{"id":49175,"date":"2016-02-23T00:00:00","date_gmt":"2016-02-23T00:00:00","guid":{"rendered":"https:\/\/www.cpr.cuhk.edu.hk\/press\/cuhk-inter-disciplinary-research-team-finds-novel-drug-candidate-for-rare-neurological-diseases\/"},"modified":"2016-02-23T00:00:00","modified_gmt":"2016-02-23T00:00:00","slug":"cuhk-inter-disciplinary-research-team-finds-novel-drug-candidate-for-rare-neurological-diseases","status":"publish","type":"press","link":"https:\/\/www.cpr.cuhk.edu.hk\/sc\/press\/cuhk-inter-disciplinary-research-team-finds-novel-drug-candidate-for-rare-neurological-diseases\/","title":{"rendered":"\u4e2d\u5927\u8de8\u5b66\u79d1\u7814\u7a76\u56e2\u961f\u4e3a\u7f55\u89c1\u795e\u7ecf\u7cfb\u7edf\u75be\u75c5\u7814\u53d1\u65b0\u836f\u7269"},"content":{"rendered":"","protected":false},"featured_media":0,"template":"","tags":[],"class_list":["post-49175","press","type-press","status-publish","hentry"],"acf":{"title_alt":"","sub_title_alt":"","press_date":"20160223","keep":false,"details":"<p>\u9999\u6e2f\u4e2d\u6587\u5927\u5b66\uff08\u4e2d\u5927\uff09\u751f\u547d\u79d1\u5b66\u5b66\u9662\u9648\u6d69\u7136\u6559\u6388\u53ca\u5176\u56e2\u961f\u591a\u5e74\u6765\u4e00\u76f4\u81f4\u529b\u7814\u7a76\u4e0e\u795e\u7ecf\u7cfb\u7edf\u6709\u5173\u7684\u7f55\u89c1\u75c5\u4e4b\u81f4\u75c5\u539f\u56e0\u53ca\u6cbb\u7597\u65b9\u6848\uff0c\u6700\u8fd1\u53d1\u660e\u4e86\u4e00\u79cd\u540d\u4e3aP3\u7684\u591a\u80bd\u5206\u5b50\uff08\u7531\u86cb\u767d\u8d28\u4e2d13\u4e2a\u6c28\u57fa\u9178\u80bd\u5408\u6210\uff09\uff0c\u80fd\u6709\u6548\u6291\u5236\u795e\u7ecf\u7ec6\u80de\u9000\u5316\uff0c\u5e76\u5df2\u53d6\u5f97\u7f8e\u56fd\u4e13\u5229\u53ca\u5546\u6807\u5c40\u7684\u4e13\u5229\uff0c\u53ef\u671b\u6210\u4e3a\u65b0\u836f\u7269\uff0c\u4e3a\u7f55\u89c1\u795e\u7ecf\u7cfb\u7edf\u75c5\u60a3\u8005\u5e26\u6765\u4ee4\u4eba\u632f\u594b\u7684\u6d88\u606f\u3002\u6709\u5173\u7814\u7a76\u5df2\u520a\u8f7d\u4e8e\u8457\u540d\u75c5\u7406\u5b66\u7814\u7a76\u671f\u520a<em>Disease Models &amp; Mechanisms<\/em>\u30022\u670829\u65e5\u4e3a\u56fd\u9645\u7f55\u89c1\u75c5\u65e5\uff0c\u5e0c\u671b\u5524\u8d77\u5168\u7403\u5bf9\u7f55\u89c1\u75c5\u7684\u5173\u6ce8\u3002&nbsp;<\/p><p><strong>\u7f55\u89c1\u795e\u7ecf\u7cfb\u7edf\u75be\u75c5\u73b0\u65f6\u672a\u53ef\u6cbb\u6108<\/strong><\/p><p>\u9648\u6d69\u7136\u6559\u6388\u53ca\u5176\u56e2\u961f\u7684\u7814\u7a76\u6240\u9488\u5bf9\u7684\u75be\u75c5\uff0c\u662f\u4e00\u7ec4\u540d\u4e3apolyQ\uff08\u591a\u805a\u8c37\u6c28\u9170\u80fa\u75be\u75c5\uff0cPolyglutamine Diseases\uff09\u7684\u795e\u7ecf\u7cfb\u7edf\u75be\u75c5\uff0c\u5305\u62ec\u516d\u79cd\u5c0f\u8111\u840e\u7f29\u75c7\u53ca\u4ea8\u5ef7\u987f\u75c7\u7b49\u7f55\u89c1\u75be\u75c5\u3002\u4ea8\u5ef7\u987f\u75c7\u53c8\u79f0\u4ea8\u5ef7\u987f\u821e\u8e48\u75c7\uff0c\u60a3\u8005\u7684\u8111\u90e8\u529f\u80fd\u9000\u5316\u4ee5\u81f4\u624b\u8db3\u548c\u9762\u4e0a\u7684\u808c\u8089\u4f1a\u4e0d\u89c4\u5219\u3001\u4e0d\u7531\u81ea\u4e3b\u5730\u62bd\u52a8\uff0c\u4ea6\u4f1a\u51fa\u73b0\u8ba4\u77e5\u969c\u788d\u53ca\u7cbe\u795e\u6df7\u4e71\u7b49\u75c7\u72b6\u3002\u5c0f\u8111\u840e\u7f29\u75c7\u53c8\u79f0\u810a\u9ad3\u5c0f\u8111\u6027\u5171\u6d4e\u5931\u8c03\uff0c\u662f\u4e00\u79cd\u5bfc\u81f4\u5c0f\u8111\u7ec6\u80de\u9010\u6b65\u6b7b\u4ea1\u7684\u6162\u6027\u9057\u4f20\u75be\u75c5\uff0c\u60a3\u8005\u4f1a\u8d70\u8def\u65f6\u6b65\u5c65\u4e0d\u7a33\uff0c\u80a2\u4f53\u6447\u6643\uff1b\u8bf4\u8bdd\u65f6\u53d1\u97f3\u542b\u7cca\u4e0d\u6e05\uff0c\u65e0\u6cd5\u63a7\u5236\u97f3\u8c03\uff0c\u751a\u81f3\u65e0\u6cd5\u8bed\u8a00\u3002\u603b\u62ec\u800c\u8a00\uff0cpolyQ\u60a3\u8005\uff08\u5305\u62ec\u4ea8\u5ef7\u987f\u75c7\u53ca\u5c0f\u8111\u840e\u7f29\u75c7\uff09\u6700\u7ec8\u4f1a\u5b8c\u5168\u5931\u53bb\u8eab\u4f53\u7684\u534f\u8c03\u80fd\u529b\uff0c\u4e0d\u80fd\u8fdb\u884c\u65e5\u5e38\u6d3b\u52a8\u3002\u5230\u76ee\u524d\u4e3a\u6b62\uff0cpolyQ\u4ecd\u662f\u65e0\u6cd5\u6cbb\u6108\u7684\u75be\u75c5\u3002&nbsp;<\/p><p><strong>\u8de8\u5b66\u79d1\u56e2\u961f\u6210\u529f\u7814\u53d1\u6291\u5236\u795e\u7ecf\u7ec6\u80de\u9000\u5316\u7684\u5408\u6210\u591a\u80bd\u5206\u5b50<\/strong>&nbsp;<\/p><p>2012\u5e74\uff0c\u9648\u6559\u6388\u53ca\u5176\u7814\u7a76\u56e2\u961f\u63ed\u793a\u4e86\u5f15\u53d1\u795e\u7ecf\u7ec6\u80de\u51cb\u4ea1\u7684\u8fc7\u7a0b\uff0c\u4e3a\u65e0\u6cd5\u6cbb\u6108\u7684\u795e\u7ecf\u7cfb\u7edf\u75be\u75c5\u5f00\u62d3\u65b0\u7684\u6cbb\u7597\u65b9\u5411\u3002\u4ed6\u4eec\u53d1\u73b0\u7ec6\u80de\u4e2d\u5177\u6bd2\u6027\u7684RNA\u5206\u5b50\u4f1a\u5e72\u6270\u86cb\u767d\u8d28\u300c\u6838\u4ec1\u7d20\u300d\u7684\u529f\u80fd\uff0c\u963b\u6b62\u300c\u6838\u4ec1\u7d20\u300d\u5728\u7ec6\u80de\u6838\u5185\u5236\u9020\u6838\u7cd6\u4f53\uff08\u6240\u6709\u7ec6\u80de\u5747\u4f9d\u8d56\u6838\u7cd6\u4f53\u7684\u6b63\u5e38\u8fd0\u4f5c\u6765\u7ef4\u6301\u751f\u547d\uff09\u3002\u5728\u4e2d\u5927\u3001\u9999\u6e2f\u7814\u7a76\u8d44\u52a9\u5c40\u3001\u98df\u7269\u53ca\u536b\u751f\u5c40\u533b\u7597\u536b\u751f\u7814\u7a76\u57fa\u91d1\u3001\u5468\u5927\u798f\u6148\u5584\u57fa\u91d1\u53ca\u9999\u6e2f\u5c0f\u8111\u840e\u7f29\u75c7\u534f\u4f1a\u7684\u652f\u6301\u53ca\u8d5e\u52a9\u4e0b\uff0c\u9648\u6559\u6388\u7684\u56e2\u961f\u7ecf\u8fc7\u56db\u5e74\u7684\u52aa\u529b\u53ca\u591a\u9879\u4e25\u683c\u7684\u6d4b\u8bd5\uff0c\u53d1\u660e\u4e86P3\u591a\u80bd\u5206\u5b50\uff0c\u80fd\u6709\u6548\u6291\u5236\u795e\u7ecf\u7ec6\u80de\u9000\u5316\u3002\u5316\u5b66\u5408\u6210\u7684P3\u5305\u542b\u4e86\u5929\u7136\u6838\u4ec1\u86cb\u767d\u4e2d\u768413\u4e2a\u6c28\u57fa\u9178\u80bd\uff0c\u80fd\u76f4\u63a5\u4e0e\u81f4\u75c5\u7684RNA\u4ea7\u751f\u4f5c\u7528\uff0c\u538b\u5236\u5176\u6bd2\u6027\uff0c\u5176\u6cbb\u7597\u6548\u7528\u5df2\u4e8e\u7ec6\u80de\u53ca\u52a8\u7269\u75be\u75c5\u6a21\u578b\u4e2d\u8bc1\u5b9e\uff0c\u4ea6\u5df2\u4e8e\u75c5\u4eba\u7ec6\u80de\u8fdb\u884c\u6d4b\u8bd5\u3002&nbsp;<\/p><p>\u9648\u6d69\u7136\u6559\u6388\u8868\u793a\uff1a\u300c2012\u5e74\uff0c\u6211\u4eec\u53d1\u73b0\u300e\u6838\u4ec1\u7d20\u300f\u8fc7\u8868\u8fbe(overexpression)\u80fd\u6709\u6548\u538b\u5236\u6bd2\u6027RNA\u5bf9\u7ec6\u80de\u6240\u9020\u6210\u7684\u4f24\u5bb3\u540e\uff0c\u6211\u79ef\u6781\u4e3b\u52a8\u5730\u9080\u8bf7\u6765\u81ea\u4e0d\u540c\u5b66\u79d1\u9886\u57df\uff0c\u5305\u62ec\u7ed3\u6784\u751f\u7269\u5b66\u3001\u591a\u80bd\u5de5\u7a0b\u6280\u672f\u3001\u6750\u6599\u79d1\u5b66\u53ca\u836f\u7406\u5b66\u7684\u4e13\u5bb6\uff0c\u7ec4\u6210\u8de8\u5b66\u79d1\u7684\u7814\u7a76\u56e2\u961f\uff0c\u5e0c\u671b\u5171\u540c\u4e3a\u7f55\u89c1\u795e\u7ecf\u7cfb\u7edf\u75be\u75c5\u7814\u53d1\u65b0\u836f\u3002\u7ecf\u8fc7\u56db\u5e74\u4e25\u683c\u6d4b\u8bd5\uff0c\u6211\u4eec\u7ec8\u4e8e\u6210\u529f\u5408\u6210P3\u591a\u80bd\u6291\u5236\u5206\u5b50\uff0c\u662f\u9996\u4e2a\u80fd\u6709\u6548\u538b\u5236RNA\u6bd2\u6027\u7684\u591a\u80bd\u5206\u5b50\u3002\u6211\u671f\u671bP3\u53ef\u4ee5\u8fdb\u4e00\u6b65\u53d1\u5c55\u6210\u4e3a\u6b63\u5f0f\u836f\u7269\uff0c\u4e3apolyQ\u60a3\u8005\u5e26\u6765\u65b0\u5e0c\u671b\u3002\u300d&nbsp;<\/p><p><strong>P3<\/strong><strong>\u79d1\u6280\u6210\u4e3a<\/strong><strong>\u672a\u6765\u6cbb\u7597\u65b9\u5411<\/strong>&nbsp;<\/p><p>\u9648\u6559\u6388\u73b0\u65f6\u5df2\u5c31polyQ\u7814\u7a76\u6210\u7acb\u4e86\u8de8\u5730\u57df\u3001\u8de8\u5b66\u79d1\u7684\u7814\u7a76\u5408\u4f5c\u7f51\u7edc\uff0c\u5176\u5408\u4f5c\u4f19\u4f34\u8986\u76d6\u81f3\u4e39\u9ea6\u3001\u6cd5\u56fd\u3001\u610f\u5927\u5229\u3001\u82f1\u56fd\u53ca\u7f8e\u56fd\u7b49\u5730\u57df\u3002\u9648\u6559\u6388\u4e0e\u4e39\u9ea6\u54e5\u672c\u54c8\u6839\u5927\u5b66\u7684\u591a\u80bd\u7814\u7a76\u4e13\u5bb6Knud J. 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