{"id":9751,"date":"2018-02-02T23:05:29","date_gmt":"2018-02-02T15:05:29","guid":{"rendered":"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&#038;mid=100003539&#038;idx=1&#038;sn=7186e749963948472b39b85c103ce30c&#038;chksm=6b72bc645c053572bf31818a195ac780a80ae85625877d48c1ca51a5275342b62928b2832dde#rd"},"modified":"2026-04-09T09:31:18","modified_gmt":"2026-04-09T01:31:18","slug":"994951","status":"publish","type":"post","link":"https:\/\/www.geneclub.net.cn\/index.php\/2018\/02\/02\/994951\/","title":{"rendered":"\u5927\u836f\u5382\u5165\u5c40\uff0c\u4e2d\u56fd\u7684\u57fa\u56e0\u6cbb\u7597\u8fd8\u6709\u591a\u8fdc\uff1f\uff08\u4e0a\uff09"},"content":{"rendered":"<p><!-- wechat_content_hash: c13ecc92d79d1722 --><\/p>\n<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\/RmFicvQBAkgDEsUia5jbfibMgeRrNibH1ns0NaxVdCMqRl9Bcn0geKXKbn4CByInShB9u6lDPnKmK1nhKxqqiaRaUzQ.gif\" 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class=\"wp-block-image aligncenter\">\n<figure class=\"\"><img decoding=\"async\" src=\"https:\/\/www.geneclub.net.cn\/wp-content\/uploads\/images\/RmFicvQBAkgCee5lwYqicicV0FGib5BHzLFwSFWq06tvHncS6fa0C7xk3PKfibCvw3mP699RQPfNuABq2CHicEFASSfA.jpeg\" alt=\"RmFicvQBAkgCee5lwYqicicV0FGib5BHzLFwSFWq06tvHncS6fa0C7xk3PKfibCvw3mP699RQPfNuABq2CHicEFASSfA\"\/><\/figure><\/div>\n\n<\/div>\n<div>Evelyn\u4e00\u5bb6\uff0c\u56fe\u6765\u81ea\u7f51\u7edc<\/div>\n<div>\u800c\u65e9\u57282012\u5e74\uff0c\u516d\u5c81\u5c0f\u5973\u5b69Emily Whitehead\u60a3\u6709\u6dcb\u5df4\u7ec6\u80de\u767d\u8840\u75c5\uff0c\u5728\u63a5\u53d7\u4e86\u521b\u65b0\u6027CAR-T\u7597\u6cd5Kymriah\u4e34\u5e8a\u8bd5\u9a8c\u540e\uff0c\u764c\u75c7\u7acb\u523b\u5f97\u5230\u7f13\u89e3\uff0c\u6b64\u540e\u4e94\u5e74\u90fd\u6ca1\u6709\u5728\u4f53\u5185\u68c0\u6d4b\u5230\u764c\u7ec6\u80de[3]\u3002<\/div>\n<div><\/div>\n<div>\u201c\u51e0\u5341\u5e74\u6765\u7684\u7814\u7a76\u52aa\u529b\u5728\u4eca\u5e74\u4e3a\u7f79\u60a3\u4e25\u91cd\u548c\u7f55\u89c1\u75be\u75c5\u7684\u60a3\u8005\u5e26\u6765\u4e863\u6b3e\u57fa\u56e0\u7597\u6cd5\uff0c\u6211\u76f8\u4fe1\u57fa\u56e0\u7597\u6cd5\u4f1a\u6210\u4e3a\u6cbb\u7597\u7684\u4e3b\u6d41\uff0c\u751a\u81f3\u6709\u671b\u6cbb\u6108\u8bb8\u591a\u6700\u4e3a\u4e25\u91cd\u548c\u68d8\u624b\u7684\u75be\u75c5\u3002\u201d<\/div>\n<div><\/div>\n<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\/RmFicvQBAkgCee5lwYqicicV0FGib5BHzLFwrjZ4ibhqlpwoqt1cIe36j3FuyOxqzA1m1fRYsbGQPbNDh47piaS46KJA.png\" alt=\"RmFicvQBAkgCee5lwYqicicV0FGib5BHzLFwrjZ4ibhqlpwoqt1cIe36j3FuyOxqzA1m1fRYsbGQPbNDh47piaS46KJA\"\/><\/figure><\/div>\n\n<div style=\"text-align: center;\">\u7f8e\u56fdFDA\u5c40\u957fScottGottlieb\u57282017\u5e7412\u670819\u65e5\uff08\u7f8e\u56fd\u65f6\u95f4\uff09Luxturna\u6210\u529f\u4e0a\u5e02\u7684\u5b98\u65b9\u901a\u544a\u4e2d\u5c31\u57fa\u56e0\u6cbb\u7597\u7ed9\u4e88\u4e86\u9ad8\u5ea6\u8bc4\u4ef7\uff0c\u56fe\u7247\u6765\u81ea\u7f51\u7edc<\/div>\n<div>\u4e8c\u3001\u57fa\u56e0\u6cbb\u7597\u574e\u5777\u4e4b\u8def\uff0c\u653f\u7b56\u4ece\u672a\u505c\u6b62\u53d8\u9769<\/div>\n<div>\u7531\u4e8e\u57fa\u56e0\u6cbb\u7597\u6d89\u53ca\u5230\u6539\u53d8\u4eba\u4f53\u7684\u9057\u4f20\u57fa\u56e0\uff0c\u6240\u4ee5\u8fd9\u4e5f\u540c\u6837\u5f15\u53d1\u4e86\u5f88\u591a\u4f26\u7406\u95ee\u9898\uff0c\u540c\u65f6\u57fa\u56e0\u7597\u6cd5\u7684\u7814\u7a76\u4e4b\u8def\u4e5f\u5145\u6ee1\u4e86\u963b\u6320\u548c\u4e89\u8bae\u3002\u00a0<\/div>\n<div>\u76f4\u52301990\u5e74\u624d\u51fa\u73b0\u8f6c\u673a\u3002\u7b2c\u4e00\u4e2a\u4eba\u7c7b\u57fa\u56e0\u7597\u6cd5\u4e34\u5e8a\u8bd5\u9a8c\u83b7\u6279\u3002 \u7f8e\u56fd\u56fd\u5bb6\u536b\u751f\u7814\u7a76\u9662NIH\u5728W. French Anderson\u548cMichael Blaese\u7684\u4e0d\u61c8\u4e89\u53d6\u4e0b\uff0c\u6279\u51c6\u4e86\u9996\u4f8b\u57fa\u56e0\u6cbb\u7597\u4e34\u5e8a\u7814\u7a76\uff0c\u56db\u5c81\u7684Ashanti DeSilva\u56e0ADA\u57fa\u56e0\u7f3a\u4e4f\u5bfc\u81f4\u4e25\u91cd\u7684\u514d\u75ab\u7cfb\u7edf\u7f3a\u9677\u800c\u63a5\u53d7\u57fa\u56e0\u6cbb\u7597\uff0c\u6210\u529f\u7684\u5c06\u57fa\u56e0\u4fee\u9970\u8fc7\u7684T\u7ec6\u80de\u6ce8\u5165\u60a3\u8005\u4f53\u5185\u3002\u968f\u7740\u8fd9\u9879\u5212\u65f6\u4ee3\u610f\u4e49\u7684\u8bd5\u9a8c\u7ed3\u675f\uff0c\u5728\u5f53\u65f6\u5f15\u8d77\u4e86\u6781\u5927\u7684\u53cd\u54cd\uff0c\u91ce\u5fc3\u52c3\u52c3\u7684\u57fa\u56e0\u6cbb\u7597\u4e13\u5bb6\u72c2\u70ed\u5730\u5f00\u542f\u4e86\u4e00\u7cfb\u5217\u57fa\u56e0\u6cbb\u7597\u7814\u7a76\u3002<\/div>\n<div>\u00a0<\/div>\n<div>\u88681\uff1a2000\u5e74\u4e4b\u524d \u90e8\u5206\u57fa\u56e0\u6cbb\u7597\u76f8\u5173\u653f\u7b56\u6c47\u603b<\/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\/RmFicvQBAkgCee5lwYqicicV0FGib5BHzLFwicaKcTo2apHcEPL9g0XNmDTsgGMe6V1tj01ib1Qia4B0stexu735KebbA.png\" alt=\"RmFicvQBAkgCee5lwYqicicV0FGib5BHzLFwicaKcTo2apHcEPL9g0XNmDTsgGMe6V1tj01ib1Qia4B0stexu735KebbA\"\/><\/figure><\/div>\n\n<\/div>\n<div>\u00a0\u6ce8\uff1a\u4ee5\u4e0a\u4fe1\u606f\u6574\u7406\u81ea\u536b\u8ba1\u59d4\u3001CFDA\u3001FDA\u3001\u57fa\u56e0\u6167\u7b49[5,6,7]<\/div>\n<div><\/div>\n<div>\u4f46\u662f\u5230\u4e861999\u5e74\uff0c\u53c2\u52a0\u4e34\u5e8a\u8bd5\u9a8c\u768418\u5c81\u5c11\u5e74Jesse Gelsinger\u56e0\u4e3a\u5f3a\u70c8\u514d\u75ab\u53cd\u5e94\u6b7b\u4ea1\uff0c\u968f\u5373\u57282000\u5e741\u6708\uff0cFDA\u8fdb\u884c\u5ba1\u67e5\u540e\u51e0\u4e4e\u4e2d\u6b62\u4e86\u6240\u6709\u4e0e\u57fa\u56e0\u6cbb\u7597\u6709\u5173\u7684\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u6574\u4e2a\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u6ca6\u4e3a\u8fc7\u5ea6\u79d1\u7814\u7684\u524d\u8f66\u4e4b\u9274\uff0c\u4fc3\u4f7f\u5168\u7403\u5404\u5730\u91cd\u65b0\u5ba1\u89c6\u81ea\u5df1\u7684\u76f8\u5173\u653f\u7b56[4]\u3002<\/div>\n<div>\u88682\uff1a2001\u5e74\u81f3\u4eca \u90e8\u5206\u57fa\u56e0\u6cbb\u7597\u76f8\u5173\u653f\u7b56\u6c47\u603b<\/div>\n<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\/RmFicvQBAkgCee5lwYqicicV0FGib5BHzLFw1DOpyW0Q0VGTJUxRLZycVTHegN7836p7iagW7s5icg3Of7Td5S5G9A8A.png\" alt=\"RmFicvQBAkgCee5lwYqicicV0FGib5BHzLFw1DOpyW0Q0VGTJUxRLZycVTHegN7836p7iagW7s5icg3Of7Td5S5G9A8A\"\/><\/figure><\/div>\n\n<\/div>\n<div>\u00a0\u6ce8\uff1a\u4ee5\u4e0a\u4fe1\u606f\u6574\u7406\u81ea\u536b\u8ba1\u59d4\u3001CFDA\u3001FDA\u3001\u57fa\u56e0\u6167\u7b49[5,6,7]<\/div>\n<div><\/div>\n<div>\u5176\u4e2d\uff0c2018\u5e741\u67089\u65e5\uff0c\u4faf\u4e91\u5fb7\u9662\u58eb\u83b7\u5f97\u56fd\u5bb6\u6700\u9ad8\u79d1\u5b66\u6280\u672f\u5956\uff0c\u4f5c\u4e3a\u6700\u65e9\u7814\u5236\u7cfb\u5217\u57fa\u56e0\u5de5\u7a0b\u8f7d\u4f53\u548c\u75c5\u6bd2\u8f7d\u4f53\u7684\u79d1\u5b66\u5bb6\uff0c\u4e3a\u4e2d\u56fd\u7684\u57fa\u56e0\u6cbb\u7597\u5960\u5b9a\u4e86\u6280\u672f\u57fa\u7840\u3002<\/div>\n<div><\/div>\n<div><\/div>\n<div>\u4e09\u6280\u672f\u6846\u67b6\u68b3\u7406<\/div>\n<div><\/div>\n<div>\u57fa\u56e0\u6cbb\u7597\uff08Gene Therapy\uff09\u662f\u4e00\u79cd\u5229\u7528\u57fa\u56e0\u53bb\u6cbb\u7597\u75be\u75c5\u7684\u6280\u672f\u3002\u65e8\u5728\u5c06\u6b63\u5e38\u7684\u9057\u4f20\u7269\u8d28\u5bfc\u5165\u7ec6\u80de\u4ee5\u5f25\u8865\u57fa\u56e0\u5f02\u5e38\u5bfc\u81f4\u7684\u529f\u80fd\u6027\u86cb\u767d\u8d28\u7f3a\u5931\u3002\u5e7f\u4e49\u7684\u57fa\u56e0\u6cbb\u7597\u5305\u62ec\u4e86\u57fa\u56e0\u91cd\u7ec4\u6280\u672f\u3001\u57fa\u56e0\u7f16\u8f91\u6280\u672f\u548c\u514d\u75ab\u6cbb\u7597\u7b49\u5e94\u7528\u8f83\u4e3a\u5e7f\u6cdb\u7684\u6280\u672f\u3002<\/div>\n<div><\/div>\n<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\/RmFicvQBAkgCee5lwYqicicV0FGib5BHzLFw3Mqsg8q442xMZ7J9S8AqdJichkEdr9hicSTvej8O3W9j473cI4CEibfhw.gif\" alt=\"RmFicvQBAkgCee5lwYqicicV0FGib5BHzLFw3Mqsg8q442xMZ7J9S8AqdJichkEdr9hicSTvej8O3W9j473cI4CEibfhw\"\/><\/figure><\/div>\n\n<\/div>\n<div style=\"text-align: center;\">\u56fe\uff1a\u57fa\u56e0\u6cbb\u7597\u7528\u4e8e\u4eba\u7684\u793a\u610f\u56fe<\/div>\n<div><\/div>\n<div>\u5176\u4e2d\u57fa\u56e0\u7f16\u8f91\u7684\u51fa\u73b0\u548c\u76f8\u5e94\u6280\u672f\u7684\u8fed\u4ee3\uff0c\u5bf9\u57fa\u56e0\u6cbb\u7597\u6709\u7740\u91cd\u8981\u7684\u52a0\u901f\u4f5c\u7528\u3002<\/div>\n<div><\/div>\n<div>\u57fa\u56e0\u7f16\u8f91\uff08Gene Editing\uff09\u6280\u672f\uff0c\u7b80\u5355\u5730\u8bf4\u53ef\u4ee5\u6982\u62ec\u4e3a\u7cbe\u51c6\u5730\u7f16\u8f91\u4eba\u7c7b\u57fa\u56e0\u7ec4\u7684\u5e8f\u5217\u4ee5\u8fbe\u5230\u6cbb\u7597\u6548\u679c\uff0c\u5305\u62ec\u7ea0\u6b63\u5f15\u53d1\u75be\u75c5\u7684\u57fa\u56e0\u7a81\u53d8\uff0c\u5c06\u6b63\u5e38\u57fa\u56e0\u52a0\u5165\u5230\u57fa\u56e0\u7ec4\u4e2d\u7684\u7279\u5b9a\u4f4d\u70b9\uff0c\u4ee5\u53ca\u53bb\u9664\u6709\u5bb3\u53d8\u5f02\u7684\u57fa\u56e0\u5e8f\u5217\u3002\u8fc4\u4eca\u4e3a\u6b62\u5df2\u7ecf\u7814\u53d1\u51fa\u56db\u7c7b\u6838\u9178\u9176\uff1a\u5927\u8303\u56f4\u6838\u9178\u9176\uff0c\u950c\u6307\u6838\u9178\u9176\uff08ZFN\uff09\u3001\u8f6c\u5f55\u6fc0\u6d3b\u56e0\u5b50\u6837\u6548\u5e94\u7269\u6838\u9178\u9176\uff08TALEN\uff09\uff0c\u548c\u6700\u8fd1\u7684CRISPR\/Cas9\u7cfb\u7edf\u3002CRISPR\/Cas9\u7cfb\u7edf\u4e0e\u524d\u51e0\u4ee3\u6280\u672f\u76f8\u6bd4\uff0c\u5236\u9020\u5de5\u827a\u7b80\u5355\uff0c\u9776\u5411\u7cbe\u786e\uff0c\u6210\u672c\u76f8\u5bf9\u4f4e\u5ec9\uff0c\u4ea7\u751f\u4e86\u66f4\u5927\u7684\u5f71\u54cd\uff0c\u88ab\u300aScience\u300b\u8bc4\u9009\u4e3a2015\u5e74\u5ea6\u7a81\u7834\u3002<\/div>\n<div><\/div>\n<div>CRISPR\/Cas9\u6765\u6e90\u4e8e\u7ec6\u83cc\u7684\u83b7\u5f97\u6027\u514d\u75ab\u7cfb\u7edf\uff0c\u5229\u7528gRNA\u9776\u5411\u8bc6\u522b\u51fa\u76ee\u6807DNA\u7528Cas9\u6838\u9178\u9176\u5bf9DNA\u5207\u5272\u3002\u5728\u57fa\u56e0\u6cbb\u7597\u4e2d\uff0c\u75c5\u53d8\u7684\u7ec6\u80de\u53ef\u4ee5\u901a\u8fc7\u4e24\u79cd\u9014\u5f84\u8fdb\u884c\u57fa\u56e0\u7ea0\u6b63\uff1a\u79bb\u4f53\u548c\u4f53\u5185\u7f16\u8f91\u3002\u5728\u4f53\u5916\u6cbb\u7597\u4e2d\uff0c\u63d0\u53d6\u51fa\u4f53\u5185\u9776\u7ec6\u80de\uff0c\u7ecf\u8fc7\u6838\u9178\u9176\u4fee\u9970\u518d\u91cd\u65b0\u6ce8\u5165\u4f53\u5185\uff0c\u907f\u514d\u514d\u75ab\u6392\u65a5\u9020\u6210\u7684\u4e0d\u826f\u53cd\u5e94\uff1b\u4f53\u5185\u9014\u5f84\u5219\u662f\u76f4\u63a5\u7528\u8f7d\u4f53\u5c06\u57fa\u56e0\u64cd\u4f5c\u5de5\u5177\u5bfc\u5165\u4f53\u5185\u53d1\u6325\u4f5c\u7528\u3002\u9664\u6b64\u4e4b\u5916\uff0c\u514d\u75ab\u6cbb\u7597\u4e2d\u7684CAR-T\u7ec6\u80de\u6cbb\u7597\u4e5f\u5c5e\u4e8e\u57fa\u56e0\u6cbb\u7597\u8303\u7574\uff0c\u901a\u8fc7\u4f53\u5916\u7684\u7279\u5f02\u6027\u589e\u5f3a\u514d\u75ab\u7cfb\u7edf\u6765\u8fbe\u5230\u6cbb\u7597\u764c\u75c7\u7b49\u75be\u75c5\u3002<\/div>\n<div><\/div>\n<div><\/div>\n<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\/RmFicvQBAkgCee5lwYqicicV0FGib5BHzLFwMy4krMGOayxJDdKmIljZWV9xt6iaicAuxctT0adfIogTbmqbdtyEK1Vw.jpeg\" alt=\"RmFicvQBAkgCee5lwYqicicV0FGib5BHzLFwMy4krMGOayxJDdKmIljZWV9xt6iaicAuxctT0adfIogTbmqbdtyEK1Vw\"\/><\/figure><\/div>\n\n<div style=\"text-align: center;\">\u56fe\uff1aCAR-T\u6cbb\u7597\u7684\u5386\u53f2\u8fdb\u5c55\uff0c\u6765\u81ea\u300aScience\u300b<\/div>\n<div>\u56db\u3001\u5927\u836f\u5382\u5165\u5c40\uff0c\u57fa\u56e0\u6cbb\u7597\u4ea7\u4e1a\u8fc8\u5411\u6210\u719f<\/div>\n<div>\u7ecf\u8fc7\u8fd130\u5e74\u7684\u63a2\u7d22\u4e0e\u53d1\u5c55\uff0c\u57fa\u56e0\u6cbb\u7597\u6b63\u5f0f\u8fdb\u5165\u9ad8\u901f\u53d1\u5c55\u9636\u6bb5\uff0c\u5176\u5b89\u5168\u6027\u548c\u6709\u6548\u6027\u6b63\u9010\u6e10\u5f97\u5230\u533b\u836f\u76d1\u7ba1\u90e8\u95e8\u548c\u533b\u836f\u5de8\u5934\u7684\u8ba4\u53ef\uff0c\u8d8a\u6765\u8d8a\u591a\u7684\u4e2d\u5c0f\u578b\u6280\u672f\u578b\u516c\u53f8\u83b7\u5f97\u8d44\u672c\u7684\u9752\u7750\uff0c\u5e76\u5728\u672c\u4e16\u7eaa\u7b2c\u4e8c\u4e2a\u5341\u5e74\u5f00\u59cb\uff0c\u83b7\u5f97\u4e86\u5feb\u901f\u6210\u957f\u7684\u673a\u4f1a\u3002\u800c\u4f20\u7edf\u7684\u5236\u836f\u5de8\u5934\u5982\u845b\u5170\u7d20\u53f2\u514b\u3001\u8bfa\u534e\u3001\u8f89\u745e\u7b49\u5219\u7eb7\u7eb7\u901a\u8fc7\u5e76\u8d2d\u6216\u5408\u4f5c\u52a0\u5165\u5230\u57fa\u56e0\u6cbb\u7597\u9886\u57df\uff0c\u63a8\u52a8\u7740\u57fa\u56e0\u6cbb\u7597\u836f\u7269\u7684\u6210\u529f\u4e0a\u5e02\u3002<\/div>\n<div>\u00a0<\/div>\n<div>2012\u5e74\uff0c\u8377\u5170UniQure\u516c\u53f8\u7684Glybera\u83b7\u6b27\u76dfEMA\u5ba1\u6279\u901a\u8fc7\uff0c\u4f5c\u4e3a\u9996\u4e2a\u897f\u65b9\u56fd\u5bb6\u83b7\u6279\u7684\u57fa\u56e0\u6cbb\u7597\u836f\u7269\uff0cGlybera\u91c7\u7528\u4e86\u817a\u76f8\u5173\u75c5\u6bd2\uff08AAV\uff09\u4f5c\u4e3a\u8f7d\u4f53\uff0c\u5bf9\u4e8e\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u5177\u6709\u8de8\u65f6\u4ee3\u7684\u610f\u4e49\u3002<\/div>\n<div>\u53d7\u6b64\u5f71\u54cd\uff0c\u7f8e\u56fd\u7684\u57fa\u56e0\u6cbb\u7597\u516c\u53f8\u4e5f\u57282013\u5e74\u5439\u54cd\u4e86\u96c6\u7ed3\u53f7\uff0c\u5728\u8fd9\u4e00\u5e74\u4e2d\uff0cCAR-T\u4e09\u5de8\u5934\uff08\u8bfa\u534e\u3001Kite\u3001Juno\uff09\u7684Juno\u5ba3\u5e03\u6210\u7acb\u5e76\u5728\u540c\u5e74\u5b8c\u62101.2\u4ebf\u7f8e\u5143\u7684A\u8f6e\u878d\u8d44\uff0c\u57fa\u56e0\u6cbb\u7597\u5148\u950bSpark\u4e5f\u6b63\u5f0f\u4ece\u8d39\u57ce\u513f\u7ae5\u533b\u9662\u4e2d\u72ec\u7acb\u51fa\u6765\uff0c\u5b8c\u6210\u4e865000\u4e07\u7f8e\u5143\u7684A\u8f6e\u878d\u8d44\uff0c\u7531\u534e\u4eba\u79d1\u5b66\u5bb6\u5f20\u5cf0\u521b\u529e\u7684\u57fa\u56e0\u7f16\u8f91\u516c\u53f8Editas Medicine\u3001\u4e13\u6ce8\u4e8e\u7814\u53d1\u8840\u53cb\u75c5\u57fa\u56e0\u6cbb\u7597\u7684Dimension Therapeutics\u3001\u4ee5\u53ca\u7814\u53d1\u5e15\u91d1\u68ee\u57fa\u56e0\u6cbb\u7597\u7684Voyager Therapeutics\u4e5f\u5747\u5728\u8fd9\u4e00\u5e74\u6210\u7acb\u5e76\u5b8c\u6210\u878d\u8d44\u3002\u66f4\u6709\u610f\u601d\u7684\u662f\u6210\u7acb\u4e8e1992\u5e74\u7684\u8001\u724c\u57fa\u56e0\u6cbb\u7597\u4f01\u4e1aBluebird Bio\u4e5f\u8d76\u4e0a\u8fd9\u6ce2\u70ed\u6f6e\u987a\u5229\u5b8c\u6210IPO\uff0c\u767b\u9646\u7eb3\u65af\u8fbe\u514b\u3002\u4e00\u65f6\u4e4b\u95f4\uff0c\u7f8e\u56fd\u7684\u57fa\u56e0\u6cbb\u7597\u6982\u5ff5\u6210\u4e3a\u4e86\u8d44\u672c\u5e02\u573a\u6700\u4eae\u773c\u7684\u7248\u5757\u3002<\/div>\n<div>\u00a0<\/div>\n<div>\u5728\u8d44\u672c\u548c\u653f\u7b56\u7684\u534f\u540c\u4e0b\uff0c\u51e0\u5bb6\u65b0\u836f\u7814\u53d1\u8fdb\u5165\u52a0\u5feb\u5ba1\u67e5\u548c\u5f00\u53d1\u8282\u594f\u30022014\u548c2015\u5e74\uff0cCalledon\u3001Spark Therapeutics\u3001Bluebird Bio\u5728\u7814\u4ea7\u54c1MYDICAR\u3001SPK-RPE65\u3001\u548cLentiGlobin\u5206\u522b\u83b7\u5f97FDA\u6388\u4e88\u7684\u7a81\u7834\u6027\u7597\u6cd5\u8d44\u683c\uff0c\u8fd1\u4e9b\u5e74\u6709\u66f4\u591a\u7684\u836f\u7269\u83b7\u5f97\u7a81\u7834\u6027\u7597\u6cd5\u8d44\u683c\uff0c\u8be5\u8d44\u683c\u662f\u4e00\u4e2a\u5feb\u901f\u901a\u9053\uff0c\u80fd\u591f\u5e2e\u52a9\u5728\u7814\u836f\u7269\u83b7\u5f97\u4f18\u5148\u5ba1\u8bc4\u5e76\u52a0\u901f\u6279\u51c6\u3002\u57fa\u56e0\u6cbb\u7597\u662f\u83b7\u5f97\u8be5\u8d44\u683c\u6700\u591a\u7684\u9886\u57df\u4e4b\u4e00\u3002<\/div>\n<div>2016\u5e745\u6708\uff0c\u5168\u7403\u5236\u836f\u5de8\u5934\u845b\u5170\u7d20\u53f2\u514b\u516c\u53f8\u7684\u57fa\u56e0\u7597\u6cd5Strimvelis\u5728\u6b27\u6d32\u83b7\u6279\uff0c\u6210\u4e3a\u4e86\u4e16\u754c\u4e0a\u7b2c\u4e00\u4e2a\u7528\u4e8e\u513f\u7ae5\u7f3a\u9677\u57fa\u56e0\u4fee\u590d\u7684\u5546\u4e1a\u5316\u57fa\u56e0\u7597\u6cd5\uff0c\u540c\u65f6\uff0c\u8fd9\u4e5f\u88ab\u89c6\u4e3a\u4f20\u7edf\u5927\u836f\u5382\u63a8\u52a8\u57fa\u56e0\u6cbb\u7597\u8d70\u5411\u5e02\u573a\u7684\u91cc\u7a0b\u7891\u4e8b\u4ef6\u3002\u5176\u5b9e\uff0c\u5c31\u5546\u4e1a\u5229\u6da6\u800c\u8a00\uff0cStrimvelis\u7684\u4e0a\u5e02\u5bf9\u845b\u5170\u7d20\u53f2\u514b\u516c\u53f8\u662f\u5fae\u4e0d\u8db3\u9053\u7684\uff0c\u4f46\u4f20\u7edf\u5927\u836f\u5382\u5374\u613f\u610f\u5728\u65b0\u65f6\u4ee3\u6765\u4e34\u524d\u505a\u597d\u5145\u8db3\u7684\u51c6\u5907\uff0c\u5728\u4ef7\u683c\u548c\u4ed8\u6b3e\u65b9\u5f0f\u4e0a\u505a\u51fa\u4e86\u8bf8\u591a\u5c1d\u8bd5\uff0c\u5e76\u4e14\u79ef\u6781\u63a8\u52a8\u7740\u6cbb\u7597\u573a\u5730\u56fa\u5b9a\u7b49\u5c40\u9650\u6027\u95ee\u9898\u3002<\/div>\n<div>\u00a0<\/div>\n<div>\u65f6\u95f4\u8fdb\u51652017\u5e74\u3002\u521a\u521a\u5728\u4e2d\u56fd\u5b8c\u6210\u5e03\u5c40\u7684Kite Pharma\u88abGilead\u4ee5119\u4ebf\u7f8e\u5143\u6536\u8d2d\u3001Juno\u4e5f\u4e8e\u8fd1\u65e5\u88ab\u751f\u7269\u5236\u836f\u9f99\u5934Celgene\u6536\u5165\u56ca\u4e2d\uff0c\u968f\u7740\u4f20\u7edf\u5927\u836f\u5382\u4eec\u4e0d\u65ad\u8fdb\u5165\u57fa\u56e0\u6cbb\u7597\u4ea7\u4e1a\u4e0b\u6e38\uff0c\u57fa\u56e0\u6cbb\u7597\u6b63\u5f0f\u6b65\u5165\u4ea7\u4e1a\u6210\u719f\u671f\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\/RmFicvQBAkgCee5lwYqicicV0FGib5BHzLFw26gqKn7dDDiaqRb6hVjzPsOtBR1YYFhicWqILRyhJhlvjJ7mP5KEiaPlg.jpeg\" alt=\"RmFicvQBAkgCee5lwYqicicV0FGib5BHzLFw26gqKn7dDDiaqRb6hVjzPsOtBR1YYFhicWqILRyhJhlvjJ7mP5KEiaPlg\"\/><\/figure><\/div>\n\n<\/div>\n<div style=\"text-align: center;\">\u56fe\uff1a\u57fa\u56e0\u6cbb\u7597\u884c\u4e1a\u4ea7\u4e1a\u94fe\u793a\u610f\u56fe<\/div>\n<div><\/div>\n<div>\u636eclinicaltrials.gov\u7edf\u8ba1\uff0c\u76ee\u524d\u6ce8\u518c\u7684\u57fa\u56e0\u7597\u6cd5\u4e34\u5e8a\u8bd5\u9a8c\u7ea6\u6709469\u9879\uff0c\u5176\u4e2d\u6b63\u5728\u8fdb\u884c\u7684208\u9879\uff0c\u5df2\u7ecf\u5b8c\u6210\u4e86156\u9879\u3002\u56de\u770b\u56fd\u5185\uff0c\u7ee72017\u5e74\u56fd\u5185\u591a\u4e2aCAR-T\u4e34\u5e8a\u8bd5\u9a8c\u53d7CFDA\u53d7\u7406\u540e\uff0c\u67093\u4e2aCAR-T\u7597\u6cd5\u5df2\u7ecf\u4e8e\u4e00\u6708\u4efd\u88ab\u62df\u7eb3\u5165\u6709\u9650\u5ba1\u8bc4\u836f\u54c1\u540d\u5355\uff0c\u56fd\u5185\u7684\u5b89\u79d1\u751f\u7269\u3001\u56fd\u9645\u7684GE\u533b\u7597\u7b49\u57fa\u56e0\u6cbb\u7597\u4e0a\u6e38\u4f01\u4e1a\u5728\u540c\u5b89\u79d1\u751f\u7269\u7b49\u56fd\u5185\u57fa\u56e0\u6cbb\u7597\u4e2d\u4e0b\u6e38\u4f01\u4e1a\u7684\u5408\u4f5c\u8054\u52a8\uff0c\u4ea7\u4e1a\u7684\u521d\u671f\u683c\u5c40\u6b63\u9010\u6e10\u5f62\u6210\u3002<\/div>\n<div><\/div>\n<div>2018\u65b0\u5e74\u4f0a\u59cb\uff0c\u57fa\u56e0\u6cbb\u7597\u8d44\u672c\u5316\u5f00\u59cb\u62ac\u5934\u3002<\/div>\n<div>\u751f\u7269\u5236\u836f\u9f99\u5934Celgene\u5ba3\u5e03\u4ee590\u4ebf\u7f8e\u5143\u6536\u8d2d\u80bf\u7624\u514d\u75ab\u6cbb\u7597\u6807\u5fd7\u6027\u4f01\u4e1aJuno\uff1b\u8bfa\u534e\u548cSpark\u5ba3\u5e03\u5408\u4f5c\uff0c\u8fbe\u6210\u4e00\u9879\u4ef7\u503c1.7\u4ebf\u7f8e\u5143\u7684\u57fa\u56e0\u7597\u6cd5\u7814\u53d1\u63a8\u5e7f\u534f\u8bae\u3002<\/div>\n<div><\/div>\n<div><\/div>\n<div>\u4e94\u8ba8\u8bba\uff1a\u57fa\u56e0\u6cbb\u7597\u672a\u6765\u7684\u673a\u9047\u548c\u6311\u6218<\/div>\n<div><\/div>\n<div>1\uff09\u5b89\u5168\u6027 \u968f\u7740\u57fa\u56e0\u6cbb\u7597\u9488\u5bf9\u7684\u75be\u75c5\u6108\u53d1\u590d\u6742\uff0c\u57fa\u56e0\u6cbb\u7597\u7684\u5b89\u5168\u6027\u548c\u65f6\u6548\u6027\u9010\u6e10\u6210\u4e3a\u4e86\u65b0\u7684\u6311\u6218\uff0c\u4f34\u968f\u7740\u7684\u8981\u6c42\u5c06\u4f1a\u66f4\u9ad8\u3002\u6cbb\u7597\u57fa\u56e0\u7684\u53ef\u6301\u7eed\u6027\u548c\u53ef\u8c03\u63a7\u6027\u8868\u8fbe\u662f\u672a\u6765\u57fa\u56e0\u6cbb\u7597\u7684\u53c8\u4e00\u4e2a\u91cd\u5927\u6311\u6218\u3002<\/div>\n<div><\/div>\n<div>\u00a02\uff09\u6280\u672f\u53ef\u884c\u6027\u4ee5\u53ca\u77e5\u8bc6\u4ea7\u6743\u95ee\u9898\u3002 \u6211\u4eec\u5728\u4e0a\u6587\u4e2d\u4ecb\u7ecd\u4e86\u8bb8\u591a\u6210\u529f\u4ee5\u53ca\u5931\u8d25\u7684\u6848\u4f8b\u3002\u8bf8\u5982\u6b7b\u4e8e\u5f3a\u70c8\u514d\u75ab\u53cd\u5e94\u7684Jesse Gelsinger\uff0c\u8fd9\u4e00\u95ee\u9898\u540c\u6837\u62f7\u95ee\u7740Juno\u516c\u53f8\u3002\u5c3d\u7ba1\u76ee\u524d\u7814\u7a76\u6570\u636e\u8bc1\u660eCAR-T\u6709\u671b\u80fd\u591f\u5f15\u53d1\u80bf\u7624\u6cbb\u7597\u7684\u9769\u547d\uff0c\u4f46\u662f\u53d7\u9650\u4e8e\u4e34\u5e8a\u7814\u7a76\u60a3\u8005\u6570\u76ee\uff0c\u8fd9\u4e00\u7ed3\u679c\u53ef\u80fd\u65e0\u6cd5\u9002\u7528\u4e8e\u6574\u4e2a\u80bf\u7624\u6cbb\u7597\u5e02\u573a\u3002<\/div>\n<div><\/div>\n<div>\u00a03\uff09\u75be\u75c5\u9776\u6807\u53ca\u9002\u5e94\u75c7\u72ed\u7a84\u3002 \u57fa\u56e0\u6cbb\u7597\u76ee\u524d\u9650\u4e8e\u57fa\u7840\u7814\u7a76\u7684\u9650\u5236\uff0c\u5728\u9002\u5e94\u75c7\u4e0a\u5e76\u6ca1\u6709\u592a\u591a\u7684\u9009\u62e9\u6027\uff0c\u800c\u4e00\u4e9b\u5e38\u89c1\u75be\u75c5\u5219\u7531\u4e8e\u673a\u7406\u590d\u6742\uff0c\u76ee\u524d\u5e76\u4e0d\u662f\u57fa\u56e0\u6cbb\u7597\u7684\u9996\u9009\u3002\u5982\u4f55\u5408\u7406\u4e14\u6709\u6548\u7684\u5f00\u5c55\u836f\u7269\u9002\u5e94\u75c7\u7684\u9009\u62e9\u4e9f\u5f85\u89e3\u51b3\u3002<\/div>\n<div><\/div>\n<div>4\uff09\u4e2a\u4f53\u5316\u6cbb\u7597 \u5728\u7cbe\u51c6\u533b\u7597\u65f6\u4ee3\uff0c\u4e2a\u4f53\u5316\u662f\u672a\u6765\u75be\u75c5\u8bca\u7597\u7684\u4e3b\u8981\u53d1\u5c55\u65b9\u5411\u3002\u00a0<\/div>\n<div><\/div>\n<div>5\uff09 \u836f\u7269\u6210\u672c \u8bf8\u5982CAR-T\u7b49\u4e2a\u4f53\u5316\u7597\u6cd5\u5728\u6cbb\u7597\u524d\u7684\u7ec6\u80de\u5236\u5907\u8fc7\u7a0b\u4e2d\u9700\u8981\u56e0\u4eba\u800c\u5f02\u5e76\u4e0d\u80fd\u5b8c\u6210\u89c4\u6a21\u5316\u751f\u4ea7\uff0c\u8fd9\u76f4\u63a5\u5bfc\u81f4\u4e86\u6210\u672c\u98d9\u5347\uff0c\u96be\u4ee5\u666e\u53ca\u3002<\/div>\n<div><\/div>\n<div>6\uff09\u5b9a\u4ef7\u8fc7\u9ad8 2017\u5e744\u6708\uff0c\u5168\u7403\u6700\u8d35\u836f\u7269Glybera\u4e0b\u67b6\uff0c\u8fd9\u6b3e\u9ad8\u8fbe120\u4e07\u7f8e\u91d1\u7684\u57fa\u56e0\u6cbb\u7597\u836f\u7269\u6b63\u5f0f\u63a8\u51fa\u4e86\u5386\u53f2\u821e\u53f0\u3002\u8fd1\u65e5\uff0c\u7f8e\u56fd\u4e34\u5e8a\u8bc4\u4f30\u4e0e\u5ba1\u67e5\u7814\u7a76\u6240\uff08ICER\uff09\u5c31\u6700\u65b0\u4e0a\u5e02\u7684\u201cLuxturna\u201d\u7684\u4ef7\u683c\u53d1\u8868\u4e86\u610f\u89c1\uff0c\u5728\u4ed6\u4eec\u4e00\u4efd\u6700\u65b0\u7684\u62a5\u544a\u4e2d\u79f0\uff1a\u4ece\u7ed9\u793e\u4f1a\u5e26\u6765\u7684\u7ecf\u6d4e\u5229\u76ca\u65b9\u9762\u6765\u770b\uff0c\u201cLuxturna\u201d\u57fa\u56e0\u7597\u6cd5\u7684\u4ef7\u683c\u6bd4\u9884\u671f\u81f3\u5c11\u9ad8\u51fa\u4e86\u4e00\u500d\u3002\u00a0<\/div>\n<div><\/div>\n<div>7\uff09\u793e\u4f1a\u53ca\u4f26\u7406 \u9664\u4e86\u653f\u5e9c\u90e8\u95e8\u7684\u76d1\u7ba1\u5916\uff0c\u8bf8\u5982\u4eba\u6743\u3001\u6ee5\u7528\u3001\u57fa\u56e0\u6b67\u89c6\u7b49\u95ee\u9898\u8fd8\u9700\u8981\u5168\u793e\u4f1a\u4e00\u8d77\u9762\u5bf9\u53ca\u53c2\u4e0e\u3002<\/div>\n<div><\/div>\n<div>\u53c2\u8003\u8d44\u6599\uff1a<\/div>\n<div>1.www.sciencemag.org\\news\\2017\\11\\gene-therapy-s-new-hope-neuron-targeting-virus-saving-infant-lives<\/div>\n<div>2.www.nejm.org\\doi\\full\\10.1056\\NEJMoa1706198www.cancerresearch.org\/we-are-cri\/cri-impact\/stories\/patients\/emily-whitehead<\/div>\n<div>3.www.nature.com\/articles\/3302354www.fda.gov\/forpatients\/approvals\/fast\/ucm405397.htm<\/div>\n<div>4.www.fda.gov\/downloads\/BiologicsBloodVaccines\/GuidanceComplianceRegulatoryInformation\/Guidances\/CellularandGeneTherapy\/UCM376521.pdf<\/div>\n<div>5.www.nhfpc.gov.cn\/yzygj\/s3585\/201507\/c529dd6bb8084e09883ae417256b3c49.shtml<\/div>\n<div>6.www.sda.gov.cn\/WS01\/CL0237\/15708.html<\/div>\n<div>7.www.nhfpc.gov.cn\/mohbgt\/s9507\/200903\/39511.shtml<\/div>\n<div><\/div>\n<div>\u7f16\u8005\uff1a\u5c18\u7070\u00a0|\u00a0\u6821\u5bf9\uff1aCindy\u00a0 | \u5ba1\u6838\uff1a\u5e03\u4e09\u5c11<\/div>\n<div><\/div>\n<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\/RmFicvQBAkgDnic1wvB8FoQwHpw7g70E2SVjbsDzSfht7UDRia5Q6iciax9PofkezPCxialjm24IwepFC8atiaDuwu75A.gif\" alt=\"RmFicvQBAkgDnic1wvB8FoQwHpw7g70E2SVjbsDzSfht7UDRia5Q6iciax9PofkezPCxialjm24IwepFC8atiaDuwu75A\"\/><\/figure><\/div>\n\n<\/div>\n<div><\/div>\n<div>\u76f8\u5173\u9605\u8bfb<\/div>\n<p><a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247486454&amp;idx=1&amp;sn=14508dce5aa4ee1dd27d7e4f444a67b6&amp;chksm=eb72b941dc05305717a5a221fa625242a50e272bce65ede14fc4bb57b2163676bafd2b8cb0ba&amp;scene=21#wechat_redirect\" \/><\/a><a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247486715&amp;idx=1&amp;sn=8127d176bb1fdf2a0eeeacd4b9304b1a&amp;chksm=eb72be4cdc05375ae86895fe8af76f1c32869f7e3ed5ebb8c4bb90004e4fea819a81a5206aef&amp;scene=21#wechat_redirect\" \/><\/a><a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247486918&amp;idx=1&amp;sn=b9f55d07dfb7ee94ee04a1d092b4af55&amp;chksm=eb72bf71dc05366795abedcd93e153194133698aef7e7ad36c7987d7dd9a3bb247049e74be7d&amp;scene=21#wechat_redirect\" \/><\/a><a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247486918&amp;idx=1&amp;sn=b9f55d07dfb7ee94ee04a1d092b4af55&amp;chksm=eb72bf71dc05366795abedcd93e153194133698aef7e7ad36c7987d7dd9a3bb247049e74be7d&amp;scene=21#wechat_redirect\" \/><\/a><a href=\"https:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247486937&amp;idx=1&amp;sn=24d2a390b24c200cd2072fcf8dab9c31&amp;chksm=eb72bf6edc0536782aff5722ab4eadd528e7e0e4d8887d3c360b77c6fc73caf106c69d40c082&amp;scene=21#wechat_redirect\" \/><\/a><a href=\"https:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247486506&amp;idx=1&amp;sn=23ab4fc6d5f69380767bc72e25bb64cc&amp;chksm=eb72be9ddc05378b8eec8dabf0e46fe4096901d6b2cbe189fb6244dd8b7a5c9ebbcd13073a20&amp;scene=21#wechat_redirect\" \/><\/a><a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzIzMzE4MzU3NA==&amp;mid=2651633639&amp;idx=2&amp;sn=b715c9e25d1108fce16fb74d2149278d&amp;chksm=f37197dec4061ec843b609f3cfc54734bf8b0588ebc6551c71ef21967c00476fc39d6b46e5b1&amp;scene=21#wechat_redirect\" \/>\u00a0\u9b4f\u4e8e\u5168\u9662\u58eb\uff1a\u751f\u7269\u6cbb\u7597\u4e3a\u764c\u75c7\u6cbb\u7597\u5e26\u6765\u65b0\u5e0c\u671b\u00a0<\/a><a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzIzMzE4MzU3NA==&amp;mid=2651633704&amp;idx=1&amp;sn=de0702ed44bc2adbbb6ff781f3a79606&amp;chksm=f3719811c4061107488da975e553f77a0df584592883c91abc3f91c9dcc8088748aaa27c9503&amp;scene=21#wechat_redirect\" \/>\u91d1\u9e3d\uff1a\u521b\u4e1a\u5c31\u662f\u628a\u5fd7\u540c\u9053\u5408\u7684\u4eba\u805a\u4e00\u8d77\u505a\u6709\u610f\u4e49\u7684\u4e8b<\/a>\u00a0 \u00a0 \u00a0<a href=\"http:\/\/mp.weixin.qq.com\/s?__biz=MzI3NjUzOTM1Mg==&amp;mid=2247486689&amp;idx=1&amp;sn=0e23e47cf0cc38c8c5c048bae5872877&amp;chksm=eb72be56dc053740efbb9d4748e4c1bfa879ede9d0a93097330cffa9ceba83e5aff7fe67aaae&amp;scene=21#wechat_redirect\" \/>\u5f00\u653e\u7684\u653f\u7b56\u52a0\u901f\u4ea7\u4e1a\u751f\u6001\u5f62\u6210 | \u57fa\u56e0\u884c\u4e1a2017\u5e74\u5ea6\u76d8\u70b9\u3010\u653f\u7b56\u7bc7\u3011<\/a><a 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