{"id":29368,"date":"2018-08-04T01:00:00","date_gmt":"2018-08-09T04:01:00","guid":{"rendered":"http:\/\/www.05921.net\/index.php\/2018\/08\/09\/%e9%80%9f%e9%80%92-%e7%bd%97%e6%b0%8f%e4%ba%a8%e5%bb%b7%e9%a1%bf%e7%97%85%e6%96%b0%e7%96%97%e6%b3%95%e8%8e%b7%e4%bc%98%e5%85%88%e8%8d%af%e5%93%81%e8%b5%84%e6%a0%bc\/"},"modified":"2018-08-09T12:01:03","modified_gmt":"2018-08-09T04:01:03","slug":"%e9%80%9f%e9%80%92-%e7%bd%97%e6%b0%8f%e4%ba%a8%e5%bb%b7%e9%a1%bf%e7%97%85%e6%96%b0%e7%96%97%e6%b3%95%e8%8e%b7%e4%bc%98%e5%85%88%e8%8d%af%e5%93%81%e8%b5%84%e6%a0%bc","status":"publish","type":"post","link":"https:\/\/www.05921.net\/index.php\/29368\/","title":{"rendered":"\u901f\u9012 | \u7f57\u6c0f\u4ea8\u5ef7\u987f\u75c5\u65b0\u7597\u6cd5\u83b7\u4f18\u5148\u836f\u54c1\u8d44\u683c"},"content":{"rendered":"
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\"\u901f\u9012<\/p>\n


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HD\u662f\u4e00\u79cd\u7f55\u89c1\u7684\u9057\u4f20\u6027\u3001\u8fdb\u884c\u6027\u75be\u75c5\u3002HD\u7684\u75c5\u56e0\u662f\u7531\u4e8e\u7f16\u7801\u4ea8\u5ef7\u987f\u86cb\u767d\uff08huntington protein\uff0cHTT\uff09\u7684\u57fa\u56e0\u51fa\u73b0\u4e09\u6838\u82f7\u9178\u5e8f\u5217CAG\u91cd\u590d\u8d85\u8fc736\u6b21\uff0c\u4f7fHTT\u4ea7\u751f\u6bd2\u6027\uff0c\u5e76\u4e14\u9010\u6e10\u635f\u5bb3\u5927\u8111\u4e2d\u7684\u795e\u7ecf\u5143\uff0c\u4ee5\u81f4\u4e8e\u4e25\u91cd\u5f71\u54cd\u60a3\u8005\u7684\u65e5\u5e38\u751f\u6d3b\u529f\u80fd\uff0c\u5982\u884c\u52a8\u80fd\u529b\u548c\u601d\u7ef4\u80fd\u529b\uff0c\u7ed9\u60a3\u8005\u751f\u6d3b\u5e26\u6765\u6bc1\u706d\u6027\u7684\u51b2\u51fb\u3002\u968f\u7740\u75be\u75c5\u7684\u8fdb\u5c55\uff0cHD\u60a3\u8005\u53ef\u80fd\u4f1a\u51fa\u73b0\u6027\u683c\u6539\u53d8\uff0c\u884c\u8d70\u548c\u541e\u54bd\u56f0\u96be\uff0c\u4ee5\u53ca\u4e25\u91cd\u7684\u8ba4\u77e5\u969c\u788d\u3002HD\u60a3\u8005\u5728\u8fd0\u52a8\u673a\u80fd\u53d1\u751f\u969c\u788d\u540e\u7684\u751f\u5b58\u671f\u5927\u7ea6\u4e3a10\u523020\u5e74\u3002HD\u7684\u9057\u4f20\u6027\u610f\u5473\u7740\u5176\u5bf9\u6574\u4e2a\u60a3\u8005\u5bb6\u5ead\u5f71\u54cd\u6df1\u8fdc\u3002HD\u662f\u53d1\u8fbe\u56fd\u5bb6\u4e2d\u6700\u5e38\u89c1\u7684\u5355\u57fa\u56e0\u795e\u7ecf\u7cfb\u7edf\u75be\u75c5\uff0c\u5168\u7403\u60a3\u75c5\u7387\u7ea6\u4e3a\u6bcf10\u4e07\u4eba\u4e2d3-10\u4f8b<\/strong>\u3002\u5728\u7f8e\u56fd\u6709\u5927\u7ea63\u4e07\u540dHD\u60a3\u8005\uff0c\u670920\u4e07\u4eba\u6709HD\u9057\u4f20\u60a3\u75c5\u98ce\u9669\u3002\u76ee\u524d\uff0c\u5373\u6ca1\u6709\u5df2\u77e5\u7684HD\u7597\u6cd5\uff0c\u4e5f\u6ca1\u6709\u9488\u5bf9\u6f5c\u5728\u75c5\u56e0\u7684\u6279\u51c6\u7597\u6cd5\u3002<\/span><\/p>\n


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\"\u901f\u9012<\/p>\n

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RG6042\u662f\u7b2c\u4e8c\u4ee3\u4fee\u9970\u53cd\u4e49\u5be1\u6838\u82f7\u9178\uff08ASO\uff09\uff0c\u901a\u8fc7\u9776\u5411\u4ebaHTT mRNA\u6765\u964d\u4f4emHTT\u86cb\u767d\u7684\u5408\u6210\u548c\u8868\u8fbe\u6c34\u5e73\u3002<\/span><\/strong>mHTT\u88ab\u8ba4\u4e3a\u662fHD\u4e3b\u8981\u81f4\u75c5\u75c5\u56e0\uff0c\u8be5\u6bd2\u6027\u86cb\u767d\u8d28\u5728\u5927\u8111\u7684\u795e\u7ecf\u5143\u4e2d\u4ea7\u751f\u5927\u91cf\u5e9f\u7269\uff0c\u6740\u6b7b\u795e\u7ecf\u5143\u7ec6\u80de\uff0c\u5bfc\u81f4\u4e25\u91cd\u7684\u795e\u7ecf\u9000\u884c\u6027\u75be\u75c5\u3002RG6042\u662f\u76ee\u524d\u4e34\u5e8a\u5f00\u53d1\u4e2d\uff0c\u7528\u4e8e\u9776\u5411mHTT\u7684\u5148\u8fdb\u5316\u5408\u7269\uff0c\u6709\u53ef\u80fd\u51cf\u7f13\u6216\u963b\u6b62\u6240\u6709HD\u60a3\u8005\u7684\u75be\u75c5\u8fdb\u5c55\u3002RG6042\u662f\u7f57\u6c0f\u548cIonis\u4e4b\u95f4\u5168\u9762\u6df1\u5165\u7684\u836f\u7269\u7b5b\u9009\u8ba1\u5212\u7684\u6210\u679c\uff0c\u8be5\u8ba1\u5212\u4e13\u6ce8\u4e8e\u4f18\u5316\u9776\u5411\u4ebaHTT mRNA\u7684ASO\u7684\u6709\u6548\u6027\uff0c\u7279\u5f02\u6027\u548c\u8010\u53d7\u6027\u3002<\/span><\/p>\n

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\u6309\u8ba1\u5212\uff0c\u7f57\u6c0f\u5c06\u5f00\u5c55\u5173\u952e3\u671f\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u65e8\u5728\u8bc4\u4f30\u5728\u8f83\u5927\u60a3\u8005\u7fa4\u4f53\u4e2d\uff0cRG6042\u7684\u5b89\u5168\u6027\u548c\u51cf\u7f13\u6210\u4ebaHD\u7684\u8fdb\u5c55\u7684\u6709\u6548\u6027\u3002\u6211\u4eec\u671f\u5f85\u7740RG6042\u80fd\u65e9\u65e5\u9020\u798fHD\u60a3\u8005\u3002<\/span><\/p>\n


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\u53c2\u8003\u8d44\u6599\uff1a<\/span><\/p>\n

[1] PRIME designation granted by European Medicines Agency for RG6042 for treatment of Huntington’s disease<\/span><\/p>\n

[2] Roche gets some regulatory love for its breakthrough quest on a PhIII Huntington\u2019s drug<\/span><\/p>\n

[3] New Data from IONIS-HTT Rx Phase 1\/2 Study Demonstrates Correlation Between Reduction of Disease-causing Protein and Improvement in Clinical Measures of Huntington’s Disease<\/span><\/p>\n

[4] \u4ea8\u5ef7\u987f\u821e\u8e48\u75c7\u65b0\u836f\u53ef\u6e05\u9664\u9ad8\u8fbe60%\u7684\u81f4\u75c5\u86cb\u767d<\/span><\/p>\n


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\"\u901f\u9012<\/p>\n

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