{"id":9019,"date":"2020-10-09T03:19:28","date_gmt":"2020-10-08T19:19:28","guid":{"rendered":"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&#038;mid=100016621&#038;idx=1&#038;sn=6dd9ef753ae7cbda38ce60975f5f9ac2&#038;chksm=6b71735a5c06fa4ce7e6d649f57112fa1e0ee94075ee100084c8741a6cf2ca40e3e0b698682a#rd"},"modified":"2026-04-08T09:41:41","modified_gmt":"2026-04-08T01:41:41","slug":"544850","status":"publish","type":"post","link":"https:\/\/www.geneclub.net.cn\/index.php\/2020\/10\/09\/544850\/","title":{"rendered":"\u83b7\u8bfa\u5956\u7684\u57fa\u56e0\u7f16\u8f91\u6280\u672f\uff0c\u80cc\u540e\u5343\u4ebf\u7ea7\u57fa\u56e0\u6cbb\u7597\u4ea7\u4e1a\u903b\u8f91"},"content":{"rendered":"<p><!-- wechat_content_hash: 6021aac901e4a90c --><\/p>\n<div>2020\u5e7410\u6708\uff0c\u56e0\u53d1\u660e\u57fa\u56e0\u7f16\u8f91\u65b9\u6cd5CRISPR-Cas9\uff0c\u4e24\u4f4d\u79d1\u5b66\u5bb6\u83b7\u5f972020\u5e74\u8bfa\u8d1d\u5c14\u5316\u5b66\u5956\u3002\u57fa\u56e0\u7f16\u8f91\u6280\u672f\u80cc\u540e\u662f\u57fa\u56e0\u6cbb\u7597\u4ea7\u4e1a\u5343\u4ebf\u7ea7\u5e02\u573a\uff0c\u5df2\u5728\u6b27\u7f8e\u6380\u8d77\u70ed\u6f6e\uff0c\u4f8b\u59822018\u5e74\u5236\u836f\u5de8\u5934\u8bfa\u534e\u4ee587\u4ebf\u7f8e\u5143\u6536\u8d2dAveXis\u3002\u5176\u80cc\u540e\u903b\u8f91\u662f\u4ec0\u4e48\uff1f\u6211\u56fd\u4ea7\u4e1a\u5316\u6709\u54ea\u4e9b\u5dee\u8ddd\u548c\u53d1\u5c55\u7a7a\u95f4\uff1f\u8be6\u60c5\u89c1\u4e0b\u6587\u53ca\u57fa\u56e0\u6167\u5373\u5c06\u516c\u5f00\u53d1\u5e03\u7684\u300a\u7ec6\u80de\u548c\u57fa\u56e0\u6cbb\u7597\u884c\u7814\u62a5\u544a\u300b\uff08\u8054\u5408\u53d1\u5e03\u5408\u4f5c\u4ec5\u4f59\u4e24\u5e2d\uff09\u3002<\/div>\n<div>\u6587\u7ae0| \u57fa\u56e0\u6167\u00a0 \u7f16\u8f91\u00a0| \u5e03\u4e09\u5c11\u00a0\u00a0<\/div>\n<div>\u5173\u952e\u8bcd | \u57fa\u56e0\u7f16\u8f91\u00a0 \u57fa\u56e0\u6cbb\u7597<\/div>\n\n<div class=\"wp-block-image aligncenter\">\n<figure class=\"\"><img decoding=\"async\" src=\"https:\/\/www.geneclub.net.cn\/wp-content\/uploads\/images\/RmFicvQBAkgDnZkwogrrnwds9QlNNE7pJVfLn0XY4IQeqfdWR4PyEABzCekxhHxRyBuRvbdeib41sF1dmcjfjyNg.gif\" alt=\"RmFicvQBAkgDnZkwogrrnwds9QlNNE7pJVfLn0XY4IQeqfdWR4PyEABzCekxhHxRyBuRvbdeib41sF1dmcjfjyNg\"\/><\/figure><\/div>\n\n<div style=\"text-align: center;\">\u56fe1\uff1a\u57fa\u56e0\u7f16\u8f91\u548c\u57fa\u56e0\u6cbb\u7597\u793a\u610f\u56fe\uff08\u6765\u6e90\/giphy)<\/div>\n<div>1. \u57fa\u56e0\u7f16\u8f91\u662f\u6280\u672f\uff0c\u57fa\u56e0\u6cbb\u7597\u662f\u5e94\u7528<\/div>\n<div>\u57fa\u56e0\u7f16\u8f91\u662f\u6280\u672f\uff0c\u901a\u4fd7\u7406\u89e3\uff0c\u628a\u201d\u9519\u7684\u201c\uff08\u7a81\u53d8\u7684\uff09\u57fa\u56e0\u4fee\u590d\uff0c\u4fee\u590d\u7684\u5de5\u5177\uff08\u7c7b\u6bd4\u526a\u5200\uff09\u5305\u62ecZFN\u3001TALEN\u7b49\u6838\u9178\u9176\u548c\u83b7\u5f97\u8bfa\u5956\u7684CRISPR-Cas9\u7cfb\u7edf\u3002<\/div>\n<div>\u00a0<\/div>\n<div>\u57fa\u56e0\u6cbb\u7597\u662f\u5e94\u7528\uff0c\u7b80\u5355\u5f52\u7eb3\uff0c\u901a\u8fc7\u4fee\u590d\u57fa\u56e0\u6765\u8fbe\u5230\u6cbb\u7597\u75be\u75c5\u7684\u76ee\u7684\u3002\u4e00\u822c\u5305\u62ec\u4e09\u79cd\u5f62\u5f0f\uff1a<\/div>\n<div>\u57fa\u56e0\u6c89\u9ed8\uff1a\u5be1\u6838\u82f7\u9178\u7597\u6cd5\uff08ASO\/RNAi\u7b49\uff09<\/div>\n<div>\u57fa\u56e0\u5bfc\u5165\u66ff\u6362\uff1a\u8f7d\u4f53\uff08\u8d28\u7c92DNA\/\u75c5\u6bd2\/\u7ec6\u83cc\uff09<\/div>\n<div>\u57fa\u56e0\u4fee\u590d\uff1a\u57fa\u56e0\u7f16\u8f91<\/div>\n<div>\u00a0<\/div>\n<div>\u5176\u4e2d\uff0c\u57fa\u4e8e\u8f7d\u4f53\u7684\u57fa\u56e0\u5bfc\u5165\u66ff\u6362\u65b9\u6cd5\u57282003\u5e74\u524d\u540e\u4ea7\u751f\u8fc7\u4e00\u8f6e\u6d6a\u6f6e\uff0c\u66fe\u56e0\u4e2a\u522b\u5931\u8d25\u6848\u4f8b\u53d7\u632b\u3002\u00a0\u57fa\u4e8eRNAi\u7b49\u5be1\u6838\u82f7\u9178\u7597\u6cd5\u8fd1\u5e74\u9010\u6b65\u53d7\u5230\u91cd\u89c6\uff0c\u622a\u6b622020\u5e741\u6708\uff0c\u5df2\u670910\u6b3e\u5be1\u6838\u82f7\u9178\u836f\u7269\u83b7FDA\u6279\u51c6\u4e0a\u5e02\uff1bGSK\u7684\u9488\u5bf9\u6162\u6027\u4e59\u809d\u7684\u5be1\u6838\u82f7\u9178\u7597\u6cd5\u4e8e2020\u5e74\u5728\u4e2d\u56fd\u9996\u6b21\u83b7\u6279\u4e34\u5e8a\u8bd5\u9a8c\u3002\u57fa\u56e0\u7f16\u8f91\u6280\u672f\u81ea2013\u83b7\u5f97\u8f83\u5927\u5173\u6ce8\u3002\u5f53\u5e74\uff0c\u5f20\u950b\u5b9e\u73b0\u4e86CRISPR\u5728\u54fa\u4e73\u52a8\u7269\u7ec6\u80de\u7684\u57fa\u56e0\u7f16\u8f91\u5e76\u4e8e\u6b21\u5e74\u83b7\u5f97\u7f8e\u56fd\u4e13\u5229\u4e0e\u5546\u6807\u5c40\u5173\u4e8eCRISPR\u7684\u9996\u4e2a\u4e13\u5229\u6388\u6743\u3002<\/div>\n<div>\u00a0<\/div>\n<div>\u7531\u4e8e\u5728\u514d\u75ab\u7ec6\u80de\u6cbb\u7597\u4e2d\u5f80\u5f80\u9700\u8981\u7528\u5230\u7ecf\u8fc7\u4fee\u590d\u7684\u57fa\u56e0\uff0c\u56e0\u6b64\u57fa\u56e0\u6cbb\u7597\u548c\u7ec6\u80de\u6cbb\u7597\u7684\u6982\u5ff5\u5e38\u6709\u91cd\u53e0\u3002\u5728FDA\u548cNMPA\u5b98\u65b9\u8868\u8ff0\u4e2d\u6574\u5408\u4e3a\u7ec6\u80de\u548c\u57fa\u56e0\u6cbb\u7597\uff08Cellular &amp; Gene Therapy\uff0c\u7b80\u79f0CGT\uff09\u3002<\/div>\n<div>\u00a0<\/div>\n<div>2. \u5343\u4ebf\u7ea7\u57fa\u56e0\u6cbb\u7597\u4ea7\u4e1a\uff0c\u65b0\u836f\u521b\u5236\u7684\u903b\u8f91<\/div>\n<div>\u636e\u5f6d\u535a\u793e\uff08Bloomberg\uff09\u6570\u636e\uff0c\u7ec6\u80de\u548c\u57fa\u56e0\u6cbb\u7597\uff08CGT\uff09\u5e02\u573a\u89c4\u6a21\u8fbe\u5230119.6\u4ebf\u7f8e\u5143\u3002\u5343\u4ebf\u7ea7\u7684\u7ec6\u80de\u548c\u57fa\u56e0\u6cbb\u7597\u4ea7\u4e1a\u7684\u80cc\u540e\uff0c\u662f\u65b0\u836f\u521b\u5236\u7684\u57fa\u7840\u903b\u8f91\u3002<\/div>\n\n<div class=\"wp-block-image aligncenter\">\n<figure class=\"\"><img decoding=\"async\" src=\"https:\/\/www.geneclub.net.cn\/wp-content\/uploads\/images\/RmFicvQBAkgDnZkwogrrnwds9QlNNE7pJakMGiacDghIQoeG5djI331SMicr2sF9dlJBWoicNzvpI2zooon2jZ2jGg.png\" alt=\"RmFicvQBAkgDnZkwogrrnwds9QlNNE7pJakMGiacDghIQoeG5djI331SMicr2sF9dlJBWoicNzvpI2zooon2jZ2jGg\"\/><\/figure><\/div>\n\n<div style=\"text-align: center;\">\u56fe2\uff1a\u7ec6\u80de\u548c\u57fa\u56e0\u6cbb\u7597\u7684\u884c\u4e1a\u80cc\u666f\uff08\u6765\u6e90\/\u57fa\u56e0\u6167)<\/div>\n<div>\u653f\u7b56\u5c42\u9762\uff08\u56fe2\uff09\uff0c\u65e9\u57281998\u5e74FDA\u5c31\u53d1\u5e03\u4e86\u4f53\u7ec6\u80deCGT\u6307\u5357\uff1bNMPA\u57282019\u5e74\u53d1\u5e03\u7684\u836f\u54c1\u76d1\u7ba1\u79d1\u5b66\u884c\u52a8\u8ba1\u5212\uff0cCGT\u4ea7\u54c1\u6210\u4e3a\u9996\u6279\u7acb\u9879\u65b9\u5411\u3002<\/div>\n<div>\u00a0<\/div>\n<div>\u5728\u5e02\u573a\u9700\u6c42\u5c42\u9762\uff0c\u8001\u9f84\u5316\u5e26\u6765\u4e2a\u4f53\u5bf9\u4e8e\u764c\u75c7\u7b49\u75be\u75c5\u7cbe\u51c6\u533b\u7597\u7684\u5e7f\u6cdb\u9700\u6c42\uff1b\u6570\u767e\u4e07\u7f8e\u5143\uff08\u4f8b\u5982Zolgensma\uff09\u7684\u836f\u7269\u4ef7\u683c\u5e26\u6765\u5bb6\u5ead\u53ca\u793e\u4f1a\u5bf9\u4e8e\u65b0\u836f\u63a7\u8d39\u9700\u6c42\uff1b\u800c\u4ea7\u4e1a\u65b9\u9762\uff0c\u751f\u7269\u836f\u6210\u4e3a\u533b\u7597\u5927\u5065\u5eb7\u4ea7\u4e1a\u7687\u51a0\u4e0a\u7684\u660e\u73e0\u3002<\/div>\n<div>\u00a0<\/div>\n<div>\u5728\u4ea7\u4e1a\u548c\u6280\u672f\u5c42\u9762\uff0c\u6839\u636e2020\u5e744\u6708\u9ea6\u80af\u9521\u7684\u6700\u65b0\u6570\u636e\uff0cCGT\u4ea7\u54c1\u5360\u5236\u836f\u7814\u53d1\u4e34\u5e8a\u7ba1\u9053\u7ea612\uff05\uff0c\u4e34\u5e8a\u524d\u7ba1\u9053\u768416\uff05\u3002CRISPR\u53d1\u5c55\u5230\u5230\u5355\u78b1\u57fa\u7f16\u8f91\u3001\u53cc\u78b1\u57fa\u7f16\u8f91\u7b49\u3002<\/div>\n<div>\u00a0<\/div>\n<div>\u56e0\u6b64\uff0c\u6211\u4eec\u770b\u5230\uff0c2018\u5e74\uff0c\u5236\u836f\u5de8\u5934\u8bfa\u534e\u4ee587\u4ebf\u7f8e\u5143\u5b8c\u6210\u6536\u8d2dAveXis\uff1b2019\u5e74\uff0c\u7f57\u6c0f\u65a5\u8d4448\u4ebf\u7f8e\u5143\u6536\u8d2dSpark Therapeutic\uff1b\u5f3a\u751f\u516c\u53f8\u4e0eMeiraGTx\u5c31\u89c6\u529b\u4e27\u5931\u7684\u57fa\u56e0\u6cbb\u7597\u8fbe\u62101\u4ebf\u7f8e\u5143\u534f\u8bae\u2026\u2026<\/div>\n<div>\u00a0<\/div>\n<div>3.\u00a0\u6211\u56fd\u5728\u57fa\u56e0\u6cbb\u7597\u4ea7\u4e1a\u5316\u4e0a\u8fd8\u6709\u5f88\u5927\u7a7a\u95f4<\/div>\n<div>\u5728\u57fa\u56e0\u6cbb\u7597\u4ea7\u4e1a\u5316\u65b9\u9762\uff0c\u6211\u4eec\u8d77\u6b65\u5e76\u4e0d\u665a\uff0c\u4f46\u6b65\u5b50\u8fc8\u5f97\u5927\uff0c\u540e\u7eed\u4e4f\u529b\uff1b2018\u5e74\u7684\u57fa\u56e0\u7f16\u8f91\u80da\u80ce\u4e8b\u4ef6\u4f7f\u5f97\u884c\u4e1a\u201c\u5664\u82e5\u5bd2\u8749\u201d\uff0c\u800c\u5728\u4f53\u7ec6\u80de\u57fa\u56e0\u7f16\u8f91\u4ea7\u4e1a\u5316\u4e0a\u6709\u7740\u5e7f\u9614\u7684\u524d\u666f\u548c\u8f83\u5927\u5dee\u8ddd\u3002<\/div>\n\n<div class=\"wp-block-image aligncenter\">\n<figure class=\"\"><img decoding=\"async\" src=\"https:\/\/www.geneclub.net.cn\/wp-content\/uploads\/images\/RmFicvQBAkgDnZkwogrrnwds9QlNNE7pJuVnlmpXOZ5aw7buCckYeHT3G1dPN62KIrTbwhuYmyA5ZcunmUrXzdg.png\" alt=\"RmFicvQBAkgDnZkwogrrnwds9QlNNE7pJuVnlmpXOZ5aw7buCckYeHT3G1dPN62KIrTbwhuYmyA5ZcunmUrXzdg\"\/><\/figure><\/div>\n\n<div style=\"text-align: center;\">\u56fe3\uff1a\u6211\u56fd\u57fa\u56e0\u6cbb\u7597\u8fdb\u5c55<\/div>\n<div style=\"text-align: center;\">\uff08\u6765\u6e90\uff1aYu Jia Cai et al. Gene Therapy\uff09<\/div>\n<div>1998\u5e74FDA\u6279\u51c6\u9996\u6b3e\u57fa\u56e0\u6cbb\u7597\u4ea7\u54c1Vitravene\uff08\u5c5e\u4e8e\u77ed\u5c0f\u53cd\u4e49\u5be1\u805a\u6838\u82f7\u9178\u7c7b\u522b\uff09\u30025\u5e74\u540e\u76842003\u5e74\uff0c\u6211\u56fd\u6279\u51c6\u9488\u5bf9\u5934\u9888\u764c\u7684\u91cd\u7ec4\u817a\u75c5\u6bd2\u57fa\u56e0\u6cbb\u7597Gendicine\uff0c\u5373\u5f53\u5e74\u540d\u566a\u4e00\u65f6\u7684\u201d\u4eca\u53c8\u751f\u201c\uff0c\u7531\u6df1\u5733\u5e02\u8d5b\u767e\u8bfa\u57fa\u56e0\u6280\u672f\u6709\u9650\u516c\u53f8\u7814\u5236\uff1b2005\u5e74\uff0c\u4e0a\u6d77\u4e09\u7ef4\u751f\u7269\u6280\u672f\u6709\u9650\u516c\u53f8\u7814\u53d1\u7684\u9488\u5bf9\u9f3b\u54bd\u764c\u6eb6\u7624\u75c5\u6bd2\u836f\u7269Oncorine\u83b7\u6279\u4e0a\u5e02\u3002<\/div>\n\n<div class=\"wp-block-image aligncenter\">\n<figure class=\"\"><img decoding=\"async\" src=\"https:\/\/www.geneclub.net.cn\/wp-content\/uploads\/images\/RmFicvQBAkgAuicYOpycUdBxZFuHkE6C9Zg7hayDocvsppHwXY7AZ7tX8iaXpEv8FEvpW31P4t9k9V77ohxMWO1dQ.png\" alt=\"RmFicvQBAkgAuicYOpycUdBxZFuHkE6C9Zg7hayDocvsppHwXY7AZ7tX8iaXpEv8FEvpW31P4t9k9V77ohxMWO1dQ\"\/><\/figure><\/div>\n\n<div style=\"text-align: center;\">\u56fe4\uff1a2003-2020\u5e74\u5168\u7403\u6279\u51c6\u7684\u57fa\u56e0\u6cbb\u7597\u836f\u7269<\/div>\n<div style=\"text-align: center;\">\uff08\u6765\u6e90\uff1aYu Jia Cai et al. Gene Therapy\uff09<\/div>\n<div>\u4e4b\u540e\uff0c2003-2020\u5e74\uff0c\u8d85\u8fc710\u6b3e\u57fa\u56e0\u7597\u6cd5\u4ea7\u54c1\u5728\u5168\u7403\u83b7\u6279\u4e0a\u5e02\uff08\u5982\u56fe4\uff09\uff0cFDA\u6536\u5230900\u6b3eIND\u7533\u8bf7\u3002\u800c\u6211\u56fd\u6279\u51c6\u4e0a\u5e02\u7684\u57fa\u56e0\u6cbb\u7597\u4ea7\u54c1\u4ecd\u505c\u7559\u572815\u5e74\u524d\u3002<\/div>\n<div>\u597d\u5728\u653f\u7b56\u6b63\u9010\u6e10\u91ca\u653e\u9f13\u52b1\u521b\u65b0\u53d1\u5c55\u7684\u4fe1\u53f7\u3002<\/div>\n<div>\u00a0<\/div>\n<div>\u5728\u56fd\u5bb6\u536b\u5065\u59d4\u201c\u91cd\u5927\u65b0\u836f\u521b\u5236\u201d\u56fd\u5bb6\u79d1\u6280\u91cd\u5927\u4e13\u98792018\u5e74\u5ea6\u8bfe\u9898\u7acb\u9879\u8ba1\u5212\u4e2d\uff0c\u660e\u786e\u652f\u6301\u7ec6\u80de\u4e0e\u57fa\u56e0\u6cbb\u7597\u836f\u7269\u7b49\u521b\u65b0\u751f\u7269\u6280\u672f\u836f\u8bc4\u4ef7\u53ca\u6807\u51c6\u5316\u7814\u7a76\u30022019\u5e745\u6708\uff0c\u7ec6\u80de\u548c\u57fa\u56e0\u6cbb\u7597\u4ea7\u54c1\u4e0e\u4eba\u5de5\u667a\u80fd\u533b\u7597\u5668\u68b0\u3001\u4e2d\u836f\u5b89\u5168\u8bc4\u4ef7\u7814\u7a76\u7b49\u4e00\u8d77\u6210\u4e3aNMPA\u7684\u836f\u54c1\u76d1\u7ba1\u79d1\u5b66\u884c\u52a8\u8ba1\u5212\u7684\u9996\u6279\u7acb\u9879\u9879\u76ee\u3002<\/div>\n<div>\u00a0<\/div>\n<div>\u5728\u653f\u7b56\u548c\u8d44\u672c\u7684\u523a\u6fc0\u4e0b\uff0c\u57fa\u56e0\u6cbb\u7597\u4ea7\u4e1a\u53d1\u5c55\u5728\u8fd1\u5e74\u83b7\u5f97\u8f83\u5927\u52a0\u901f\u5ea6\u3002<\/div>\n<div>2019\u5e7412\u6708\uff0c\u535a\u96c5\u8f91\u56e0\u5728\u7f8e\u56fd\u8840\u6db2\u5b66\u5e74\u4f1a\u4e0a\u53d1\u5e03\u5176 \u03b2-\u5730\u4e2d\u6d77\u8d2b\u8840\u57fa\u56e0\u6cbb\u7597\u7684\u89c4\u6a21\u5316\u751f\u4ea7\u53ca\u4e34\u5e8a\u524d\u5b89\u5168\u6027\u548c\u6709\u6548\u6027\u8bd5\u9a8c\u6570\u636e\uff1b2020\u5e748\u6708\uff0c\u90a6\u8000\u751f\u7269\u548c\u6e58\u96c5\u533b\u9662\u5c55\u793a\u4e86\u5168\u4e16\u754c\u9996\u6b21\u57fa\u56e0\u6cbb\u7597\u03b20\/\u03b20\u578b\u91cd\u5ea6\u5730\u8d2b\u7684\u6210\u679c\uff1b9\u6708\uff0c\u548c\u5143\u751f\u7269\u5ba3\u5e03\u5b8c\u6210\u8d853\u4ebf\u5143C\u8f6e\u878d\u8d44\uff0c\u5e76\u4f5c\u4e3a\u6211\u56fd\u552f\u4e00\u4e00\u5bb6\u57fa\u56e0\u6cbb\u7597CDMO\u4f01\u4e1a\u53d7\u9080\u53c2\u52a0WHO\u7684\u6807\u51c6\u54c1\u6807\u5b9a\u9879\u76ee\uff1b\u540c\u6708\uff0c\u5409\u51ef\u57fa\u56e0\u63a5\u53d7\u4e0a\u5e02\u8f85\u5bfc\u3002<\/div>\n<div>\u6211\u56fd\u7ec6\u80de\u548c\u57fa\u56e0\u6cbb\u7597\u4ea7\u4e1a\u5316\u521a\u521a\u8d77\u6b65\uff0c\u4e9f\u9700\u4ea7\u5b66\u7814\u878d\u5408\uff0c\u5728\u65e9\u671f\u52a0\u901f\u53d1\u5c55\u4e2d\u5c55\u793a\u51fa\u653f\u7b56\u7a97\u53e3\u671f\u4e0b\u5229\u597d\u7684\u4ea7\u4e1a\u571f\u58e4\u548c\u53d1\u5c55\u524d\u666f\u3002<\/div>\n<div>\u00a0<\/div>\n<div>\u57fa\u56e0\u7f16\u8f91\u6280\u672f\u6b63\u9010\u6b65\u4ece\u79d1\u7814\u8d70\u5411\u4e34\u5e8a\uff0c\u76ee\u524d\u9700\u653b\u514b\u7684\u96be\u70b9\u5305\u62ec\uff1a<\/div>\n<div>\u53ef\u63a7\u5730\u9776\u5411\u9012\u9001<\/div>\n<div>\u57fa\u56e0\u7f16\u8f91\u7684\u7cbe\u51c6\u8868\u8fbe<\/div>\n<div>\u89c4\u6a21\u5316\u7a33\u5b9a\u751f\u4ea7\u7b49<\/div>\n<div>\u66f4\u591a\u5185\u5bb9\uff0c\u8bf7\u53c2\u8003\u57fa\u56e0\u6167\u5373\u5c06\u516c\u5f00\u53d1\u5e03\u7684\u84dd\u76ae\u4e66\u300a\u7ec6\u80de\u548c\u57fa\u56e0\u6cbb\u7597\u884c\u7814\u62a5\u544a\u300b\u3002<\/div>\n<div>\u00a0<\/div>\n<div>\u3010\u63a8\u5e7f\u3011\u8fd1\u65e5\uff0c\u57fa\u56e0\u6167\u542f\u52a8\u7b56\u5212\u300a\u7ec6\u80de\u548c\u57fa\u56e0\u6cbb\u7597\u884c\u7814\u62a5\u544a\u300b\uff0c\u5411\u884c\u4e1a\u516c\u5f00\u514d\u8d39\u53d1\u5e03\u5168\u7403\u7ec6\u80de\u548c\u57fa\u56e0\u6cbb\u7597\u653f\u7b56\u3001\u6280\u672f\u548c\u4ea7\u4e1a\u56fe\u8c31\uff0c\u6b22\u8fce\u673a\u6784\u53ca\u4e13\u5bb6\u53c2\u4e0e\u8054\u5408\u53d1\u5e03\u53ca\u8054\u5408\u64b0\u5199\u5408\u4f5c\uff08\u626b\u7801\u6216\u8054\u7cfbinfo@genonet.cn)\u3002<\/div>\n\n<div class=\"wp-block-image aligncenter\">\n<figure class=\"\"><img decoding=\"async\" src=\"https:\/\/www.geneclub.net.cn\/wp-content\/uploads\/images\/RmFicvQBAkgCwpibd4YnaAkAjuazKyR0FcSfibMDQ2o510AXTv7ialXG1tDkRwmhpEU42BbUa9sXIXKx7hmjKPJLCw.png\" alt=\"RmFicvQBAkgCwpibd4YnaAkAjuazKyR0FcSfibMDQ2o510AXTv7ialXG1tDkRwmhpEU42BbUa9sXIXKx7hmjKPJLCw\"\/><\/figure><\/div>\n\n<div>\u57fa\u56e0\u6167\u7684\u884c\u7814\u62a5\u544a\u53c2\u8003\uff1a<a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247500152&amp;idx=1&amp;sn=cb0d0456356751e8238f8c909caef357&amp;chksm=eb7173cfdc06fad91673b17999836ea987d2e0ecd266adbacd229dfb35d476727c35891586f0&amp;scene=21#wechat_redirect\">\u300a\u57fa\u56e0\u5927\u6570\u636e\u884c\u7814\u62a5\u544a\u300b<\/a><\/div>\n<div><a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247497755&amp;idx=1&amp;sn=8e70661c98f02397231418e884cc40ef&amp;chksm=eb714aacdc06c3ba5a0c3e9e402aaecc1812a6e93cc55873baa32d1fad80348366e97deca041&amp;scene=21#wechat_redirect\">\u300a\u5355\u7ec6\u80de\u884c\u7814\u62a5\u544a\u300b<\/a>\u00a0<a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247496560&amp;idx=1&amp;sn=492086c4f0896685d6a492e24f96f42a&amp;chksm=eb7141c7dc06c8d1bcd4bb734dec7864bba7caefe37e2c4a3982ef611aa0c426af6f2f5e8da9&amp;scene=21#wechat_redirect\">\u300a2020\u57fa\u56e0\u884c\u4e1a\u62a5\u544a\u300b<\/a><\/div>\n<div>\u00a0<\/div>\n<div>\u53c2\u8003\u8d44\u6599\uff1a<\/div>\n<div>1. \u77e5\u8bc6\u5206\u5b50\uff1a\u300a\u4f17\u671b\u6240\u5f52\uff1a\u4e24\u4f4d\u5973\u79d1\u5b66\u5bb6\u56e0\u4e3a\u57fa\u56e0\u7f16\u8f91\u83b7\u5316\u5b66\u8bfa\u5956\u57fa\u56e0\u7f16\u8f91\u6280\u672f\u83b7\u8bfa\u5956\uff01\u300b<\/div>\n<div>2.\u00a0Toby et al., \u201cBiopharma portfolio strategy in the era of cell and gene therapy\u201d, McKinsey, April 2020<\/div>\n<div>3. Dawei Wang et al., &#8220;An overview of development in gene therapeutics in China&#8221;,\u00a0Gene Therapy,June 2020<\/div>\n<div>\u00a0<\/div>\n<div>\u63a8\u8350\u9605\u8bfb<\/div>\n<div><a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247500152&amp;idx=1&amp;sn=cb0d0456356751e8238f8c909caef357&amp;chksm=eb7173cfdc06fad91673b17999836ea987d2e0ecd266adbacd229dfb35d476727c35891586f0&amp;scene=21#wechat_redirect\">\u57fa\u56e0\u6167\/\u534e\u5927\/\u534e\u4e3a\u7b49\u8054\u8882\u9996\u53d1\u57fa\u56e0\u5927\u6570\u636e\u62a5\u544a<\/a><\/div>\n<div><a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247499963&amp;idx=2&amp;sn=6acbe8758163be065483c130eb8ae74f&amp;chksm=eb71720cdc06fb1a1ffe64da4c5be5ee84c414894970c4b6769ecb6019ed4b52e2cee185ffcb&amp;scene=21#wechat_redirect\">Nature\u5b50\u520a | \u6211\u56fd\u57fa\u56e0\u6cbb\u7597\u4e09\u5927\u73b0\u72b6<\/a><\/div>\n<div><a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247496902&amp;idx=1&amp;sn=206fe46837c27f558a523aa23aaff27a&amp;chksm=eb714671dc06cf67b03651902bdef8279051ea95d56324a2723d1bfb8fc25710e5909abbdaac&amp;scene=21#wechat_redirect\">2019\u5168\u7403\u751f\u7269\u6280\u672f\u6295\u8d44\u4e2d\u57fa\u56e0\u6cbb\u7597\u7099\u624b\u53ef\u70ed<\/a><\/div>\n<div>\u3010\u58f0\u660e\u3011\u4e3a\u4f20\u64ad\u79d1\u5b66\u4fe1\u606f\uff0c\u63a8\u52a8\u57fa\u56e0\u53ca\u6570\u5b57\u751f\u547d\u5065\u5eb7\u4ea7\u5b66\u7814\u8fde\u63a5\uff0c\u6211\u4eec\u79c9\u6301\u4e2d\u7acb\u3001\u4e13\u4e1a\u3001\u8d4b\u80fd\u7684\u7406\u5ff5\u6536\u96c6\u3001\u5206\u6790\u6216\u53d1\u5e03\u4fe1\u606f\u3002\u4f46\u7531\u4e8e\u65f6\u6548\u6027\u53ca\u884c\u4e1a\u7279\u6b8a\u6027\uff0c\u6240\u520a\u767b\u5185\u5bb9\u4ec5\u4f9b\u7814\u7a76\u53c2\u8003\uff0c\u4e0d\u4f5c\u4e3a\u51b3\u7b56\u4f9d\u636e\uff1b\u672c\u6587\u76f8\u5173\u4fe1\u606f\u4e0d\u4ee3\u8868\u57fa\u56e0\u6167\u673a\u6784\u7684\u89c2\u70b9\uff1b\u201c\u57fa\u56e0\u6167\u201d\u520a\u767b\u7684\u539f\u521b\u5185\u5bb9\u7684\u77e5\u8bc6\u4ea7\u6743\u4e3a\u201c\u57fa\u56e0\u6167\u201d\u5546\u6807\u62e5\u6709\u8005\u53ca\u76f8\u5173\u6743\u5229\u4eba\u6240\u6709\uff1b\u6b22\u8fce\u8f6c\u8f7d\uff0c\u8f6c\u8f7d\u8bf7\u7533\u8bf7\u5e76\u6ce8\u660e\u6765\u6e90\u3002\u6b22\u8fce\u4e2a\u4eba\u53ca\u673a\u6784\u6295\u7a3f\u53ca\u5408\u4f5c\u3002<\/div>\n<div>\u00a0<\/div>\n<div>\u00a0\u5173\u4e8e\u57fa\u56e0\u6167\u00a0<\/div>\n<div>\u57fa\u56e0\u6167\u662f\u6570\u5b57\u751f\u547d\u5065\u5eb7\u9886\u57df\u521b\u65b0\u670d\u52a1\u5e73\u53f0\u3002\u56e2\u961f\u6df1\u8015\u884c\u4e1a\u5341\u4f59\u5e74\uff0c\u4ece\u884c\u7814\u54a8\u8be2\u3001\u5a92\u4f53\u8d44\u8baf\u3001\u4ea7\u4e1a\u6570\u636e\u5e93\u7b49\u65b9\u9762\u63d0\u4f9b\u4f18\u8d28\u5185\u5bb9\u670d\u52a1\u3002\u57fa\u56e0\u6167\u4f5c\u4e3a\u56fd\u53d1\u6539\u4ea7\u4e1a\u7814\u7a76\u5408\u4f5c\u5355\u4f4d\uff0c\u8054\u5408\u591a\u5bb6\u4e13\u4e1a\u673a\u6784\u53d1\u5e03\u4e86\u591a\u4efd\u57fa\u56e0\u53ca\u6570\u5b57\u751f\u547d\u5065\u5eb7\u9886\u57df\u884c\u7814\u62a5\u544a\uff0c\u53c2\u4e0e\u7ec4\u7ec7\u53d1\u5e03\u4e86\u4e24\u4efd\u884c\u4e1a\u5171\u8bc6\u548c\u4e00\u9879\u56e2\u4f53\u6807\u51c6\uff0c\u81f4\u529b\u4e8e\u5efa\u8bbe\u4ea7\u4e1a\u4fe1\u606f\u5927\u6570\u636e\u5e73\u53f0\uff0c\u670d\u52a1\u751f\u547d\u79d1\u6280\u521b\u65b0\u521b\u4e1a\u3002<\/div>\n<div>\u2606\u00a0<a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247496351&amp;idx=1&amp;sn=d50d36d5214ba002667525cb884d6590&amp;chksm=eb714028dc06c93e5de4292889b3127f14b385c3fff3e8ded5c9788b23c6a59d23b1d0d01671&amp;scene=21#wechat_redirect\">\u56fd\u53d1\u6539\u300a\u6218\u7565\u6027\u65b0\u5174\u4ea7\u4e1a\u53d1\u5c55\u5c55\u671b\u300b\u7f16\u59d4<\/a><\/div>\n<div>\u2606\u00a0\u53c2\u4e0e\u7ec4\u7ec7\u53d1\u5e03<a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247496540&amp;idx=1&amp;sn=23f504b79153aa504a6210075240d3bd&amp;chksm=eb7141ebdc06c8fd594371705ea519c453fc6d9824620bb801d8699461d116c5c79388db51fc&amp;scene=21#wechat_redirect\">\u884c\u4e1a\u5171\u8bc6<\/a>\u3001<a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247492716&amp;idx=2&amp;sn=42172a31609509cab04d3db2bb0d33a0&amp;chksm=eb7156dbdc06dfcd33b2eb613ec6aaee77893218c4531c1eda9d0cba5f855d767e430d76e7f5&amp;scene=21#wechat_redirect\">\u56e2\u4f53\u6807\u51c6<\/a><\/div>\n<div>\u2606\u00a0\u53d1\u5e03\u4ea7\u4e1a\u5927\u6570\u636e\u5e73\u53f0<a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247487212&amp;idx=1&amp;sn=eb777cac45525cc85b37a9bb65b907a6&amp;chksm=eb72bc5bdc05354d20954103276e0dd35481b5f535719f8fad3e4eeb4fe49a7b200cd2239990&amp;scene=21#wechat_redirect\">\u4f18\u8109\u901aYourMap<\/a><\/div>\n<div>\u2606\u00a0\u4e2d\u56fd\u9057\u4f20\u5b66\u4f1a\u751f\u7269\u4ea7\u4e1a\u4fc3\u8fdb\u59d4\u5458\u4f1a\u59d4\u5458<\/div>\n<div>\u2606\u00a0\u53d1\u5e03\u6570\u5341\u4efd<a 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href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247493932&amp;idx=1&amp;sn=7e68c7e9c7d5b5479e6e4aa540ca569f&amp;chksm=eb715b9bdc06d28d4944c5584d99a68977413620e4dfbb4716376a4b8a5ce7b6b1b1129b5d4f&amp;scene=21#wechat_redirect\">\u7b2c\u4e8c\u5c4a<\/a><a href=\"https:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247493932&amp;idx=1&amp;sn=7e68c7e9c7d5b5479e6e4aa540ca569f&amp;chksm=eb715b9bdc06d28d4944c5584d99a68977413620e4dfbb4716376a4b8a5ce7b6b1b1129b5d4f&amp;scene=21&amp;token=775963598&amp;lang=zh_CN#wechat_redirect\">\u4f1a\u8bae<\/a><\/div>\n<div>\u2606\u00a0\u4e3b\u529e<a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247488145&amp;idx=1&amp;sn=d62b3d6ead2512e2c448d9a9ee9ab61f&amp;chksm=eb72a026dc052930fc4a58ec38fb718549780823661d0e67740c037a222066010d10f220fa6f&amp;scene=21#wechat_redirect\">\u6570\u5b57\u5065\u5eb7\u79c1\u8463\u4f1a<\/a>\u3001<a 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