{"id":102222,"date":"2019-04-28T00:00:50","date_gmt":"2019-05-17T08:42:40","guid":{"rendered":"http:\/\/www.05921.net\/index.php\/102222\/"},"modified":"2019-05-17T16:42:42","modified_gmt":"2019-05-17T08:42:42","slug":"ce990b1f9d","status":"publish","type":"post","link":"https:\/\/www.05921.net\/index.php\/102222\/","title":{"rendered":"\u91cd\u78c5\uff01\u5168\u7403\u9996\u4e2a\u6cbb\u7597\u810a\u9ad3\u6027\u808c\u840e\u7f29\u75c7\u836f\u7269\u4e2d\u56fd\u4e0a\u5e02\uff01"},"content":{"rendered":"
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\u8bf8\u591a\u6311\u6218\u963b\u788d\u6211\u56fdSMA\u8bca\u6cbb\u53d1\u5c55<\/strong><\/span><\/p>\n<\/section>\n<\/section>\n<\/section>\n

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\u957f\u671f\u4ee5\u6765\uff0cSMA\u5728\u4e2d\u56fd\u7684\u8bca\u6cbb\u5b58\u5728\u8bf8\u591a\u6311\u6218\uff0c\u5305\u62ec\u75be\u75c5\u77e5\u6653\u7a0b\u5ea6\u4f4e\uff0c\u63a5\u53d7\u795e\u7ecf\u808c\u8089\u75c5\u4e13\u5bb6\u8bca\u6cbb\u548c\u57fa\u56e0\u68c0\u6d4b\u7684\u6e20\u9053\u5c11\uff0c\u7f3a\u4e4f\u6709\u6548\u6cbb\u7597\u836f\u7269\uff0c\u60a3\u8005\u7f3a\u4e4f\u89c4\u8303\u5316\u957f\u671f\u968f\u8bbf\u3001\u7ba1\u7406\u7b49\u3002\u8fd9\u4e9b\u6311\u6218\u4e25\u91cd\u963b\u788d\u4e86\u6211\u56fdSMA\u8bca\u7597\u7684\u53d1\u5c55\uff0c\u4e5f\u8ba9SMA\u60a3\u8005\u53ca\u5bb6\u5ead\u9762\u4e34\u56f0\u5883\u3002<\/span><\/p>\n


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\u5728\u8bfa\u897f\u90a3\u751f\u94a0\u6ce8\u5c04\u6db2\u6279\u51c6\u4e4b\u524d\uff0cSMA\u65e0\u6709\u6548\u6cbb\u7597\u624b\u6bb5\uff0c\u75be\u75c5\u7ba1\u7406\u4ec5\u9650\u4e8e\u547c\u5438\u652f\u6301\u3001\u8425\u517b\u652f\u6301\u3001\u9aa8\u79d1\u77eb\u5f62\u7b49\u8f85\u52a9\u6cbb\u7597\u65b9\u6cd5\u30022018\u5e745\u6708\uff0cSMA\u88ab\u5217\u5165\u4e2d\u56fd\u56fd\u5bb6\u536b\u751f\u5065\u5eb7\u59d4\u5458\u4f1a\u7b49\u90e8\u95e8\u8054\u5408\u5236\u5b9a\u7684\u300a\u7b2c\u4e00\u6279\u7f55\u89c1\u75c5\u76ee\u5f55\u300b\uff0c\u8be5\u76ee\u5f55\u7684\u63a8\u51fa\u65e8\u5728\u652f\u6301\u7f55\u89c1\u75c5\u7684\u8bca\u65ad\u548c\u6cbb\u7597\u3002<\/span><\/p>\n


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2018\u5e747\u6708\uff0c\u56fd\u5bb6\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\u5ba3\u5e03\u5bf9\u5728\u76d1\u7ba1\u4f53\u7cfb\u5b8c\u5584\u7684\u56fd\u5bb6\u548c\u5730\u533a\u62e5\u6709\u4e34\u5e8a\u7814\u7a76\u6570\u636e\u7684\u521b\u65b0\u836f\u7269\u53ef\u5b9e\u65bd\u4f18\u5148\u5ba1\u8bc4\u5ba1\u6279\u7a0b\u5e8f\u30022018\u5e749\u6708\uff0c\u8bfa\u897f\u90a3\u751f\u94a0\u6ce8\u5c04\u6db2\u4f5c\u4e3a\u5df2\u5728\u5883\u5916\u4e0a\u5e02\u4e14\u4e34\u5e8a\u6025\u9700\u7684\u7f55\u89c1\u75c5\u6cbb\u7597\u65b0\u836f\u88ab\u56fd\u5bb6\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\u7eb3\u5165\u4f18\u5148\u5ba1\u8bc4\u5ba1\u6279\u7a0b\u5e8f\u3002<\/span><\/p>\n


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2019\u5e742\u6708\uff0c\u56fd\u5bb6\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\u6b63\u5f0f\u6279\u51c6\u8bfa\u897f\u90a3\u751f\u94a0\u6ce8\u5c04\u6db2\u7528\u4e8e\u6cbb\u75975q SMA\u30025q SMA\u662f\u8be5\u75be\u75c5\u6700\u5e38\u89c1\u7684\u5f62\u5f0f\uff0c\u7ea6\u5360\u6240\u6709SMA\u75c5\u4f8b\u768495%\u3002\u4eca\u5929\uff0c\u4f5c\u4e3a\u5168\u7403\u9996\u4e2a\u4e14\u76ee\u524d\u552f\u4e00\u4e00\u4e2a\u6cbb\u7597SMA\u7684\u836f\u7269\uff0c\u8bfa\u897f\u90a3\u751f\u94a0\u6ce8\u5c04\u6db2\u7684\u6b63\u5f0f\u4e0a\u5e02\uff0c\u6807\u5fd7\u7740SMA\u5728\u4e2d\u56fd\u4e0d\u518d\u662f\u4e00\u79cd\u201c\u65e0\u836f\u53ef\u533b\u201d\u7684\u7f55\u89c1\u75c5\u3002<\/span><\/p>\n


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\u5728\u836f\u7269\u4e0a\u5e02\u7684\u540c\u65f6\uff0c\u4e3a\u63d0\u9ad8SMA\u7684\u89c4\u8303\u5316\u8bca\u6cbb\u6c34\u5e73\uff0c\u4e2d\u56fd\u513f\u79d1\u4e13\u5bb6\u3001\u513f\u7ae5\u795e\u7ecf\u75c5\u5b66\u4e13\u5bb6\u3001\u6210\u4eba\u795e\u7ecf\u79d1\u4e13\u5bb6\u5171\u540c\u547c\u5401\uff0c\u5021\u5bfc\u6210\u7acb\u201c\u4e2d\u56fdSMA\u8bca\u6cbb\u4e2d\u5fc3\u8054\u76df\u201d\u3002\u8fd9\u4e00\u547c\u5401\u5f97\u5230\u4e86\u5168\u56fd25\u5bb6\u533b\u9662\u7684\u79ef\u6781\u54cd\u5e94\uff0c\u5e76\u6210\u4e3a\u8054\u76df\u7684\u9996\u6279\u6210\u5458\u5355\u4f4d\u3002<\/span><\/p>\n


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\"\u91cd\u78c5\uff01\u5168\u7403\u9996\u4e2a\u6cbb\u7597\u810a\u9ad3\u6027\u808c\u840e\u7f29\u75c7\u836f\u7269\u4e2d\u56fd\u4e0a\u5e02\uff01\"<\/p>\n


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\u5efa\u7acb\u5171\u4ed8\u673a\u5236\u63d0\u9ad8\u836f\u7269\u53ef\u53ca\u6027<\/strong><\/span><\/p>\n<\/section>\n<\/section>\n<\/section>\n

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\u4eca\u5e743\u6708\u56fd\u5bb6\u533b\u4fdd\u5c40\u516c\u5e03\u4e86\u300a2019\u5e74\u56fd\u5bb6\u533b\u4fdd\u836f\u54c1\u76ee\u5f55\u8c03\u6574\u65b9\u6848\uff08\u5f81\u6c42\u610f\u89c1\u7a3f\uff09\u300b\uff0c\u8fd9\u662f\u56fd\u5bb6\u533b\u4fdd\u76ee\u5f55\u8c03\u6574\u7b2c\u4e00\u6b21\u660e\u786e\u63d0\u51fa\u4f18\u5148\u8003\u8651\u7f55\u89c1\u75c5\u3001\u513f\u7ae5\u7528\u836f\uff0c\u5145\u5206\u4f53\u73b0\u4e86\u56fd\u5bb6\u5bf9\u7f55\u89c1\u75c5\u3001\u513f\u7ae5\u7528\u836f\u4fdd\u969c\u7684\u9ad8\u5ea6\u5173\u6ce8\u3002\u6211\u4eec\u8877\u5fc3\u5e0c\u671b\u5404\u7ea7\u653f\u5e9c\u80fd\u9488\u5bf9\u4e34\u5e8a\u7597\u6548\u663e\u8457\u7684SMA\u6cbb\u7597\u836f\u7269\u542f\u52a8\u8c08\u5224\uff0c\u5c06SMA\u836f\u7269\u7eb3\u5165\u56fd\u5bb6\u6216\u5730\u65b9\u533b\u4fdd\u836f\u54c1\u76ee\u5f55\uff0c\u5e2e\u52a9SMA\u60a3\u8005\u5bb6\u5ead\u89e3\u51b3\u6cbb\u7597\u8d1f\u62c5\uff0c\u8ba9\u4fdd\u969c\u7f55\u89c1\u75c5\u60a3\u8005\u7528\u836f\u771f\u6b63\u6210\u4e3a\u73b0\u5b9e\u3002\u201c<\/span><\/p>\n


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\u5173\u4e8e\u8bfa\u897f\u90a3\u751f\u94a0\u6ce8\u5c04\u6db22-5<\/span><\/strong><\/p>\n

\u4f5c\u4e3a\u7b2c\u4e00\u4e2a\u4e5f\u662f\u552f\u4e00\u4e00\u4e2a\u83b7\u6279\u6cbb\u7597SMA\u7684\u836f\u7269\uff0c\u8bfa\u897f\u90a3\u751f\u94a0\u6ce8\u5c04\u6db2\u76ee\u524d\u5df2\u572840\u591a\u4e2a\u56fd\u5bb6\u548c\u5730\u533a\u83b7\u6279\u3002\u622a\u81f32018\u5e7412\u670831\u65e5\uff0c\u5168\u7403\u5df2\u6709\u8d85\u8fc76600\u540d\u5a74\u513f\u3001\u513f\u7ae5\u6216\u6210\u4ebaSMA\u60a3\u8005\u63a5\u53d7\u4e86\u8bfa\u897f\u90a3\u751f\u94a0\u6ce8\u5c04\u6db2\u7684\u6cbb\u7597\u3002<\/span><\/p>\n


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\u6e24\u5065\u4ece\u53cd\u4e49\u6280\u672f\u7684\u9886\u5148\u4f01\u4e1aIonis Pharmaceuticals\u83b7\u5f97\u8bfa\u897f\u90a3\u751f\u94a0\u6ce8\u5c04\u6db2\u5728\u5168\u7403\u7684\u5f00\u53d1\u3001\u751f\u4ea7\u548c\u5546\u4e1a\u63a8\u5e7f\u6743\u5229\u3002\u6e24\u5065\u548cIonis\u5171\u540c\u5f00\u5c55\u4e86SMA\u9886\u57df\u6700\u5927\u7684\u4e00\u9879\u521b\u65b0\u4e34\u5e8a\u5f00\u53d1\u9879\u76ee\uff0c\u8be5\u9879\u76ee\u4f7f\u8bfa\u897f\u90a3\u751f\u94a0\u6ce8\u5c04\u6db2\u4ece2011\u5e74\u7684\u9996\u6b21\u4eba\u4f53\u6ce8\u5c04\u5230\u4e94\u5e74\u5185\u83b7\u5f97\u9996\u6b21\u6279\u51c6\u3002<\/span><\/p>\n


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\u5173\u4e8e\u6e24\u5065<\/span><\/strong><\/p>\n

\u5728\u6e24\u5065\uff0c\u6211\u4eec\u7684\u4f7f\u547d\u6e05\u6670\u660e\u786e\uff1a\u6211\u4eec\u662f\u795e\u7ecf\u79d1\u5b66\u9886\u57df\u7684\u5148\u950b\u3002\u6e24\u5065\u4e3a\u5168\u7403\u7f79\u60a3\u4e25\u91cd\u795e\u7ecf\u548c\u795e\u7ecf\u9000\u884c\u6027\u75be\u75c5\u7684\u60a3\u8005\u63a2\u5bfb\u3001\u7814\u53d1\u548c\u63d0\u4f9b\u521b\u65b0\u7597\u6cd5\u3002\u6e24\u5065\u662fCharles Weissmann\uff0cHeinz Schaller\uff0cKenneth Murray\u4e0e\u8bfa\u8d1d\u5c14\u5956\u83b7\u5f97\u8005Walter Gilbert\u548cPhillip Sharp\u643a\u624b\u57281978\u5e74\u6210\u7acb\u7684\u5168\u7403\u9996\u6279\u751f\u7269\u6280\u672f\u516c\u53f8\u4e4b\u4e00\u3002\u4eca\u5929\u6e24\u5065\u62e5\u6709\u6cbb\u7597\u591a\u53d1\u6027\u786c\u5316\u7684\u9886\u5148\u836f\u7269\u7ec4\u5408; \u63a8\u51fa\u7b2c\u4e00\u4e2a\u4e5f\u662f\u552f\u4e00\u4e00\u4e2a\u6279\u51c6\u7528\u4e8e\u810a\u9ad3\u6027\u808c\u840e\u7f29\u75c7\u7684\u6cbb\u7597\u836f\u7269\uff1b\u5e76\u4e13\u6ce8\u4e8e\u63a8\u8fdb\u963f\u5c14\u8328\u6d77\u9ed8\u75c5\u548c\u75f4\u5446\u3001\u591a\u53d1\u6027\u786c\u5316\u3001\u795e\u7ecf\u514d\u75ab\u5b66\u3001\u8fd0\u52a8\u969c\u788d\u3001\u795e\u7ecf\u808c\u8089\u75be\u75c5\u3001\u795e\u7ecf\u79d1\u6025\u75c7\u3001\u795e\u7ecf\u8ba4\u77e5\u969c\u788d\u3001\u75bc\u75db\u3001\u773c\u79d1\u9886\u57df\u7684\u79d1\u5b66\u7814\u7a76\u9879\u76ee\u3002\u6e24\u5065\u8fd8\u751f\u4ea7\u548c\u63a8\u5e7f\u5148\u8fdb\u751f\u7269\u5236\u5242\u7684\u751f\u7269\u4eff\u5236\u836f\u3002<\/span><\/p>\n


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\u53c2\u8003\u6587\u732e\uff1a<\/span><\/em><\/p>\n

1. Darras B, Markowitz J, Monani U, De Vivo D. Chapter 8 – Spinal Muscular Atrophies. In: Vivo BTD, ed. Neuromuscular Disorders of Infancy, Childhood, and Adolescence (Second Edition). San Diego: Academic Press; 2015:117-145. <\/span><\/em><\/p>\n

2. Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, Krainer AR. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes Dev. 2010 Aug 1; 24(15):16344-44.<\/span><\/em><\/p>\n

3. Finkel R, Chiriboga C, Vajsar J, et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016;388(10063):3017-3026.<\/span><\/em><\/p>\n

4. Evers MM, Toonen LJ, van Roon-Mom WM. Antisense oligonucleotides in therapy for neurodegenerative disorders. Adv Drug Deliv Rev. 2015;87:90-103.<\/span><\/em><\/p>\n

5. Lunn MR, Wang CH. Spinal muscular atrophy. Lancet. 2008;371(9630):2120-2133.<\/span><\/em><\/p>\n


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