u5f15\u53d1\u8f6c\u57fa\u56e0\u8868\u8fbe\u3002\u8fd9\u9879\u91cd\u5927\u53d1\u73b0\u5e26\u6765\u591a\u9879\u9488\u5bf9CNS\u75be\u75c5\u7684\u7814\u7a76\uff0c\u5176\u4e2d\u5305\u62ecSMA\uff0c\u808c\u840e\u7f29\u4fa7\u7d22\u786c\u5316\u75c7\uff08ALS\uff09\uff0cGM1\u795e\u7ecf\u8282\u82f7\u8102\u75c5\uff0c\u548c\u7c98\u591a\u7cd6\u8d2e\u79ef\u75c7III\u578b\u7b49\u7b49\u3002<\/span><br  \/><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: center;\"><img class=\"rich_pages\"      style=\"\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2019\/05\/7-1558510946.jpg\" alt=\"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f\" title=\"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f\"  \/><\/p>\n<p style=\"text-align: center;\"><img class=\"rich_pages\"      style=\"\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2019\/05\/2-1558510946.jpg\" alt=\"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f\" title=\"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f\"  \/><\/p>\n<h1  style=\"white-space: normal;line-height: 1.75em;text-align: center;\"><strong><span style=\"font-size: 14px;color: rgb(0, 112, 192);\">\u25b2\u90e8\u5206\u5e72\u9884\u6027AAV\u57fa\u56e0\u7597\u6cd5\u4fe1\u606f\uff08\u56fe\u7247\u6765\u6e90\uff1a\u53c2\u8003\u8d44\u6599[1]\uff09<\/span><\/strong><\/h1>\n<p style=\"line-height: 1.75em;\"><br  \/><\/p>\n<p style=\"line-height: 1.75em;\"><strong><span style=\"font-size: 15px;color: rgb(0, 112, 192);\">AAV\u57fa\u56e0\u7597\u6cd5\u7b56\u7565<\/span><\/strong><\/p>\n<p style=\"line-height: 1.75em;\"><br  \/><\/p>\n<p style=\"line-height: 1.75em;\"><strong><span style=\"font-size: 15px;\">\u57fa\u56e0\u66ff\u4ee3<\/span><\/strong><span style=\"font-size: 15px;\">\uff1a\u8fd9\u4e00\u7b56\u7565\u7684\u76ee\u6807\u662f\u9012\u9001\u8868\u8fbe\u6b63\u5e38\u86cb\u767d\u7684\u57fa\u56e0\u6765\u5f25\u8865\u529f\u80fd\u4e27\u5931\u6027\u7a81\u53d8\u7684\u5f71\u54cd\u3002<strong>\u5b83\u9002\u7528\u4e8e\u6cbb\u7597\u9690\u6027\u5355\u57fa\u56e0\u75be\u75c5\uff0c\u5df2\u7ecf\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u83b7\u5f97\u6781\u5927\u6210\u529f<\/strong>\u3002Glybera\u548cLuxturna\u5c31\u662f\u5f88\u597d\u7684\u4f8b\u5b50\u3002Glybera\u57fa\u4e8e\u91cd\u7ec4AAV1\u5e73\u53f0\u9012\u9001\u8868\u8fbe\u7f16\u7801\u8102\u86cb\u767d\u8102\u80aa\u9176\uff08LPL\uff09\u7684\u8f6c\u57fa\u56e0\uff0c\u5b83\u7528\u4e8e\u6cbb\u7597LPL\u7f3a\u5931\u60a3\u8005\u3002\u800cLuxturna\u5219\u4f7f\u7528AAV2\u5e73\u53f0\u6765\u9012\u9001\u6b63\u5e38<em>RPE65<\/em>\u57fa\u56e0\u3002\u8fd9\u4e24\u6b3e\u7597\u6cd5\u90fd\u4f7f\u7528\u5c40\u90e8\u6ce8\u5c04\u7684\u65bd\u836f\u65b9\u6cd5\uff0c\u5206\u522b\u6ce8\u5c04\u5230\u60a3\u8005\u7684\u808c\u8089\u548c\u773c\u775b\u4e2d\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u65b0\u4e00\u4ee3AAV\u8840\u6e05\u578b\u7684\u53d1\u73b0\u8ba9\u7814\u7a76\u4eba\u5458\u53ef\u4ee5\u901a\u8fc7\u9759\u8109\u6ce8\u5c04AAV\u8f7d\u4f53\u5c06\u8f6c\u57fa\u56e0\u9012\u9001\u5230\u5168\u8eab\u3002\u8fd9\u4e9b\u65b0\u7684AAV\u8f7d\u4f53\u4e3a\u6cbb\u7597A\u578b\u548cB\u578b\u8840\u53cb\u75c5\uff08\u9776\u5411\u809d\u810f\uff09\uff0cDMD\uff08\u9776\u5411\u5168\u8eab\u808c\u8089\uff09\u548cSMA\uff08\u5e7f\u53d1\u9776\u5411CNS\uff0c\u5305\u62ec\u810a\u9ad3\uff09\u5e26\u6765\u4e86\u6709\u6548\u7597\u6cd5\u3002\u672c\u6708\u6709\u671b\u83b7\u6279\u7684Zolgensma\u5c31\u662f\u4e00\u4e2a\u8303\u4f8b\uff0c\u8fd9\u662f\u4e00\u6b3e\u5229\u7528AAV9\u8f7d\u4f53\u9012\u9001\u7f16\u7801\u8fd0\u52a8\u795e\u7ecf\u5143\u751f\u5b58\u86cb\u767d\uff08SMN\uff09\u7684SMA\u8f6c\u57fa\u56e0\u7684\u57fa\u56e0\u7597\u6cd5\u3002<strong>\u5728\u5f88\u591a\u75be\u75c5\u4e2d\uff0c\u53ea\u9700\u5728\u90e8\u5206\u7ec6\u80de\u4e2d\u8868\u8fbe\u6b63\u5e38\u57fa\u56e0\u5c31\u8db3\u4ee5\u7f13\u89e3\u75be\u75c5\u7684\u75c7\u72b6\u3002<\/strong><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><strong><span style=\"font-size: 15px;\">\u57fa\u56e0\u6c89\u9ed8\uff1a<\/span><\/strong><span style=\"font-size: 15px;\">\u4e0e\u57fa\u56e0\u66ff\u4ee3\u76f8\u53cd\uff0c<strong>\u57fa\u56e0\u6c89\u9ed8\u7684\u76ee\u6807\u662f\u6cbb\u7597\u7531\u4e8e\u529f\u80fd\u83b7\u5f97\u6027\u57fa\u56e0\u7a81\u53d8\u5bfc\u81f4\u7684\u5355\u57fa\u56e0\u75be\u75c5<\/strong>\uff0c\u4f8b\u5982\u4ea8\u5ef7\u987f\u75c5\uff08Huntington disease\uff09\u3002RNAi\u7597\u6cd5\u76ee\u524d\u662fAAV\u57fa\u56e0\u6c89\u9ed8\u5e73\u53f0\u7684\u9996\u9009\u7b56\u7565\u3002\u7136\u800c\uff0c\u4e0e\u5408\u6210RNAi\u7597\u6cd5\u7684\u98de\u901f\u8fdb\u5c55\u76f8\u6bd4\uff0c\u57fa\u4e8eAAV\u9012\u9001\u5e73\u53f0\u7684RNAi\u7597\u6cd5\u76ee\u524d\u4ecd\u5904\u4e8e\u4e34\u5e8a\u524d\u5f00\u53d1\u9636\u6bb5\u3002\u57fa\u56e0\u6c89\u9ed8\u9700\u8981\u5728\u5927\u90e8\u5206\u7ec4\u7ec7\u4e2d\u53d1\u751f\u624d\u80fd\u591f\u8d77\u5230\u6709\u610f\u4e49\u7684\u6cbb\u7597\u6548\u679c\uff0c\u5728\u67d0\u4e9b\u5668\u5b98\u4e2d\uff08\u4f8b\u5982\u5927\u8111\uff09\uff0c\u8fd9\u5bf9\u4e8eAAV\u8f7d\u4f53\u6765\u8bf4\u8fd8\u662f\u4e00\u4e2a\u6311\u6218\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u5728RNAi\u7b56\u7565\u4ee5\u5916\uff0c\u4f7f\u7528CRISPR-Cas\u7cfb\u7edf\u6765\u9776\u5411RNA\u4e5f\u662f\u4e00\u79cd\u57fa\u56e0\u9759\u9ed8\u7684\u7b56\u7565\u3002\u4e0d\u8fc7\u8fd9\u4e00\u7b56\u7565\u9700\u8981\u514b\u670d\u7684\u969c\u788d\u662f\u91cd\u7ec4AAV\u5bb9\u7eb3\u7684\u8f6c\u57fa\u56e0\u5927\u5c0f\u6709\u9650\uff0c\u800c\u4e14\u4ece\u7ec6\u83cc\u4e2d\u53d1\u73b0\u7684Cas\u86cb\u767d\u53ef\u80fd\u5f15\u53d1\u514d\u75ab\u53cd\u5e94\uff0c\u4ece\u800c\u964d\u4f4e\u7597\u6548\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><strong><span style=\"font-size: 15px;\">\u9012\u9001\u65b0\u57fa\u56e0\uff1a<\/span><\/strong><span style=\"font-size: 15px;\">\u9664\u4e86\u6cbb\u7597\u5355\u57fa\u56e0\u75be\u75c5\u4ee5\u5916\uff0c<strong>AAV\u4ecb\u5bfc\u7684\u57fa\u56e0\u7597\u6cd5\u5177\u6709\u901a\u8fc7\u9012\u9001\u65b0\u57fa\u56e0\u6765\u6cbb\u7597\u590d\u6742\u9057\u4f20\u75c5\u6216\u8005\u4f20\u67d3\u75c5\u7684\u6f5c\u529b<\/strong>\u3002\u4f8b\u5982\uff0c\u5fc3\u529b\u8870\u7aed\u548c\u4f20\u67d3\u75c5\u4ee3\u8868\u7740\u5177\u6709\u9ad8\u5ea6\u672a\u7ecf\u9700\u6c42\u7684\u75be\u75c5\u9886\u57df\uff0c\u901a\u8fc7\u5f15\u5165\u65b0\u57fa\u56e0\uff0c\u57fa\u56e0\u7597\u6cd5\u53ef\u4ee5\u63d0\u4f9b\u751f\u957f\u56e0\u5b50\uff0c\u8c03\u8282\u4e0e\u5fc3\u529b\u8870\u7aed\u76f8\u5173\u7684\u4fe1\u53f7\u901a\u8def\uff0c\u4ee5\u53ca\u8868\u8fbe\u4e2d\u548c\u81f4\u547d\u75c5\u6bd2\u611f\u67d3\u7684\u6297\u4f53\u3002\u76ee\u524d\u7684\u4e34\u5e8a\u8bd5\u9a8c\u6b63\u5728\u68c0\u9a8c\uff0c\u4f7f\u7528\u808c\u8089\u4f5c\u4e3a\u751f\u6210\u6cbb\u7597\u6027\u6297\u4f53\u7684\u201c\u751f\u7269\u5de5\u5382\u201d\uff0c\u6cbb\u7597HIV\u611f\u67d3\u7684\u6cbb\u7597\u65b9\u6cd5\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<section style=\"white-space: normal;max-width: 100%;box-sizing: border-box;letter-spacing: 0.544px;color: rgb(62, 62, 62);font-size: 16px;background-color: rgb(255, 255, 255);overflow-wrap: break-word !important;\">\n<section class=\"\"  style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"margin-top: 10px;max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"padding-left: 5px;max-width: 100%;box-sizing: border-box;display: inline-block;width: 638.4px;border-width: 0px 0px 0px 10px;border-style: solid;border-left-color: rgb(0, 112, 192);border-right-color: rgb(0, 112, 192);overflow-wrap: break-word !important;\">\n<section class=\"\"  style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"max-width: 100%;box-sizing: border-box;color: rgb(0, 112, 192);overflow-wrap: break-word !important;\">\n<p style=\"max-width: 100%;box-sizing: border-box;min-height: 1em;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\"><span style=\"font-size: 15px;\"><span style=\"font-size: 15px;\">\u6539\u8fdbAAV\u8f7d\u4f53\u7684\u624b\u6bb5<\/span><\/span><\/strong><\/p>\n<\/section>\n<\/section>\n<\/section>\n<\/section>\n<\/section>\n<\/section>\n<section class=\"\"  style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"margin-bottom: 8px;max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"max-width: 100%;box-sizing: border-box;background-color: rgb(0, 112, 192);height: 1px;overflow-wrap: break-word !important;\"><\/section>\n<\/section>\n<\/section>\n<\/section>\n<p style=\"line-height: 1.75em;\"><br  \/><span style=\"font-size: 15px;\"><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u5f53\u91cd\u7ec4AAV\u572820\u591a\u5e74\u524d\u7b2c\u4e00\u6b21\u8868\u73b0\u51fa\u4f5c\u4e3a\u57fa\u56e0\u7597\u6cd5\u8f7d\u4f53\u7684\u4e34\u5e8a\u6d3b\u6027\u4e4b\u540e\uff0c\u7814\u7a76\u4eba\u5458\u5c31\u5728\u4e0d\u65ad\u5bf9\u8863\u58f3\u86cb\u767d\u8fdb\u884c\u6539\u9020\uff0c\u8bd5\u56fe\u8d4b\u4e88\u5b83\u65b0\u7684\u529f\u80fd\u548c\u7279\u5f81\u3002\u968f\u7740\u79d1\u6280\u7684\u8fdb\u6b65\uff0c\u5f00\u53d1\u65b0\u8863\u58f3\u86cb\u767d\u7684\u7b56\u7565\u4e5f\u4e0d\u65ad\u6f14\u53d8\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u76ee\u524d\uff0c<strong>\u8863\u58f3\u86cb\u767d\u5f00\u53d1\u7684\u7b56\u7565\u53ef\u4ee5\u5206\u4e3a3\u5927\u7c7b\uff1a\u5929\u7136\u53d1\u73b0\uff0c\u7406\u6027\u8bbe\u8ba1\uff0c\u548c\u5b9a\u5411\u8fdb\u5316<\/strong>\u3002\u968f\u7740\u8fd1\u5e74\u6765\u8ba1\u7b97\u80fd\u529b\u548c\u751f\u7269\u4fe1\u606f\u5b66\u9884\u6d4b\u6a21\u578b\u7684\u8fdb\u5c55\uff0c\u4e00\u4e2a\u65b0\u7684\u8863\u58f3\u86cb\u767d\u8bbe\u8ba1\u5206\u652f\u51fa\u73b0\u4e86\u3002\u8fd9\u5c31\u662f<strong>\u8ba1\u7b97\u673a\u53d1\u73b0\uff08in silico discovery\uff09\u7b56\u7565<\/strong>\u3002\u4e0b\u9762\u6211\u4eec\u6765\u770b\u4e00\u770b\u8fd9\u4e9b\u4e0d\u540c\u7b56\u7565\u7684\u4f18\u7f3a\u70b9\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: center;\"><img class=\"rich_pages\"      style=\"\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2019\/05\/9-1558510947.jpg\" alt=\"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f\" title=\"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f\"  \/><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><\/span><\/p>\n<h1  style=\"white-space: normal;line-height: 1.75em;text-align: center;\"><strong><span style=\"font-size: 14px;color: rgb(0, 112, 192);\">\u25b2\u6539\u8fdbAAV\u8f7d\u4f53\u8863\u58f3\u86cb\u767d\u7684\u56db\u79cd\u7b56\u7565\uff08\u56fe\u7247\u6765\u6e90\uff1a\u53c2\u8003\u8d44\u6599[1]\uff09<\/span><\/strong><\/h1>\n<p style=\"line-height: 1.75em;\"><br  \/><\/p>\n<p style=\"line-height: 1.75em;\"><strong><span style=\"font-size: 15px;color: rgb(0, 112, 192);\">\u5929\u7136\u53d1\u73b0<\/span><\/strong><\/p>\n<p style=\"line-height: 1.75em;\"><strong><span style=\"font-size: 15px;color: rgb(0, 112, 192);\"><br  \/><\/span><\/strong><\/p>\n<p style=\"line-height: 1.75em;\"><strong><span style=\"font-size: 15px;\">\u76ee\u524d\u6700\u5177\u6709\u4e34\u5e8a\u5e94\u7528\u6f5c\u529b\u7684AAV\u8840\u6e05\u578b\u90fd\u662f\u4ece\u5929\u7136\u6765\u6e90\u4e2d\u5206\u79bb\u51fa\u6765\u7684<\/span><\/strong><span style=\"font-size: 15px;\">\u3002\u5176\u4e2d\u7684\u8303\u4f8b\u5c31\u662fAAV9\uff0c\u5b83\u662f\u4ece\u4eba\u7c7b\u809d\u7ec4\u7ec7\u4e2d\u5206\u79bb\u51fa\u6765\u7684\u3002AAV9\u5177\u6709\u7a7f\u8d8a\u8840\u8111\u5c4f\u969c\u7684\u80fd\u529b\uff0c\u8fd9\u8ba9\u5b83\u6210\u4e3a\u5728\u4e2d\u67a2\u795e\u7ecf\u7cfb\u7edf\uff08CNS\uff09\u4e2d\u9012\u9001\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8863\u58f3\u3002\u9664\u4e86\u80fd\u591f\u611f\u67d3CNS\u7684AAV\u8840\u6e05\u578b\u4ee5\u5916\uff0c\u5929\u7136\u4eba\u7c7b\u548c\u975e\u4eba\u7075\u957f\u7c7b\uff08NHP\uff09AAV\u611f\u67d3\u7684\u4e3b\u8981\u5668\u5b98\u4e3a\u809d\u810f\u548c\u813e\u810f\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u7136\u800c\uff0c<strong>\u4f7f\u7528\u4eba\u7c7bAAV\u8863\u58f3\u7684\u4e00\u4e2a\u7f3a\u9677\u662f\u5927\u90e8\u5206\u4eba\u7531\u4e8e\u5728\u4e00\u751f\u4e2d\u53d7\u5230\u8fc7AAV\u7684\u611f\u67d3\uff0c\u5bfc\u81f4\u4ed6\u4eec\u4f53\u5185\u643a\u5e26\u9488\u5bf9AAV\u8863\u58f3\u7684\u6297\u4f53<\/strong>\u3002\u636e\u4f20\u67d3\u75c5\u5b66\u7edf\u8ba1\uff0c40-80%\u7684\u4eba\u4f53\u5185\u643a\u5e26\u9488\u5bf9AAV\u7684\u6297\u4f53\u3002\u8fd9\u53ef\u80fd\u5bfc\u81f4AAV\u4f5c\u4e3a\u57fa\u56e0\u7597\u6cd5\u8f7d\u4f53\u5728\u672a\u9012\u9001\u8f6c\u57fa\u56e0\u65f6\u5c31\u88ab\u514d\u75ab\u7cfb\u7edf\u6467\u6bc1\uff0c\u964d\u4f4e\u8f6c\u57fa\u56e0\u7684\u8868\u8fbe\u6c34\u5e73\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 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15px;\">\u3002\u5df2\u6709\u7814\u7a76\u8868\u660e\uff0c\u901a\u8fc7\u70b9\u7a81\u53d8\u6539\u53d8\u8863\u58f3\u8868\u9762\u916a\u6c28\u9178\u53ef\u4ee5\u5728\u5c0f\u9f20\u6a21\u578b\u4e2d\u5c06\u8f6c\u57fa\u56e0\u5728\u809d\u7ec6\u80de\u4e2d\u7684\u8868\u8fbe\u6c34\u5e73\u63d0\u9ad830\u500d\u3002\u8fd9\u4e00\u6539\u53d8\u4e0d\u4f46\u6291\u5236\u4e86\u86cb\u767d\u9176\u4f53\u5bf9AAV\u8863\u58f3\u7684\u964d\u89e3\uff0c\u800c\u4e14\u6539\u5584\u4e86\u75c5\u6bd2\u8f7d\u4f53\u5728\u7ec6\u80de\u5185\u7684\u8fd0\u8f93\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><strong><span style=\"font-size: 15px;color: rgb(0, 112, 192);\">\u5b9a\u5411\u8fdb\u5316<\/span><\/strong><\/p>\n<p style=\"line-height: 1.75em;\"><strong><span style=\"font-size: 15px;color: rgb(0, 112, 192);\"><br  \/><\/span><\/strong><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 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style=\"line-height: 1.75em;\"><strong><span style=\"font-size: 15px;color: rgb(0, 112, 192);\"><br  \/><\/span><\/strong><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u751f\u7269\u4fe1\u606f\u5b66\u548c\u8ba1\u7b97\u673a\u5de5\u5177\u53ef\u4ee5\u901a\u8fc7\u6bd4\u8f83\u4e0d\u540cAAV\u7684\u8863\u58f3\u86cb\u767d\u5e8f\u5217\uff0c\u63a8\u65ad\u51fa\u8863\u58f3\u86cb\u767d\u7684\u8fdb\u5316\u8fc7\u7a0b\uff0c\u5e76\u4e14\u53d1\u73b0\u8863\u58f3\u86cb\u767d\u4e0a\u5177\u5907\u9ad8\u5ea6\u591a\u6837\u6027\u7684\u533a\u57df\u3002\u8fd9\u53ef\u4ee5\u5e2e\u52a9\u5728\u8fd9\u4e9b\u533a\u57df\u5f15\u5165\u53d8\u5f02\uff0c\u4e0e\u5b9a\u5411\u8fdb\u5316\u624b\u6bb5\u76f8\u7ed3\u5408\uff0c\u66f4\u6709\u6548\u5730\u7b5b\u9009\u51fa\u65b0\u578b\u75c5\u6bd2\u8863\u58f3\u86cb\u767d\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<section style=\"white-space: normal;max-width: 100%;box-sizing: 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192);overflow-wrap: break-word !important;\">\n<p style=\"max-width: 100%;box-sizing: border-box;min-height: 1em;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\"><span style=\"font-size: 15px;\"><span style=\"font-size: 15px;\">\u7ed3\u8bed<\/span><\/span><\/strong><\/p>\n<\/section>\n<\/section>\n<\/section>\n<\/section>\n<\/section>\n<\/section>\n<section class=\"\"  style=\"max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"margin-bottom: 8px;max-width: 100%;box-sizing: border-box;overflow-wrap: break-word !important;\">\n<section class=\"\" style=\"max-width: 100%;box-sizing: border-box;background-color: rgb(0, 112, 192);height: 1px;overflow-wrap: break-word !important;\"><\/section>\n<\/section>\n<\/section>\n<\/section>\n<p style=\"line-height: 1.75em;\"><br  \/><span style=\"font-size: 15px;\"><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u57fa\u56e0\u7597\u6cd5\u662f\u83b7\u5f97\u4e1a\u754c\u5173\u6ce8\u7684\u521b\u65b0\u6cbb\u7597\u5e73\u53f0\u3002\u800c\u4f5c\u4e3a\u9012\u9001\u8f6c\u57fa\u56e0\u7684\u6709\u529b\u624b\u6bb5\uff0cAAV\u8f7d\u4f53\u5e73\u53f0\u7684\u7814\u53d1\u4e5f\u5728\u51fa\u73b0\u6307\u6570\u578b\u589e\u957f\u3002\u867d\u7136\u5bf9AAV\u7684\u7814\u7a76\u5c5e\u4e8e\u75c5\u6bd2\u5b66\u9886\u57df\uff0c\u4f46\u662f\u5c06AAV\u8f6c\u5316\u4e3a\u57fa\u56e0\u7597\u6cd5\u8f7d\u4f53\u7684\u8fc7\u7a0b\u9700\u8981\u591a\u5b66\u79d1\u7684\u8054\u5408\u7814\u7a76\u3002\u5206\u5b50\u751f\u7269\u5b66\u3001\u751f\u7269\u4fe1\u606f\u5b66\u3001\u4f20\u67d3\u75c5\u5b66\u3001\u7ed3\u6784\u751f\u7269\u5b66\u3001\u514d\u75ab\u5b66\u3001\u548c\u57fa\u56e0\u7ec4\u5b66\u7b49\u65b9\u9762\u7684\u7a81\u7834\u5bf9AAV\u8f7d\u4f53\u5e73\u53f0\u7684\u6210\u529f\u5f00\u53d1\u90fd\u8d77\u5230\u4e0d\u53ef\u6216\u7f3a\u7684\u4f5c\u7528\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">AAV\u8f7d\u4f53\u5e73\u53f0\u7684\u53d1\u5c55\u4ecd\u7136\u9700\u8981\u514b\u670d\u591a\u9879\u6311\u6218\uff0c\u5176\u4e2d\u5305\u62ec\u5982\u4f55\u7ecf\u6d4e\u6709\u6548\u5730\u751f\u6210\u8db3\u591f\u6570\u91cf\u7684\u6cbb\u7597\u6027AAV\u8f7d\u4f53\uff0c\u4ee5\u53ca\u514b\u670d\u4eba\u4f53\u514d\u75ab\u7cfb\u7edf\u5bf9AAV\u8f7d\u4f53\u548c\u8f6c\u57fa\u56e0\u4ea7\u7269\u7684\u514d\u75ab\u6392\u65a5\u4f5c\u7528\u3002\u514b\u670d\u8fd9\u4e9b\u6311\u6218\u5bf9\u63a8\u5e7fAAV\u4ecb\u5bfc\u7684\u57fa\u56e0\u7597\u6cd5\u5177\u6709\u5173\u952e\u6027\u7684\u610f\u4e49\u3002<\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p><span style=\"font-size: 15px;color: rgb(0, 112, 192);\">\u76f8\u5173\u9605\u8bfb\uff1a<\/span><a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzAwMDA5NTIxNQ==&amp;mid=2649980510&amp;idx=1&amp;sn=d81fff31821b21e138819ac35414d12b&amp;chksm=82e9f8deb59e71c8d50f35d479ee8a5be25ed790d564301864ac40461cee8bbc9325b879b8fc&amp;scene=21#wechat_redirect\" target=\"_blank\"  style=\"text-decoration: underline;font-size: 15px;color: rgb(0, 112, 192);\" ><span style=\"font-size: 15px;color: rgb(0, 112, 192);\">\u57fa\u56e0\u7597\u6cd5\u53ea\u80fd\u8ba9\u4e00\u5c0f\u90e8\u5206\u60a3\u8005\u83b7\u76ca\uff0c\u5982\u4f55\u7a81\u7834\u8fd9\u4e00\u5c40\u9650\uff1f<\/span><\/a><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"line-height: 1.75em;\"><span style=\"font-size: 15px;\">\u6211\u4eec\u671f\u5f85\u968f\u7740AAV\u7814\u53d1\u9886\u57df\u7684\u4e0d\u65ad\u6269\u5c55\u548c\u591a\u5b66\u79d1\u624b\u6bb5\u7684\u5e94\u7528\uff0c\u6211\u4eec\u80fd\u591f\u514b\u670d\u76ee\u524d\u9047\u5230\u7684\u6311\u6218\uff0c\u5b9e\u73b0AAV\u57fa\u56e0\u7597\u6cd5\u7684\u5168\u90e8\u6f5c\u529b\u3002<\/span><\/p>\n<p><br  \/><\/p>\n<p style=\"line-height: normal;\"><span style=\"font-size: 10px;color: rgb(178, 178, 178);\">\u53c2\u8003\u8d44\u6599\uff1a<\/span><\/p>\n<p style=\"line-height: normal;\"><span style=\"font-size: 10px;color: rgb(178, 178, 178);\">[1] Wang et al, (2019). Adeno-associated virus vector as a platform for gene therapy delivery. Nature Reviews Drug Discovery. https:\/\/doi.org\/10.1038\/s41573-019-0012-9<\/span><\/p>\n<p style=\"line-height: normal;\"><span style=\"font-size: 10px;color: rgb(178, 178, 178);\">[2] AveXis. Retrieved May 20, 2019, from https:\/\/www.avexis.com\/research-and-development<\/span><\/p>\n<p style=\"line-height: normal;\"><span style=\"font-size: 10px;color: rgb(178, 178, 178);\">[3] 372\u9879\u57fa\u56e0\u6cbb\u7597\u4e34\u5e8a\u8bd5\u9a8c\u6b63\u5728\u8fdb\u884c\u4e2d\uff0c25\u4e2a\u57fa\u56e0\u6cbb\u7597\u836f\u7269\u5373\u5c06\u5230\u6765. Retrieved May 21, 2019, from https:\/\/vcbeat.top\/NzNkMjczZmJjMjEwMjU5ODBhMDJkY2RmMTU0YWY1OGQ=<\/span><\/p>\n<p style=\"line-height: normal;\"><span style=\"font-size: 10px;color: rgb(178, 178, 178);\">[4] 25 Up-and-Coming Gene Therapies of 2019. Retrieved May 21, 2019, from https:\/\/www.genengnews.com\/a-lists\/25-up-and-coming-gene-therapies-of-2019\/<\/span><\/p>\n<p style=\"line-height: normal;\"><span style=\"font-size: 10px;color: rgb(178, 178, 178);\"><br  \/><\/span><\/p>\n<p style=\"white-space: normal;color: rgb(51, 51, 51);font-family: -apple-system-font, BlinkMacSystemFont, &quot;Helvetica Neue&quot;, &quot;PingFang SC&quot;, &quot;Hiragino Sans GB&quot;, &quot;Microsoft YaHei UI&quot;, &quot;Microsoft YaHei&quot;, Arial, sans-serif;font-size: 17px;letter-spacing: 0.544px;text-align: justify;background-color: rgb(255, 255, 255);line-height: normal;\"><span style=\"font-size: 10px;color: rgb(178, 178, 178);\">\u672c\u6587\u6765\u81ea\u5fae\u4fe1\u56e2\u961f\uff0c\u6b22\u8fce\u8f6c\u53d1\u5230\u670b\u53cb\u5708\uff0c\u8c22\u7edd\u8f6c\u8f7d\u5230\u5176\u4ed6\u5e73\u53f0\uff1b\u5982\u6709\u5f00\u8bbe\u767d\u540d\u5355\u9700\u6c42\uff0c\u8bf7\u5728\u6587\u7ae0\u5e95\u90e8\u7559\u8a00\uff1b\u5982\u6709\u5176\u4ed6\u5408\u4f5c\u9700\u6c42\uff0c\u8bf7\u8054\u7cfbwuxi_media@wuxiapptec.com<\/span><\/p>\n<p style=\"white-space: normal;text-align: center;\"><br  \/><\/p>\n<p style=\"white-space: normal;text-align: center;\"><img class=\"__bg_gif\"      style=\"color: rgb(51, 51, 51);font-family: -apple-system-font, BlinkMacSystemFont, &quot;Helvetica Neue&quot;, &quot;PingFang SC&quot;, &quot;Hiragino Sans GB&quot;, &quot;Microsoft YaHei UI&quot;, &quot;Microsoft YaHei&quot;, Arial, sans-serif;font-size: 17px;letter-spacing: 0.544px;white-space: normal;background-color: rgb(255, 255, 255);text-align: center;box-sizing: border-box !important;overflow-wrap: break-word !important;visibility: visible !important;width: 640px !important;\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2019\/05\/1-1558510948.gif\" alt=\"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f\" title=\"Nature\u7efc\u8ff0\uff1aAAV\u5982\u4f55\u6210\u4e3a\u57fa\u56e0\u7597\u6cd5\u7684\u9996\u9009\u8f7d\u4f53\uff1f\"  \/><\/p>\n<\/p><\/div>\n","protected":false},"excerpt":{"rendered":"<p>\u258e\u836f\u660e\u5eb7\u5fb7\u7f16\u8bd1\u6574\u7406\uff08\u6765\u6e90\uff1aNature Reviews Drug Discovery\uff09 \u8fd1\u4e24\u5e74\u6765\uff0c\u57fa\u56e0\u7597\u6cd5\u9886\u57df [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":112325,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":[],"categories":[1],"tags":[],"_links":{"self":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/112324"}],"collection":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/comments?post=112324"}],"version-history":[{"count":1,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/112324\/revisions"}],"predecessor-version":[{"id":112333,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/112324\/revisions\/112333"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/media\/112325"}],"wp:attachment":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/media?parent=112324"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/categories?post=112324"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/tags?post=112324"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}