{"id":8394,"date":"2018-06-14T01:00:00","date_gmt":"2018-07-27T09:32:02","guid":{"rendered":"http:\/\/www.05921.net\/index.php\/2018\/07\/27\/2018%e5%b9%b4%e5%85%a8%e7%90%83%e5%9f%ba%e5%9b%a0%e6%b2%bb%e7%96%97%e9%a2%86%e5%9f%9f%e8%bf%9b%e5%b1%95%ef%bc%88%e7%a0%94%e7%a9%b6%e4%b8%8b%e7%af%87%ef%bc%89\/"},"modified":"2018-07-27T17:32:03","modified_gmt":"2018-07-27T09:32:03","slug":"2018%e5%b9%b4%e5%85%a8%e7%90%83%e5%9f%ba%e5%9b%a0%e6%b2%bb%e7%96%97%e9%a2%86%e5%9f%9f%e8%bf%9b%e5%b1%95%ef%bc%88%e7%a0%94%e7%a9%b6%e4%b8%8b%e7%af%87%ef%bc%89","status":"publish","type":"post","link":"https:\/\/www.05921.net\/index.php\/8394\/","title":{"rendered":"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08\u7814\u7a76\u4e0b\u7bc7\uff09"},"content":{"rendered":"<div class=\"bpp-post-content\">\n<p style=\"white-space: normal;max-width: 100%;min-height: 1em;color: rgb(51, 51, 51);text-align: center;\"><span style=\"max-width: 100%;color: rgb(0, 209, 0);font-size: 14px;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;\"><\/strong><\/span><\/p>\n<p style=\"white-space: normal;max-width: 100%;min-height: 1em;color: rgb(51, 51, 51);\"><strong style=\"max-width: 100%;box-sizing: border-box !important;word-wrap: break-word !important;overflow-wrap: break-word !important;\"><span style=\"max-width: 100%;font-size: 15px;color: rgb(0, 82, 255);box-sizing: border-box !important;word-wrap: break-word !important;overflow-wrap: break-word !important;\">\u4f5c\u8005 l&nbsp;<strong style=\"max-width: 100%;color: rgb(62, 62, 62);font-size: 16px;line-height: 25.6px;box-sizing: border-box !important;word-wrap: break-word !important;overflow-wrap: break-word !important;\"><span style=\"max-width: 100%;font-size: 15px;color: rgb(0, 82, 255);box-sizing: border-box !important;word-wrap: break-word !important;overflow-wrap: break-word !important;\">\u5403\u74dc\u7fa4\u4f17<\/span><\/strong><span style=\"max-width: 100%;\"><\/span><\/span><\/strong><\/p>\n<p style=\"white-space: normal;max-width: 100%;min-height: 1em;color: rgb(51, 51, 51);\"><strong style=\"max-width: 100%;box-sizing: border-box !important;word-wrap: break-word !important;overflow-wrap: break-word !important;\"><span style=\"max-width: 100%;font-size: 15px;color: rgb(0, 82, 255);box-sizing: border-box !important;word-wrap: break-word !important;overflow-wrap: break-word !important;\">\u7f16\u8f91&nbsp;<strong style=\"max-width: 100%;box-sizing: border-box !important;word-wrap: break-word !important;overflow-wrap: break-word !important;\"><span style=\"max-width: 100%;box-sizing: border-box !important;word-wrap: break-word !important;overflow-wrap: break-word !important;\"><strong style=\"max-width: 100%;box-sizing: border-box !important;word-wrap: break-word !important;overflow-wrap: break-word !important;\"><span style=\"max-width: 100%;box-sizing: border-box !important;word-wrap: break-word !important;overflow-wrap: break-word !important;\">l<\/span><\/strong>&nbsp;Childlikecat<\/span><\/strong><\/span><\/strong><\/p>\n<p style=\"white-space: normal;max-width: 100%;min-height: 1em;color: rgb(51, 51, 51);\"><br  \/><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">\u5728\u4e0a\u4e00\u7bc7\u4e2d\uff0c\u5c0f\u7f16\u7ed9\u5927\u5bb6\u68b3\u74062018\u5e74\u4ee5\u6765\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u7684\u5df2\u7ecf\u6216\u6709\u671b\u8fd1\u671f\u4e0a\u5e02\u7684\u57fa\u56e0\u6cbb\u7597\u4ea7\u54c1\uff0c\u91cd\u70b9\u7ed9\u5927\u5bb6\u4ecb\u7ecd\u4e86\u56db\u5bb6\u4ee3\u8868\u6027\u516c\u53f8\uff0c\u540c\u65f6\u4ecb\u7ecd\u4e86\u90e8\u5206\u83b7\u5f97\u4f18\u5148\u5ba1\u8bc4\u8d44\u683c\u7684\u57fa\u56e0\u6cbb\u7597\u4ea7\u54c1\u3002\u8fd9\u90e8\u5206\uff0c\u5c0f\u7f16\u91cd\u70b9\u4ecb\u7ecd\u5c1a\u5904\u5728\u65e9\u671f\u4e34\u5e8a\u53ca\u90e8\u5206\u4e34\u5e8a\u524d\u7684\u7814\u7a76\u5de5\u4f5c\u3002<\/span><\/p>\n<p style=\"text-align: justify;\"><br  \/><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 16px;\"><strong><span style=\"font-size: 16px;color: rgb(0, 128, 255);\">\u8840\u53cb\u75c5\u3001\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u3001\u5e15\u91d1\u68ee\u75c5<\/span><\/strong><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">5\u6708\u4efd\uff0c3\u5bb6\u516c\u53f8\u5206\u522b\u516c\u5e03\u4e86\u8840\u53cb\u75c5\u57fa\u56e0\u6cbb\u7597\u7684\u6700\u65b0\u4e34\u5e8a\u8fdb\u5c55\u3002BioMarin Pharmaceuticals\u516c\u53f8\u57282018\u5e74\u4e16\u754c\u8840\u53cb\u75c5\u8054\u5408\u4f1a\uff08WFH\uff09\u4e16\u754c\u5927\u4f1a\u4e0a\u516c\u5e03\u4e86\u8be5\u516c\u53f8\u6cbb\u7597A\u578b\u8840\u53cb\u75c5\u7684\u65b0\u7597\u6cd5valoctogogene roxaparvovec\u5728\u5f00\u653e\u6807\u7b7e1\/2\u671f\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u7684\u6700\u65b0\u7ed3\u679c\u3002\u8be5\u8bd5\u9a8c\u7ed3\u679c\u8868\u660e\uff0c\u8be5\u7597\u6cd5\u80fd\u591f\u5728\u63a5\u53d7\u6cbb\u7597\u540e\u7b2c\u4e8c\u5e74\u6d88\u9664\u60a3\u8005\u63a5\u53d7\u9884\u9632\u6027\u51dd\u8840\u56e0\u5b50\u7597\u6cd5\u7684\u9700\u6c42\uff0c\u800c\u4e14\u60a3\u8005\u4e0d\u4f1a\u51fa\u73b0\u81ea\u53d1\u51fa\u8840\u4e8b\u4ef6\uff0c\u751f\u6d3b\u8d28\u91cf\u6307\u6570\u5728\u63a5\u53d7\u6cbb\u7597\u540e\u7b2c\u4e8c\u5e74\u7ee7\u7eed\u4e0a\u5347\u3002\u8be5\u7597\u6cd5\u5df2\u7ecf\u83b7\u5f97\u7f8e\u56fdFDA\u6388\u4e88\u7684\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\u3002<\/span><\/p>\n<p><img class=\"\"       style=\"\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2018\/07\/beepress4-1532683923.jpeg\" alt=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08\u7814\u7a76\u4e0b\u7bc7\uff09\" title=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08\u7814\u7a76\u4e0b\u7bc7\uff09\"  \/><\/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;\">\u540c\u6837\u662f\u5728WFH\u4e16\u754c\u5927\u4f1a\u4e0a\uff0c\u521a\u521a\u4e8e\u53bb\u5e74\u5e74\u5e95\u65a9\u83b7\u7f8e\u56fd\u9996\u4e2a\u57fa\u56e0\u6cbb\u7597\u836f\u7269\u7684Spark Therapeutics\u4e0e\u8f89\u745e\u516c\u53f8\u5ba3\u5e03\uff0c\u4e24\u8005\u8054\u5408\u5f00\u53d1\u7684SPK-9001\u57281\/2\u671f\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u8868\u73b0\u4f18\u5f02\uff0c\u63a5\u53d7\u6cbb\u7597\u768415\u540d\u4e2d\u5ea6\u81f3\u91cd\u5ea6B\u578b\u8840\u53cb\u75c5\u60a3\u8005\u5df2\u7ecf\u505c\u6b62\u4e86\u5e38\u89c4\u8f93\u6ce8\u56e0\u5b50IX\u6d53\u7f29\u7269\u3002SPK-9001\u662f\u4e00\u79cd\u8fd0\u7528\u751f\u7269\u5de5\u7a0b\u6280\u672f\u8bbe\u8ba1\u7684AAV\u8f7d\u4f53\uff0c\u542b\u6709\u5bc6\u7801\u5b50\u4f18\u5316\u7684\u9ad8\u6d3b\u6027\u4eba\u7c7bFactor IX\u57fa\u56e0\u3002\u901a\u8fc7\u9759\u8109\u8f93\u6ce8\u5bfc\u5165\u4f53\u5185\u540e\uff0c\u8fd9\u4e00\u8f7d\u4f53\u80fd\u591f\u5728\u809d\u810f\u8868\u8fbe\u9ad8\u6d3b\u6027\u7684\u56e0\u5b50IX\u3002\u8be5\u7597\u6cd5\u66fe\u83b7\u5f97FDA\u9881\u53d1\u7684\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\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;\">\u6700\u540e\u4e00\u9879\u7a81\u7834\u6765\u81ea\u8377\u5170\u7684\u57fa\u56e0\u6cbb\u7597\u516c\u53f8uniQure\uff0c\u5176\u4e8e2012\u5e74\u7814\u53d1\u4e86\u9996\u4e2a\u6b27\u76df\u4e0a\u5e02\u7684\u57fa\u56e0\u6cbb\u7597\u836f\u7269\u3002\u5728\u91cd\u65b0\u5206\u67901\/2\u671f\u4e34\u5e8a\u8bd5\u9a8cAMT-060\u4e2d10\u4f8b\u60a3\u8005\u7684\u6cbb\u7597\u524d\u8840\u6e05\u6837\u672c\u540e\uff0c\u8be5\u516c\u53f8\u6ca1\u6709\u53d1\u73b0\u6cbb\u7597\u524d\u6297AAV5\u4e2d\u548c\u6297\u4f53\u7684\u5b58\u5728\u4e0eAMT-060\u5728B\u578b\u8840\u53cb\u75c5\u60a3\u8005\u4e2d\u7684\u4e34\u5e8a\u7ed3\u679c\u6709\u5173\uff0c\u8fd9\u4e9b\u7ed3\u679c\u53d1\u8868\u5728\u7f8e\u56fd\u57fa\u56e0\u4e0e\u7ec6\u80de\u7597\u6cd5\u534f\u4f1a\uff08ASGCT\uff09\u5e74\u4f1a\u4e0a\u3002AMT-060\u4f7f\u75285\u578b\u817a\u76f8\u5173\u75c5\u6bd2\uff08AAV5\uff09\u5c06\u7279\u5f02\u6027\u5728\u809d\u810f\u7ec6\u80de\u4e2d\u8868\u8fbe\u7684Factor IX\u57fa\u56e0\u6ce8\u5165\u60a3\u8005\u4f53\u5185\u3002\u901a\u8fc7\u4e00\u6b21\u6ce8\u5c04\u5c31\u53ef\u4ee5\u4f7f\u60a3\u8005\u5728\u76f8\u5f53\u957f\u4e00\u6bb5\u65f6\u95f4\u5185\u7a33\u5b9a\u5730\u8868\u8fbe\u56e0\u5b50IX\uff0c\u4ece\u800c\u4f7f\u60a3\u8005\u5728\u65e0\u9700\u5b9a\u671f\u8f93\u8840\u7684\u60c5\u51b5\u4e0b\uff0c\u7ef4\u6301\u81ea\u4e3b\u51dd\u8840\u529f\u80fd\uff0c\u663e\u8457\u6539\u5584\u60a3\u8005\u751f\u6d3b\u8d28\u91cf\u3002\u8be5\u7597\u6cd5\u5df2\u7ecf\u83b7\u5f97\u7f8e\u56fdFDA\u6388\u4e88\u7684\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\u3002<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><\/span><\/p>\n<p><img class=\"\"       style=\"\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2018\/07\/beepress9-1532683923-1.jpeg\" alt=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08\u7814\u7a76\u4e0b\u7bc7\uff09\" title=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08\u7814\u7a76\u4e0b\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: 15px;\">&nbsp;\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u60a3\u8005\u7684\u6cbb\u7597\u65b9\u9762\uff0c2018\u5e74\u4ee5\u6765\u5df2\u7ecf\u5f00\u5c55\u4e86\u4e24\u9879\u4e34\u5e8a\u8bd5\u9a8c\u30021\u6708\u4efd\uff0c\u81f4\u529b\u4e8e\u6839\u6cbbDMD\u7684\u975e\u8425\u5229\u7ec4\u7ec7Parent Project Muscular Dystrophy\uff08PPMD\uff09\u5ba3\u5e03\u9996\u4f8bDMD\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff08microdystrophin\uff09\u57fa\u56e0\u7597\u6cd5\u6b63\u5f0f\u542f\u52a8\uff0c\u8be5\u8bd5\u9a8c\u90e8\u5206\u7531PPMD\u57282017\u5e74\u521d\u63d0\u4f9b\u7684220\u4e07\u7f8e\u5143\u8d44\u91d1\u8d44\u52a9\uff0c\u662f\u8be5\u7ec4\u7ec7\u201c\u57fa\u56e0\u7597\u6cd5\u884c\u52a8\u201d\u7684\u4e00\u90e8\u5206\u30024\u6708\u4efd\uff0c\u8f89\u745e\u516c\u53f8\u5ba3\u5e03\u5df2\u7ecf\u542f\u52a8\u7528\u201c\u8ff7\u4f60\u201d\u6297\u808c\u840e\u7f29\u86cb\u767d\uff08mini-dystrophin\uff09\u57fa\u56e0\u7597\u6cd5PF-06939926\u6cbb\u7597DMD\u60a3\u8005\u76841b\u671f\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u9996\u4f4d\u60a3\u8005\u4e8e3\u670822\u65e5\u63a5\u53d7\u4e86\u6cbb\u7597\uff0c\u9884\u8ba1\u8be5\u7814\u7a76\u7684\u65e9\u671f\u6570\u636e\u5c06\u4e8e2019\u5e74\u4e0a\u534a\u5e74\u5b8c\u6210\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;\">3\u670810\u65e5\uff0c Voyager Therapeutics\u516c\u5e03\u4e86\u6b63\u5728\u8fdb\u884c\u7684VY-AADC 1b\u671f\u4e34\u5e8a\u8bd5\u9a8c\u5728\u665a\u671f\u5e15\u91d1\u68ee\u75c5\u4e2d\u7684\u957f\u671f\u6570\u636e\u3002\u5728\u665a\u671f\u5e15\u91d1\u68ee\u75c5\u4e2d\uff0c\u9ed1\u8d28\u4e2d\u7684\u795e\u7ecf\u5143\u9000\u5316\uff0c\u58f3\u6838\u4e2d\u7684AADC\u9176\u4e5f\u663e\u8457\u51cf\u5c11\uff0c\u9650\u5236\u4e86\u5927\u8111\u5c06\u5de6\u65cb\u591a\u5df4\u8f6c\u5316\u4e3a\u591a\u5df4\u80fa\u7684\u80fd\u529b\u3002VY-AADC\u7531\u817a\u76f8\u5173\u75c5\u6bd2-2\u8863\u58f3\u548c\u542f\u52a8AADC\u57fa\u56e0\u8868\u8fbe\u7684\u5de8\u7ec6\u80de\u75c5\u6bd2\u542f\u52a8\u5b50\u7ec4\u6210\uff0c\u53ef\u4ee5\u5c06AADC\u57fa\u56e0\u76f4\u63a5\u9012\u9001\u81f3\u591a\u5df4\u80fa\u53d7\u4f53\u6240\u5728\u7684\u58f3\u6838\u795e\u7ecf\u5143\u4e2d\u3002\u5728\u4e00\u6b21\u6027\u65bd\u7528\u57fa\u56e0\u7597\u6cd5\u540e\uff0c\u60a3\u8005\u7684\u591a\u4e2a\u8fd0\u52a8\u529f\u80fd\u6d4b\u91cf\u90fd\u5f97\u5230\u6301\u7eed\u3001\u5242\u91cf\u4f9d\u8d56\u548c\u65f6\u95f4\u4f9d\u8d56\u7684\u6539\u5584\u3002<\/span><\/p>\n<p><img class=\"\"       style=\"\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2018\/07\/beepress1-1532683923.jpeg\" alt=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08\u7814\u7a76\u4e0b\u7bc7\uff09\" title=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08\u7814\u7a76\u4e0b\u7bc7\uff09\"  \/><\/p>\n<p><br  \/><\/p>\n<p style=\"text-align: justify;\"><strong><span style=\"color: rgb(0, 128, 255);font-size: 16px;\">\u8131\u9776\u6548\u5e94\u3001\u7ed9\u836f\u9014\u5f84\u3001\u4f53\u5185\u91cd\u7f16\u7a0b<\/span><\/strong><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">3\u6708\u4efd\uff0cAlnylam Pharmaceuticals\u516c\u53f8\u7684\u7814\u7a76\u4eba\u5458\u5bf9N-\u4e59\u9170\u534a\u4e73\u7cd6 (GalNAc) \u7f00\u5408\u7684RNAi 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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\u6708\u4efd\uff0cAlnylam\u516c\u53f8\u53c8\u53d6\u5f97\u53e6\u4e00\u9879\u7a81\u7834\u3002\u8be5\u516c\u53f8\u5728\u5927\u9f20\u4e2d\u8fdb\u884c\u7684\u7814\u7a76\u5df2\u80fd\u5c06\u65b0\u578b\u5c0f\u5e72\u6270RNA\uff08siRNA\uff09\u7ed3\u5408\u7269\u6210\u529f\u9012\u9001\u81f3\u4e2d\u67a2\u795e\u7ecf\u7cfb\u7edf\u4e2d\uff0c\u5355\u6b21\u9798\u5185\u6ce8\u5c04\uff08intrathecal 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