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style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">2\u6708\u4efd\uff0cIonis Pharmaceuticals\u548cBiogen\u548c\u516c\u53f8\u5728\u300aThe New England Journal of Medicine\u300b\uff08NEJM\uff09\u4e0a\u53d1\u8868\u4e863\u671f\u4e34\u5e8a\u7814\u7a76CHERISH\u7684\u7ed3\u679c\uff0c\u8bc4\u4f30\u4e86SPINRAZA\uff08nusinersen\uff0c\u901a\u8fc7\u6539\u53d8SMN2\u524d\u4f53mRNA\u7684\u526a\u63a5\u4ee5\u589e\u52a0\u5168\u957fSMN\u86cb\u767d\u751f\u6210\uff09\u6cbb\u7597\u665a\u53d1\u578b\u810a\u9ad3\u6027\u808c\u840e\u7f29\u75c7\uff08SMA\uff09\u60a3\u8005\u7684\u7597\u6548\u548c\u5b89\u5168\u6027\u3002CHERISH\u662f\u4e00\u98793\u671f\u3001\u591a\u4e2d\u5fc3\u3001\u968f\u673a\u3001\u53cc\u76f2\u3001\u5047\u624b\u672f\u5bf9\u7167\u7814\u7a76\uff0c\u4e3a\u671f15\u4e2a\u6708\uff0c\u5165\u7ec4\u4e86126\u540d\u67096\u4e2a\u6708\u4ee5\u4e0a\u75c7\u72b6\u53d1\u4f5c\u76842\uff5e12\u5c81\u975e\u95e8\u8bca\u60a3\u8005\uff0c\u4e3b\u8981\u7597\u6548\u7ec8\u70b9\u662f\u60a3\u8005\u8fd0\u52a8\u529f\u80fd\u6539\u5584\u3002\u6700\u7ec8\u7684\u5206\u6790\u8868\u660e\uff0c\u7528SPINRAZA\u6cbb\u7597\u60a3\u8005\u4e0e\u5bf9\u7167\u7ec4\u76f8\u6bd4\uff0c\u60a3\u8005\u7684\u8fd0\u52a8\u529f\u80fd\u6709\u9ad8\u5ea6\u7edf\u8ba1\u5b66\u663e\u8457\u548c\u4e34\u5e8a\u610f\u4e49\u7684\u6539\u5584\u3002<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">4\u6708\u4efd\uff0cAlnylam 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style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">5\u670824\u65e5\uff0cbluebird\u516c\u53f8\u5ba3\u5e03\u7f8e\u56fdFDA\u5df2\u6388\u4e88\u5176\u57fa\u56e0\u7597\u6cd5Lenti-D\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\uff0c\u6cbb\u7597\u8111\u80be\u4e0a\u817a\u8111\u767d\u8d28\u8425\u517b\u4e0d\u826f\uff08cerebral 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style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">\u540c\u6837\u662f5\u6708\u4efd\uff0cSangamo Therapeutics\u548cBioverativ\u516c\u53f8\u8054\u5408\u5ba3\u5e03\uff0c\u7f8e\u56fdFDA\u63a5\u53d7\u4e86\u6cbb\u7597\u9570\u72b6\u7ec6\u80de\u75c5\u7684\u5019\u9009\u57fa\u56e0\u7597\u6cd5BIVV003\u7684IND\u7533\u8bf7\u3002\u8fd9\u610f\u5473\u7740Bioverativ\u516c\u53f8\u53ef\u4ee5\u5f00\u59cb\u4e34\u5e8a1\/2\u671f\u8bd5\u9a8c\u6765\u68c0\u9a8cBIVV003\u5728\u9570\u72b6\u7ec6\u80de\u75c5\u60a3\u8005\u4e2d\u7684\u5b89\u5168\u6027\u3001\u8010\u53d7\u6027\u548c\u7597\u6548\u3002\u8fd9\u4e00\u7597\u6cd5\u7684\u4f18\u52bf\u5728\u4e8e\u4e0d\u4f9d\u9760\u75c5\u6bd2\u8f7d\u4f53\u5bf9\u57fa\u56e0\u8fdb\u884c\u7f16\u8f91\uff0c\u4ece\u800c\u907f\u514d\u75c5\u6bd2\u8f7d\u4f53\u53ef\u80fd\u5e26\u6765\u7684\u526f\u4f5c\u7528\u3002<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 16px;\"><strong><span style=\"color: rgb(0, 128, 255);\">\u4f18\u5148\u5ba1\u8bc4<\/span><\/strong><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">\u7531\u4e8e\u57fa\u56e0\u6cbb\u7597\u9488\u5bf9\u7684\u901a\u5e38\u662f\u65e0\u836f\u53ef\u6cbb\u7684\u9057\u4f20\u75c5\uff0c\u56e0\u6b64\uff0c\u5404\u56fd\u653f\u5e9c\u90fd\u9f13\u52b1\u8fd9\u65b9\u9762\u7684\u836f\u7269\u7814\u53d1\u5de5\u4f5c\uff0c\u76f8\u5e94\u7684\u5404\u79cd\u4f18\u5148\u5ba1\u8bc4\u653f\u7b56\u4e5f\u5f80\u5f80\u5782\u9752\u8fd9\u4e9b\u9879\u76ee\u3002\u5bf9\u4e8e\u57fa\u56e0\u6cbb\u7597\u836f\u7269\uff0c\u4e00\u822c\u4e0d\u4f1a\u5361\u5728\u5ba1\u8bc4\u73af\u8282\u3002<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 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style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><\/span><\/p>\n<p style=\"text-align: center;\"><img class=\"\"       style=\"\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2018\/07\/beepress1-1532683930.jpeg\" alt=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08\u7814\u7a76\u4e0a\u7bc7\uff09\" title=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08\u7814\u7a76\u4e0a\u7bc7\uff09\"  \/><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><\/span><br  \/><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 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14px;box-sizing: border-box !important;word-wrap: break-word !important;\"><strong style=\"max-width: 100%;color: rgb(170, 33, 94);line-height: 25.6px;box-sizing: border-box !important;word-wrap: break-word !important;\"><span style=\"max-width: 100%;color: rgb(0, 209, 0);line-height: 25.6px;box-sizing: border-box !important;word-wrap: break-word !important;\">\u6b22\u8fce\u6295\u7a3f\u5230\u5c0f\u7f16\u90ae\u7bb1\uff1a<strong style=\"max-width: 100%;box-sizing: border-box !important;word-wrap: break-word !important;\">wuwj06@163.com<\/strong><\/span><\/strong><\/span><strong style=\"max-width: 100%;color: rgb(170, 33, 94);line-height: 25.6px;box-sizing: border-box !important;word-wrap: break-word !important;\"><span style=\"max-width: 100%;font-size: 14px;box-sizing: border-box !important;word-wrap: break-word !important;\"><span style=\"max-width: 100%;line-height: 25.6px;font-size: 12px;\"><strong style=\"max-width: 100%;box-sizing: border-box !important;word-wrap: break-word !important;\"><\/strong><\/span><span style=\"max-width: 100%;line-height: 25.6px;\"><\/span><\/span><\/strong><\/p>\n<p style=\"white-space: normal;max-width: 100%;min-height: 1em;color: rgb(51, 51, 51);\"><img class=\"\"     style=\"line-height: 25.6px;vertical-align: middle;box-sizing: border-box !important;word-wrap: break-word !important;visibility: visible !important;max-height: 1.2em !important;width: 640px !important;\" width=\"555\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2018\/07\/beepress10-1532683930.png\" alt=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08\u7814\u7a76\u4e0a\u7bc7\uff09\" title=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08\u7814\u7a76\u4e0a\u7bc7\uff09\"  \/><br style=\"max-width: 100%;box-sizing: border-box !important;word-wrap: break-word !important;\"  \/><\/p>\n<section style=\"white-space: normal;max-width: 100%;color: rgb(51, 51, 51);\">\n<section style=\"margin-top: 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!important;word-wrap: break-word !important;\"><span style=\"max-width: 100%;color: rgb(170, 33, 94);box-sizing: border-box !important;word-wrap: break-word !important;\"><strong style=\"max-width: 100%;box-sizing: border-box !important;word-wrap: break-word !important;\"><span style=\"max-width: 100%;font-size: 18px;box-sizing: border-box !important;word-wrap: break-word !important;\"><\/span><\/strong><\/span>\u7248<\/section>\n<\/section>\n<section style=\"max-width: 100%;border-radius: 100%;width: 2em;height: 2em;text-align: center;color: rgb(255, 255, 255);line-height: 2em;vertical-align: top;display: inline-block;background-color: rgb(170, 33, 94);box-sizing: border-box !important;word-wrap: break-word !important;\">\n<section style=\"max-width: 100%;box-sizing: border-box !important;word-wrap: break-word !important;\">\u6743<\/section>\n<\/section>\n<section style=\"max-width: 100%;border-radius: 100%;width: 2em;height: 2em;text-align: center;color: rgb(255, 255, 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