{"id":12491,"date":"2013-01-15T13:22:49","date_gmt":"2013-01-15T05:22:49","guid":{"rendered":"https:\/\/case.ntu.edu.tw\/blog\/?p=12491"},"modified":"2017-08-01T22:06:28","modified_gmt":"2017-08-01T14:06:28","slug":"%e3%80%90%e6%8e%a2%e7%b4%a2%e5%85%ab%e3%80%91%e6%89%ad%e8%bd%89%e5%ae%bf%e5%91%bd%e7%9a%84%e5%9f%ba%e5%9b%a0%e7%b4%b0%e8%83%9e%e6%b2%bb%e7%99%82","status":"publish","type":"post","link":"https:\/\/case.ntu.edu.tw\/blog\/?p=12491","title":{"rendered":"\u3010\u63a2\u7d22\u516b\u3011\u626d\u8f49\u5bbf\u547d\u7684\u57fa\u56e0\u7d30\u80de\u6cbb\u7642"},"content":{"rendered":"<h2 style=\"text-align: center;\"><strong><span style=\"color: #800000;\">\u7b2c\u516b\u8b1b\u2027\u7279\u7a3f<\/span><\/strong><\/h2>\n<p><strong><span style=\"color: #800000;\"><strong><span style=\"color: #333399;\"><strong>\u25a0 \u547d\u904b\u4e26\u4e0d\u662f\u65e9\u5df2\u8a3b\u5b9a\uff0c\u800c\u662f\u59cb\u7d42\u90fd\u638c\u63e1\u5728\u81ea\u5df1\u624b\u88e1\u3002<\/strong><\/span><\/strong><\/span><\/strong><\/p>\n<figure id=\"attachment_12492\" aria-describedby=\"caption-attachment-12492\" style=\"width: 270px\" class=\"wp-caption alignleft\"><a href=\"http:\/\/case.ntu.edu.tw\/blog\/?attachment_id=12492\" rel=\"attachment wp-att-12492\"><img fetchpriority=\"high\" decoding=\"async\" class=\"size-full wp-image-12492\" title=\"200355378-001\" src=\"http:\/\/case.ntu.edu.tw\/blog\/wp-content\/uploads\/2013\/01\/\u7279\u7a3f\u7b2c\u516b\u8b1b\u7279\u7a3f\u5716\u7247.jpg\" alt=\"\" width=\"270\" height=\"406\" srcset=\"https:\/\/case.ntu.edu.tw\/blog\/wp-content\/uploads\/2013\/01\/\u7279\u7a3f\u7b2c\u516b\u8b1b\u7279\u7a3f\u5716\u7247.jpg 338w, https:\/\/case.ntu.edu.tw\/blog\/wp-content\/uploads\/2013\/01\/.jpg-200x300.jpg 200w\" sizes=\"(max-width: 270px) 100vw, 270px\" \/><\/a><figcaption id=\"caption-attachment-12492\" class=\"wp-caption-text\">\u5716\u7247\u4f86\u6e90: Marili Forastieri\u2502Getty Images<\/figcaption><\/figure>\n<p><span style=\"color: #800000;\"><span style=\"color: #000000;\"><strong>\u64b0\u6587<\/strong>\u2502\u5f35\u601d\u9060<\/span><\/span><\/p>\n<p><strong>\u7d30\u80de\u6cbb\u7642<\/strong><\/p>\n<p>\u5168\u5c31\u662f\u4e00\uff0c\u4e00\u5c31\u662f\u5168\uff0c\u4eba\u9ad4\u5404\u90e8\u4f4d\u7d30\u80de\u5343\u8b8a\u842c\u5316\uff0c\u5e7e\u4e4e\u90fd\u662f\u5206\u5316\u81ea\u6700\u521d\u7684\u80da\u80ce\u5e79\u7d30\u80de(Embryonic stem cell\uff1bESCs)\uff0c\u52d5\u7269\u5be6\u9a57\u900f\u904e\u4eba\u5de5\u57f9\u990a\u80da\u80ce\u5e79\u7d30\u80de\uff0c\u8b93\u5e79\u7d30\u80de\u5206\u88c2\u6210\u66f4\u591a\u7684\u500b\u9ad4\uff0c\u9019\u4e9b\u5e79\u7d30\u80de\u518d\u4e0d\u65b7\u5206\u5316\u70ba\u5b8c\u6574\u751f\u7269\u9ad4\u3002<\/p>\n<p>\u5e79\u7d30\u80de\u5ee3\u6cdb\u61c9\u7528\u65bc\u91ab\u5b78\u7d30\u80de\u6cbb\u7642\uff0c\u7d30\u80de\u6cbb\u7642\u7684\u539f\u7406\u5c31\u662f\u900f\u904e\u690d\u5165\u65b0\u7684\u7d30\u80de\uff0c\u66ff\u63db\u5df2\u7d93\u58de\u6b7b\u7684\u7d30\u80de\uff0c\u4f7f\u7d44\u7e54\u6062\u5fa9\u5065\u5eb7\u904b\u4f5c\u3002\u9aa8\u9ad3\u79fb\u690d\u662f\u6700\u65e9\u7684\u7d30\u80de\u6cbb\u7642\u6848\u4f8b\uff0c\u4f46\u9069\u5408\u75c5\u4eba\u79fb\u690d\u7684\u9aa8\u9ad3\uff0c\u5f80\u5f80\u53ef\u9047\u4e0d\u53ef\u6c42\uff0c\u5f8c\u4f86\u7814\u7a76\u767c\u73fe\u81cd\u5e36\u8840\u4e2d\u5bcc\u542b\u9020\u8840\u5e79\u7d30\u80de\uff0c\u79fb\u690d\u9020\u8840\u5e79\u7d30\u80de\u53ef\u4ee5\u5f97\u5230\u8207\u9aa8\u9ad3\u79fb\u690d\u4e0d\u76f8\u4e0a\u4e0b\u7684\u6cbb\u7642\u6548\u679c\uff0c\u800c\u4e14\u53d6\u5f97\u76f8\u5c0d\u5bb9\u6613\u3002\u8207\u9aa8\u9ad3\u79fb\u690d\u540c\u6a23\u7684\u56f0\u64fe\u662f\uff0c\u5408\u9069\u7684\u89d2\u819c\u4e5f\u662f\u53ef\u9047\u4e0d\u53ef\u6c42\uff0c\u4f46\u66f4\u68d8\u624b\u7684\u662f\uff0c\u89d2\u819c\u56b4\u91cd\u71d2\u50b7\u7684\u60a3\u8005\uff0c\u7531\u65bc\u540c\u6642\u50b7\u53ca\u89d2\u819c\u4e0a\u5c64\u7d30\u80de\uff0c\u6062\u5fa9\u8996\u529b\u9664\u4e86\u9032\u884c\u89d2\u819c\u79fb\u690d\u624b\u8853\u5916\uff0c\u9084\u9700\u8981\u914d\u5408\u4fee\u5fa9\u89d2\u819c\u4e0a\u5c64\u7d30\u80de\uff0c\u56e0\u6b64\uff0c\u5f9e\u89d2\u819c\u63d0\u53d6\u90e8\u5206\u89d2\u819c\u7de3\u5e79\u7d30\u80de\uff0c\u52a0\u4ee5\u57f9\u990a\u5f8c\u518d\u79fb\u690d\u5230\u89d2\u819c\uff0c\u4f7f\u53d7\u640d\u7684\u89d2\u819c\u7d44\u7e54\u518d\u751f\uff0c\u75c5\u4eba\u5f97\u4ee5\u91cd\u898b\u5149\u660e\u3002\u5f88\u591a\u4eba\u88ab\u6389\u9aee\u7684\u554f\u984c\u6240\u56f0\u64fe\uff0c\u5c0d\u982d\u76ae\u690d\u5165\u6bdb\u56ca\u795e\u7d93\u810a\u5e79\u7d30\u80de\uff0c\u982d\u9aee\u4e5f\u80fd\u300c\u7a2e\u300d\u51fa\u4f86\uff0c\u56de\u5fa9\u6fc3\u5bc6\u79c0\u9aee\u518d\u4e5f\u4e0d\u662f\u5922\uff0c\u53e6\u5916\uff0c\u5728\u57f9\u990a\u6bdb\u56ca\u5e79\u7d30\u80de\u904e\u7a0b\u4e2d\u8b8a\u5316\u57f9\u990a\u6db2\u6210\u5206\uff0c\u8b93\u5e79\u7d30\u80de\u5206\u5316\u6210\u9ed1\u8272\u7d20\u7d30\u80de\uff0c\u9ed1\u8272\u7d20\u5c0d\u6cbb\u7642\u767d\u6591\u6709\u76f8\u7576\u6210\u6548\uff0c\u7814\u7a76\u6307\u51fa\u5c0d\u75c5\u8b8a\u76ae\u819a\u90e8\u4f4d\u76f4\u63a5\u6ce8\u5c04\u6bdb\u56ca\u5e79\u7d30\u80de\u5c31\u80fd\u6cbb\u7652\u767d\u6591\u3002<\/p>\n<p>\u4eba\u985e\u80da\u80ce\u5e79\u7d30\u80de\u53ef\u4ee5\u5206\u5316\u70ba\u4e0d\u540c\u7684\u7d44\u7e54\u5668\u5b98\uff0c\u76f8\u95dc\u7684\u7814\u7a76\u4e0d\u514d\u727d\u6d89\u5230\u502b\u7406\u9053\u5fb7\u7684\u722d\u8b70\uff0c\u53cd\u5176\u9053\u800c\u884c\u7684\u662f\uff0c\u7531\u65e5\u672c\u91ab\u5b78\u5bb6\u5c71\u4e2d\u7533\u5f4c\u9818\u5c0e\u7684\u7814\u7a76\u5718\u968a\u767c\u73fe\uff0c\u53ef\u4ee5\u628a\u4eba\u9ad4\u7684\u76ae\u819a\u7d30\u80de\u7d93\u904e\u8655\u7406\u5f8c\u8b8a\u6210\u5e79\u7d30\u80de\uff0c\u5c31\u662f\u7531\u300c\u4e00\u300d\u56de\u5230\u300c\u5168\u300d\u7684\u72c0\u614b\uff0c\u7a31\u70ba\u300c\u8a98\u767c\u5168\u80fd\u5e79\u7d30\u80de\u300d(Induced pluripotent stem cells\uff1biPS)\uff0c\u5f97\u5230\u7684iPS\u7d30\u80de\u5e7e\u4e4e\u80fd\u5920\u5206\u5316\u70ba\u4eba\u9ad4\u4efb\u4f55\u7684\u7d30\u80de\u7d44\u7e54\uff0c\u5c71\u4e2d\u7533\u5f4c\u4ea6\u56e0\u70ba\u7d30\u80de\u91cd\u7de8\u7a0b\u7684\u7814\u7a76\uff0c\u8207\u82f1\u570b\u91ab\u5b78\u5bb6John Gurdon\u5171\u540c\u5206\u4eab2012\u5e74\u8afe\u8c9d\u723e\u91ab\u5b78\u734e\u3002<!--more--><\/p>\n<p>\u5e38\u8a00\u9053\u91ab\u5b78\u660c\u660e\uff0c\u4f46\u7d30\u80de\u6cbb\u7642\u904b\u7528\u65bc\u5fc3\u8840\u7ba1\u75be\u75c5\u7684\u73fe\u72c0\uff0c\u4ecd\u7136\u8655\u65bc\u778e\u5b50\u6478\u8c61\u7684\u968e\u6bb5\uff0c\u5be6\u969b\u4e0a\u91ab\u5b78\u754c\u5b58\u5728\u4e0d\u5c11\u76f2\u9ede\u8207\u4fb7\u9650\u7b49\u5f85\u91d0\u6e05\u8207\u7a81\u7834\uff0c\u7d30\u80de\u6cbb\u7642\u4e5f\u4e0d\u662f\u7121\u6240\u4e0d\u80fd\uff0c\u63a5\u53d7\u6cbb\u7642\u524d\u52d9\u5fc5\u505a\u597d\u529f\u8ab2\uff0c\u5207\u52ff\u8f15\u4fe1\u8b20\u8a00\u3002<\/p>\n<p><strong>\u57fa\u56e0\u6cbb\u7642<\/strong><\/p>\n<p>\u300c\u82ef\u916e\u5c3f\u75c7\u300d(Phenylketonuria\uff1bPKU)\u662f\u4e00\u7a2e\u907a\u50b3\u75be\u75c5\uff0c\u60a3\u8005\u5728\u51fa\u751f\u6642\u6c92\u6709\u751a\u9ebc\u7570\u6a23\uff0c\u537b\u5728\u9010\u6f38\u6210\u9577\u4e2d\u7522\u751f\u667a\u529b\u969c\u7919\u7b49\u75c7\u72c0\uff0c\u632a\u5a01\u91ab\u5e2bIvar Asbj\u00f8rn F\u00f8lling\u901a\u904e\u89c0\u5bdf\u7814\u7a76\u767c\u73fe\uff0c\u9019\u4e9b\u60a3\u8005\u7684\u5c3f\u6db2\u4e2d\u82ef\u916e\u9178\u7684\u542b\u91cf\u90fd\u504f\u9ad8\uff0c\u75be\u75c5\u6a5f\u5236\u662f\u7531\u65bc\u4eba\u9ad4\u5167\u6c28\u57fa\u9178\u4e2d\u7684\u82ef\u4e19\u80fa\u9178\u4ee3\u8b1d\u5931\u5e38\uff0c\u9020\u6210\u8166\u90e8\u4e0d\u540c\u7a0b\u5ea6\u7684\u7834\u58de\uff0c\u6cbb\u7642\u65b9\u6848\u5f9e\u5982\u4f55\u907f\u514d\u4eba\u9ad4\u651d\u53d6\u82ef\u4e19\u80fa\u9178\u7684\u89d2\u5ea6\u5207\u5165\uff0c\u5fb7\u570b\u6559\u6388Horst Bickel\u9996\u5148\u8abf\u914d\u51fa\u4e0d\u542b\u82ef\u4e19\u80fa\u9178\u7684\u6c28\u57fa\u9178\u98f2\u6599\uff0c\u80fd\u5920\u6709\u6548\u63a7\u5236\u75c5\u60c5\uff0c\u4f46\u795e\u7d93\u9000\u5316\u662f\u4e0d\u53ef\u9006\u7684\u50b7\u5bb3\uff0c\u75c7\u72c0\u986f\u73fe\u6642\u5f80\u5f80\u70ba\u6642\u5df2\u665a\u3002\u96a8\u8457\u91ab\u5b78\u7684\u9032\u6b65\uff0c\u73fe\u5728\u53ef\u4ee5\u900f\u904e\u65b0\u751f\u5152\u7684\u7be9\u6aa2\u53ca\u65e9\u767c\u73fe\u75c5\u60a3\uff0c\u7d66\u4e88\u98f2\u98df\u6cbb\u7642\u3002\u7136\u800c\u5373\u4fbf\u5982\u6b64\uff0c\u4ecd\u7136\u6709\u4e00\u90e8\u5206\u60a3\u8005\u75c5\u60c5\u4e26\u6c92\u6709\u56e0\u6b64\u53d7\u5230\u63a7\u5236\uff0c\u7a31\u70ba\u300c\u60e1\u6027\u82ef\u916e\u5c3f\u75c7\u300d\uff0c\u9019\u4e9b\u75c5\u60a3\u53ef\u4ee5\u900f\u904e\u85e5\u7269\u88dc\u5145\u591a\u5df4\u80fa\u7b49\u795e\u7d93\u50b3\u5c0e\u7269\u8cea\u4f86\u63a7\u5236\u75c5\u60c5\u3002\u53e6\u5916\uff0c\u6eb6\u5c0f\u9ad4\u662f\u7d30\u80de\u5167\u7684\u5783\u573e\u8655\u7406\u5834\uff0c\u4f9d\u9760\u6eb6\u5c0f\u9ad4\u9175\u7d20\u5206\u89e3\u5783\u573e\uff0c\u7576\u9019\u4e9b\u9175\u7d20\u5931\u9748\uff0c\u9ad4\u5167\u5783\u573e\u5c31\u6703\u56e0\u70ba\u6c92\u6709\u5f97\u5230\u6709\u6548\u5206\u89e3\u800c\u7d2f\u7a4d\u81f4\u75c5\uff0c\u9019\u5c31\u662f\u300c\u6eb6\u5c0f\u9ad4\u5132\u7a4d\u75c7\u300d(Lysosomal storage diseases)\u3002\u6eb6\u5c0f\u9ad4\u5132\u7a4d\u75c7\u662f\u4e00\u7a2e\u7f55\u898b\u907a\u50b3\u75be\u75c5\uff0c\u7406\u8ad6\u4e0a\u53ef\u4ee5\u900f\u904e\u88dc\u5145\u7f3a\u4e4f\u7684\u86cb\u767d\u8cea\u4f86\u6cbb\u7642\uff0c\u4f46\u5be6\u969b\u4e0a\u86cb\u767d\u8cea\u5316\u5b78\u7d50\u69cb\u975e\u5e38\u8907\u96dc\uff0c\u73fe\u4eca\u91ab\u5b78\u6280\u8853\u662f\u501f\u52a9\u751f\u7269\u9ad4\u4f86\u88fd\u9020\uff0c\u5177\u9ad4\u505a\u6cd5\u662f\u628a\u76ee\u6a19\u57fa\u56e0\u7684\u8f09\u9ad4\uff0c\u5229\u7528\u7d50\u69cb\u8207\u7d30\u80de\u819c\u76f8\u8fd1\u7684\u8102\u80aa\u5305\u88f9\u5f8c\u6ce8\u5165\u751f\u7269\u9ad4\u7d30\u80de\u5167\uff0c\u8102\u80aa\u5c0f\u7403\u8207\u7d30\u80de\u819c\u76f8\u4e92\u78b0\u649e\u5f8c\u7d50\u5408\uff0c\u60c5\u5f62\u5c31\u597d\u50cf\u5169\u500b\u6c23\u6ce1\u78b0\u649e\u5f8c\u878d\u5408\u5728\u4e00\u8d77\uff0c\u76ee\u6a19\u57fa\u56e0\u5c31\u9019\u6a23\u9472\u5165\u5230\u751f\u7269\u9ad4\u57fa\u56e0\u5167\uff0c\u900f\u904e\u7d30\u80de\u4e0d\u65b7\u7e41\u6b96\uff0c\u4f7f\u76f8\u95dc\u86cb\u767d\u8cea\u5f97\u4ee5\u5927\u898f\u6a21\u88fd\u9020\u3002<\/p>\n<p>\u57fa\u56e0\u6cbb\u7642\u7684\u65b9\u5f0f\uff0c\u53ef\u4ee5\u662f\u88ab\u52d5\u5730\u5c0d\u4e0d\u8db3\u7684\u7d66\u4e88\u88dc\u5145\u6216\u8005\u662f\u5c0d\u904e\u5269\u7684\u9032\u884c\u63a7\u5236\uff0c\u4e5f\u53ef\u4ee5\u662f\u4e3b\u52d5\u5730\u5c0d\u81f4\u75c5\u57fa\u56e0\u9032\u884c\u4fee\u5fa9\u3002\u628a\u87a2\u5149\u57fa\u56e0\u6ce8\u5165\u5c0f\u9f20\u7684\u5375\u5b50\uff0c\u65e5\u5f8c\u8a72\u53d7\u7cbe\u5375\u5c31\u6703\u767c\u80b2\u6210\u87a2\u5149\u9f20\uff0c\u57fa\u56e0\u8f49\u6b96\u6280\u8853\u4e5f\u5ee3\u6cdb\u61c9\u7528\u65bc\u8c6c\u3001\u96de\u3001\u9b5a\u7b49\u52d5\u7269\u8eab\u4e0a\uff0c\u4f46\u4ee5\u9019\u7a2e\u9010\u4e00\u6ce8\u5c04\u7684\u65b9\u5f0f\uff0c\u5c0d\u52d5\u8f12\u4e0a\u5104\u8a08\u7684\u4eba\u9ad4\u7d30\u80de\u9032\u884c\u57fa\u56e0\u6cbb\u7642\uff0c\u6280\u8853\u4e0a\u76f8\u7576\u56f0\u96e3\uff0c\u56e0\u6b64\uff0c\u5229\u7528\u75c5\u6bd2\u5fc5\u9808\u4f9d\u9644\u5bbf\u4e3b\u7d30\u80de\u7684\u5feb\u901f\u7e41\u6b96\u6a21\u5f0f\uff0c\u62bd\u96e2\u75c5\u6bd2\u539f\u6709\u7684\u57fa\u56e0\uff0c\u7136\u5f8c\u6ce8\u5165\u6cbb\u7642\u6240\u9700\u7684\u57fa\u56e0\uff0c\u8b93\u75c5\u6bd2\u4f9d\u820a\u611f\u67d3\u4eba\u9ad4\u7d30\u80de\u3002\u4e0d\u540c\u7684\u662f\uff0c\u6b64\u6642\u75c5\u6bd2\u6b63\u627f\u8f09\u8457\u6cbb\u7642\u7528\u7684\u57fa\u56e0\uff0c\u9032\u884c\u57fa\u56e0\u6cbb\u7642\u3002\u7528\u4f5c\u626e\u6f14\u8f09\u9ad4\u89d2\u8272\u7684\u75c5\u6bd2\u985e\u578b\u5305\u62ec\u817a\u75c5\u6bd2\u3001\u817a\u75c5\u6bd2\u9644\u5c6c\u75c5\u6bd2(AAV)\u3001\u53cd\u8f49\u9304\u75c5\u6bd2\u3001Lenti\u75c5\u6bd2\u7b49\u3002\u7f55\u898b\u907a\u50b3\u75be\u75c5\u300c\u840a\u4f2f\u6c0f\u5148\u5929\u6027\u9ed1\u77c7\u75c7\u300d(Leber\u2019s Congenital Amaurosis\uff1bLCA)\u7531\u65bc\u8996\u7db2\u819c\u4e0a\u611f\u5149\u7d30\u80de\u904b\u4f5c\u6240\u5fc5\u9808\u7684\u9175\u7d20\u7f3a\u4e4f\uff0c\u60a3\u8005\u5728\u51fa\u751f\u5f8c\u8996\u529b\u6703\u4e0d\u65b7\u8870\u9000\uff0c\u6700\u5f8c\u751a\u81f3\u5931\u660e\uff0c\u5229\u7528\u57fa\u56e0\u6cbb\u7642\u7684\u65b9\u5f0f\uff0c\u628a\u76ee\u6a19\u57fa\u56e0\u7d93\u7531\u75c5\u6bd2\u5e36\u5230\u8996\u7db2\u819c\u9032\u884c\u7f3a\u9677\u4fee\u5fa9\uff0c\u8853\u5f8c\u75c5\u4eba\u8996\u529b\u6062\u5fa9\uff0c\u53ef\u4ee5\u6b63\u5e38\u5730\u751f\u6d3b\u3002\u800c\u5728\u624b\u8853\u524d\uff0c\u5149\u662f\u7e5e\u904e\u4e00\u822c\u7684\u969c\u7919\u7269\u5c31\u8db3\u4ee5\u8b93\u75c5\u4eba\u5403\u76e1\u82e6\u982d\u3002\u53e6\u5916\uff0c\u5df4\u91d1\u68ee\u6c0f\u75c7(Parkinson disease)\u60a3\u8005\u6703\u6709\u8eab\u9ad4\u986b\u6296\u3001\u884c\u52d5\u7de9\u6162\u7b49\u904b\u52d5\u6280\u80fd\u969c\u7919\uff0c\u5c0d\u75c5\u60a3\u8166\u90e8\u76f4\u63a5\u6ce8\u5c04\u75c5\u6bd2\u8f09\u9ad4\u9032\u884c\u6cbb\u7642\uff0c\u8853\u5f8c\u75c5\u4eba\u7684\u904b\u52d5\u80fd\u529b\u6703\u5f97\u5230\u660e\u986f\u6539\u5584\u3002<\/p>\n<p>\u57fa\u56e0\u6cbb\u7642\u9664\u4e86\u6ce8\u5c04\u904b\u8f09\u76ee\u6a19\u57fa\u56e0\u7684\u75c5\u6bd2\u5230\u60a3\u8005\u9ad4\u5167\u9032\u884c\u57fa\u56e0\u4fee\u88dc\u5916\uff0c\u9084\u53ef\u4ee5\u62bd\u53d6\u60a3\u8005\u7684\u5e79\u7d30\u80de\u5230\u9ad4\u5916\u57f9\u990a\uff0c\u628a\u75c5\u6bd2\u8f09\u9ad4\u9001\u5230\u5e79\u7d30\u80de\u9032\u884c\u57fa\u56e0\u4fee\u5fa9\uff0c\u4fee\u5fa9\u5b8c\u6210\u5f8c\u518d\u9001\u56de\u4eba\u9ad4\u5167\u3002\u7f8e\u570b\u66fe\u7d93\u6709\u4e00\u500b\u7f79\u60a3\u91cd\u5ea6\u514d\u75ab\u7f3a\u4e4f\u75c7(Severe combined immunodeficiency\uff1bSCID) \u7684\u5c0f\u7537\u5b69\uff0c\u88ab\u5b89\u6392\u751f\u6d3b\u5728\u6c92\u6709\u81f4\u75c5\u56e0\u5b50\u7684\u5851\u81a0\u6ce1\u6ce1\u5167\uff0c\u7576\u6642\u9019\u7a2e\u75c5\u75c7\u53ea\u80fd\u900f\u904e\u9aa8\u9ad3\u79fb\u690d\u6cbb\u7642\uff0c\u4f46\u9069\u5408\u79fb\u690d\u7684\u9aa8\u9ad3\u5230\u6700\u5f8c\u90fd\u6c92\u6709\u51fa\u73fe\uff0c\u5c0f\u7537\u5b69\u7d42\u5176\u4e00\u751f\u90fd\u9700\u8981\u8207\u4e16\u9694\u7d55\u3002\u73fe\u5728\uff0c\u9664\u4e86\u9aa8\u9ad3\u79fb\u690d\u5916\uff0c\u900f\u904e\u62bd\u53d6\u75c5\u4eba\u7684\u8840\u6db2\u5e79\u7d30\u80de\uff0c\u5229\u7528\u75c5\u6bd2\u904b\u8f09\u76ee\u6a19\u57fa\u56e0\uff0c\u5c0d\u75c5\u60a3\u5e79\u7d30\u80de\u5167\u81f4\u75c5\u57fa\u56e0\u9032\u884c\u77ef\u6b63\uff0c\u63a5\u8457\u628a\u77ef\u6b63\u5f8c\u5e79\u7d30\u80de\u6ce8\u5c04\u56de\u75c5\u4eba\u9ad4\u5167\u7684\u6cbb\u7642\uff0c\u4e5f\u80fd\u9054\u5230\u76f8\u8fd1\u7684\u7642\u6548\u3002\u4f46\u81e8\u5e8a\u4e0a\u6709\u90e8\u5206\u6848\u4f8b\u5728\u6cbb\u7642\u5f8c\u51fa\u73fe\u56b4\u91cd\u526f\u4f5c\u7528\uff0c\u6cbb\u7642\u6210\u679c\u4ecd\u6709\u76f8\u7576\u6539\u9032\u7a7a\u9593\u3002\u800c\u76f8\u540c\u7684\u6cbb\u7642\u65b9\u6cd5\u61c9\u7528\u65bc\u5927\u8166\u767d\u8cea\u9000\u5316\u75c7(Adrenoleukodystrophy\uff1bALD)\u7684\u75c5\u4eba\u4e0a\u6642\uff0c\u9996\u5148\u5f9e\u75c5\u60a3\u8eab\u4e0a\u62bd\u53d6\u9020\u8840\u5e79\u7d30\u80de\uff0c\u63a5\u8457\u5229\u7528\u5e36\u6709\u76ee\u6a19\u57fa\u56e0\u7684\u75c5\u6bd2\u53bb\u611f\u67d3\u60a3\u8005\u7684\u5e79\u7d30\u80de\uff0c\u4fee\u5fa9\u539f\u6709\u7f3a\u9677\u7684\u57fa\u56e0\uff0c\u7136\u5f8c\u518d\u628a\u5b8c\u6210\u4fee\u5fa9\u7684\u5e79\u7d30\u80de\u6ce8\u5c04\u5230\u75c5\u4eba\u9ad4\u5167\uff0c\u6cbb\u7642\u6548\u679c\u4ee4\u4eba\u76f8\u7576\u6eff\u610f\u3002<\/p>\n<p>\u8166\u795e\u7d93\u4e4b\u9593\u4f9d\u9760\u795e\u7d93\u50b3\u5c0e\u7d20\u50b3\u905e\u8a0a\u606f\uff0c\u300c\u82b3\u9999\u65cfL-\u80fa\u57fa\u9178\u812b\u7fa7\u9175\u7d20\u7f3a\u4e4f\u75c7\u300d(Aromatic L-amino acid decarboxylase\uff1bAADC)\u662f\u7531\u65bc\u76f8\u95dc\u7684\u795e\u7d93\u50b3\u5c0e\u7d20\u7f3a\u4e4f\u6d3b\u6027\uff0c\u8166\u90e8\u6307\u4ee4\u7121\u6cd5\u9806\u5229\u50b3\u9054\u800c\u81f4\u75c5\uff0c\u800c\u7d93\u904e\u57fa\u56e0\u6cbb\u7642\u5f8c\u539f\u672c\u751f\u9577\u9072\u7de9\u4e26\u4e14\u81ea\u5f8b\u795e\u7d93\u7cfb\u7d71\u529f\u80fd\u5931\u8abf\u7684\u5c0f\u9f20\uff0c\u8b8a\u5f97\u6d3b\u8e66\u4e82\u8df3\u800c\u4e14\u5145\u6eff\u6d3b\u529b\u3002\u6b64\u5916\uff0c\u6a61\u76ae\u7d93\u904e\u53cd\u8986\u62c9\u5f35\u5f8c\u6703\u51fa\u73fe\u9b06\u5f1b\u73fe\u8c61\uff0c\u9ecf\u591a\u91a3\u8ca0\u8cac\u7dad\u6301\u4eba\u9ad4\u76ae\u819a\u53ca\u7d50\u7de0\u7d44\u7e54\u7684\u5f48\u6027\uff0c\u540c\u6a23\u5730\uff0c\u9ecf\u591a\u91a3\u4e5f\u9700\u8981\u4e0d\u65b7\u6dd8\u6c70\u66f4\u65b0\u4f86\u4fdd\u6301\u5f48\u6027\uff0c\u7576\u8ca0\u8cac\u65b0\u9673\u4ee3\u8b1d\u7684\u9175\u7d20\u6d3b\u6027\u7f3a\u4e4f\uff0c\u4eba\u9ad4\u5c31\u6703\u56e0\u70ba\u592a\u591a\u63ee\u4e4b\u4e0d\u53bb\u7684\u9ecf\u591a\u91a3\u800c\u81f4\u75c5\uff0c\u9019\u5c31\u662f\u300c\u9ecf\u591a\u91a3\u5132\u7a4d\u75c7\u300d(Mucopolysaccharidosis\uff1bMPS)\uff0c\u5171\u6709\u4e5d\u7a2e\u985e\u578b\uff0c\u5176\u4e2d\u9ecf\u591a\u91a3\u75c7\u7b2c\u516d\u578b\u7684\u6cbb\u7642\uff0c\u76ee\u524d\u4e3b\u8981\u662f\u900f\u904e\u6ce8\u5c04\u9175\u7d20\u4ee5\u5354\u52a9\u5206\u89e3\u4eba\u9ad4\u5167\u591a\u9918\u9ecf\u591a\u91a3\u7684\u65b9\u5f0f\u9032\u884c\uff0c\u4f46\u6301\u7e8c\u6027\u9175\u7d20\u6ce8\u5c04\u82b1\u8cbb\u751a\u9245\uff0c\u800c\u4e14\u904e\u91cf\u7684\u9ecf\u591a\u91a3\u53ef\u80fd\u6703\u50b7\u53ca\u8166\u90e8\u7d44\u7e54\uff0c\u4f46\u9019\u4e9b\u9175\u7d20\u537b\u7121\u6cd5\u9032\u5165\u8166\u90e8\uff0c\u8af8\u591a\u4e8b\u5be6\u90fd\u8868\u660e\uff0c\u57fa\u56e0\u6cbb\u7642\u4e26\u4e0d\u662f\u591a\u6b64\u4e00\u8209\uff0c\u800c\u662f\u52e2\u5728\u5fc5\u884c\uff0c\u5c0d\u65bc\u7b2c\u516d\u578b\u7684\u57fa\u56e0\u6cbb\u7642\u76ee\u524d\u5c1a\u8655\u65bc\u8a66\u9a57\u968e\u6bb5\uff0c\u5df2\u77e5\u5728\u90e8\u5206\u52d5\u7269\u5be6\u9a57\u4e0a\u53d6\u5f97\u6210\u529f\uff0c\u53ef\u671b\u5728\u4e0d\u4e45\u7684\u5c07\u4f86\u666e\u53ca\u5230\u81e8\u5e8a\u6cbb\u7642\u3002<\/p>\n<p><strong>\u626d\u8f49\u5bbf\u547d<\/strong><\/p>\n<p>\u77e5\u8b58\u8d77\u6e90\u65bc\u7d93\u9a57\uff0c\u4eba\u985e\u7531\u4e00\u7121\u6240\u77e5\uff0c\u900f\u904e\u4e0d\u65b7\u5617\u8a66\u7576\u4e2d\u7e3d\u7d50\u898f\u5f8b\uff0c\u5f9e\u53e4\u6642\u5019\u795e\u8fb2\u6c0f\u5617\u767e\u8349\uff0c\u5230\u73fe\u4ee3\u57fa\u56e0\u7d30\u80de\u6cbb\u7642\uff0c\u63a2\u7d22\u5230\u767c\u73fe\uff0c\u5468\u800c\u5fa9\u59cb\uff0c\u904e\u53bb\u66fe\u7d93\u8b93\u4eba\u675f\u624b\u7121\u7b56\u7684\u7591\u96e3\u96dc\u75c7\uff0c\u5f88\u591a\u90fd\u5df2\u7d93\u53d7\u5230\u63a7\u5236\uff0c\u9084\u8655\u65bc\u7814\u767c\u968e\u6bb5\u7684\uff0c\u5f88\u591a\u90fd\u5c07\u8fd1\u61c9\u7528\u5230\u81e8\u5e8a\u6cbb\u7642\u4e0a\uff0c\u9019\u4e0d\u662f\u4e0d\u8457\u908a\u969b\u7684\u865b\u60f3\uff0c\u800c\u662f\u89f8\u624b\u53ef\u53ca\u7684\u5be6\u5728\uff0c\u53ea\u8981\u638c\u63e1\u57fa\u56e0\u907a\u50b3\u7684\u904b\u4f5c\u898f\u5f8b\uff0c\u627e\u51fa\u96b1\u60e1\u63da\u5584\u4e4b\u9053\uff0c\u547d\u904b\u4e26\u4e0d\u662f\u65e9\u5df2\u8a3b\u5b9a\uff0c\u800c\u662f\u59cb\u7d42\u90fd\u638c\u63e1\u5728\u81ea\u5df1\u624b\u88e1\u3002<\/p>\n<p>--<\/p>\n<pre><span style=\"color: #333333;\">\u672c\u6587\u6574\u7406\u81ea\uff1a101\/12\/15\u4e0b\u5348\u7531\u80e1\u52d9\u4eae\u91ab\u5e2b\u5728\u81fa\u5927\u61c9\u529b\u6240\u570b\u969b\u6f14\u8b1b\u5ef3\u6240\u4e3b\u8b1b\u4e4b\u300c\u626d\u8f49\u5bbf\u547d\u7684\u57fa\u56e0\u7d30\u80de\u6cbb\u7642\u300d\u7684\u6f14\u8b1b\u5167\u5bb9<\/span><\/pre>\n<pre><span style=\"color: #333333;\">\u5ef6\u4f38\u95b1\u8b80\uff1a\u53f0\u5927\u79d1\u5b78\u6559\u80b2\u767c\u5c55\u4e2d\u5fc3\u63a2\u7d22\u57fa\u790e\u79d1\u5b78\u8b1b\u5ea72012\u5e7412\u670815\u65e5\u7b2c\u516b\u8b1b\u3008\u626d\u8f49\u5bbf\u547d\u7684\u57fa\u56e0\u7d30\u80de\u6cbb\u7642\u3009<\/span><a href=\"https:\/\/case.ntu.edu.tw\/CASTUDIO\/course.php?speech_ID=3179#video_top\" target=\"_blank\" rel=\"noopener noreferrer\"><span style=\"color: #333333;\">\u5168\u7a0b\u5f71\u97f3<\/span><\/a><\/pre>\n<p><span style=\"color: #333399;\"><strong><strong>\u8cac\u4efb\u7de8\u8f2f\uff1aNita Hsu<\/strong><\/strong><\/span><\/p>\n","protected":false},"excerpt":{"rendered":"<p>\u7b2c\u516b\u8b1b\u2027\u7279\u7a3f \u25a0 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