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id=\"attachment_601243\" style=\"width: 410px\" class=\"wp-caption alignnone\"><img loading=\"lazy\" decoding=\"async\" aria-describedby=\"caption-attachment-601243\" src=\"https:\/\/chinadigitaltimes.net\/chinese\/files\/2018\/12\/Screen-Shot-2018-12-08-at-12.58.00-PM-e1544291908266.png\" alt=\"\" width=\"400\" height=\"321\" class=\"size-full wp-image-601243\" \/><p id=\"caption-attachment-601243\" class=\"wp-caption-text\">\u683c\u5c14\u8f9b\u57fa\uff08Jesse Gelsinger\uff09<\/p><\/div>\n<p>1990\u5e74\u4ee3\uff0c\u7f8e\u56fd\u5bbe\u5915\u6cd5\u5c3c\u4e9a\u5927\u5b66\u7684James Wilson\u6210\u529f\u5730\u5c06\u817a\u75c5\u6bd2 (Adeno-associated 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<\/p>\n<p>Luxturna\u548cCAR-T\u90fd\u53ea\u7f16\u8f91\u5f88\u5c0f\u4e00\u90e8\u5206\u4f53\u7ec6\u80de\u3002\u4f53\u7ec6\u80de\u5df2\u7ecf\u9ad8\u5ea6\u5206\u5316\uff0c\u6dcb\u5df4\u7ec6\u80de\u4e0d\u4f1a\u8f6c\u4e3a\u808c\u8089\u7ec6\u80de\u6216\u795e\u7ecf\u7ec6\u80de\uff0c\u66f4\u4e0d\u4f1a\u8f6c\u5316\u4e3a\u751f\u6b96\u7ec6\u80de\uff0c\u56e0\u6b64\u6ca1\u6709\u9057\u4f20\u7ed9\u540e\u4ee3\u7684\u98ce\u9669\u3002\u4eba\u4f53\u5185\u7ec6\u80de\u603b\u6570\u4ee5\u5341\u4e07\u4ebf\u4e3a\u6570\u91cf\u7ea7\uff0c\u800cCAR-T\u7597\u6cd5\u5bfc\u5165\u4eba\u4f53\u7684\u57fa\u56e0\u7f16\u8f91\u7ec6\u80de\u5373\u4f7f\u9ad8\u8fbe\u4e0a\u767e\u4e07\uff0c\u4e5f\u5dee\u4e8610000000\u500d\uff01<\/p>\n<p>\u7136\u800c\u5bf9\u4e8e\u663e\u6027\u7684\u9057\u4f20\u75be\u75c5\uff0c\u5982\u6e10\u51bb\u4eba\u75c7\uff08ALS\uff09\uff0c\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08muscular dystrophy), \u6216\u9057\u4f20\u6027\u7684\u963f\u5c14\u5179\u6d77\u9ed8\u75c7\uff0c\u5176\u57fa\u56e0\u7a81\u53d8\u4e0d\u4ec5\u5bfc\u81f4\u6b63\u5e38\u7684\u86cb\u767d\u6d3b\u6027\u6d88\u5931\uff0c\u8fd8\u9020\u6210\u5f02\u5e38\u7684\u6d3b\u6027\u51fa\u73b0\uff0c\u201c\u7f3a\u4ec0\u4e48\u8865\u4ec0\u4e48\u201d\u8fd9\u79cd\u7b80\u5355\u7b56\u7565\u884c\u4e0d\u901a\u3002 \u8981\u6839\u6cbb\u8fd9\u7c7b\u9057\u4f20\u75c5\uff0c\u9700\u8981\u5207\u9664\u5f02\u5e38\u7684\u57fa\u56e0\u62f7\u8d1d\uff0c\u7528\u6b63\u5e38\u7684\u62f7\u8d1d\u53d6\u800c\u4ee3\u4e4b\u3002<\/p>\n<p>\u817a\u75c5\u6bd2\u8f7d\u4f53\u53ea\u80fd\u5c06\u5916\u6e90\u57fa\u56e0\u5bfc\u5165\u7ec6\u80de\u8d28\u4e2d\uff0c\u5bf9\u57fa\u56e0\u7ec4\u6ca1\u6709\u5f71\u54cd\u3002\u6162\u75c5\u6bd2\u8f7d\u4f53\u867d\u7136\u53ef\u4ee5\u8ba9\u5916\u6e90\u57fa\u56e0\u63d2\u5165\u7ec6\u80de\u672c\u8eab\u7684\u57fa\u56e0\u7ec4\u4e2d\uff0c\u4f46\u63d2\u5165\u662f\u968f\u673a\u7684\uff0c\u867d\u7136\u6574\u4e2a\u57fa\u56e0\u7ec4\u4e2d\u53ea\u67092%\u7684\u5e8f\u5217\u662f\u76f4\u63a5\u7f16\u7801\u86cb\u767d\u8d28\u7684\u57fa\u56e0\uff0c\u4f46\u8fd9\u4e2a\u6982\u7387\u5df2\u7ecf\u53ef\u80fd\u9020\u6210\u6b63\u5e38\u57fa\u56e0\u7684\u7834\u574f\uff0c\u8fd9\u8fdc\u4e0d\u8db3\u4ee5\u5bf9\u6ee1\u8db3\u4e34\u5e8a\u5b89\u5168\u6027\u7684\u8981\u6c42\u3002\u6700\u5173\u952e\u7684\u662f\uff0c\u75c5\u6bd2\u8f7d\u4f53\u53ea\u80fd\u6dfb\u52a0\u989d\u5916\u7684\u57fa\u56e0\uff0c\u4e0d\u80fd\u5220\u9664\u3001\u4fee\u6539\u5df2\u7ecf\u5b58\u5728\u4e0e\u67d3\u8272\u4f53\u4e0a\u7684\u57fa\u56e0\u3002\u524d\u8005\u597d\u6bd4\u628a\u4e00\u6839\u9488\u4e22\u8fdb\u7a3b\u8349\u5806\u91cc\uff0c\u540e\u8005\u5c31\u50cf\u4ece\u7a3b\u8349\u5806\u91cc\u6311\u51fa\u4e00\u6839\u9488\uff0c\u5b9e\u73b0\u8d77\u6765\u7684\u96be\u5ea6\u5f88\u660e\u663e\u662f\u4e0d\u540c\u7684\uff0c\u4e3a\u6b64\u6211\u4eec\u9700\u8981\u65b0\u7684\u5de5\u5177\u3002<\/p>\n<p>\u57fa\u56e0\u7f16\u8f91\u7684\u5229\u5668\uff1aCRISPR<\/p>\n<p>\u4eca\u5929\u6211\u4eec\u6240\u8bf4\u7684\u201c\u57fa\u56e0\u7f16\u8f91\u201d\uff0c\u5176\u5b9e\u6307\u7684\u662f\u201c\u57fa\u56e0\u7ec4\u7f16\u8f91\u201d\u3002\u5982\u679c\u53ea\u662f\u8981\u4fee\u6539\u8bd5\u7ba1\u4e2d\u7684\u4e00\u6bb5\u6e38\u79bbDNA\uff0c\u90a3\u6280\u672f\u5df2\u582a\u79f0\u7089\u706b\u7eaf\u9752\uff0c\u5b9e\u73b0\u8d77\u6765\u6beb\u65e0\u96be\u5ea6\u3002\u4e3a\u4ec0\u4e48\u4fee\u6539\u57fa\u56e0\u7ec4\u4f1a\u8fd9\u4e48\u56f0\u96be\uff0c\u800c\u57fa\u56e0\u7ec4\u7f16\u8f91\u6280\u672f\u88ab\u79f0\u4e3a\u8fd1\u5e74\u6765\u751f\u7269\u6280\u672f\u6700\u91cd\u8981\u7684\u7a81\u7834\u5462\uff1f<\/p>\n<p>\u9996\u5148\u57fa\u56e0\u7ec4\u6709\u6d69\u701a\u7684\u6570\u636e\u91cf\u3002\u4eba\u7c7b\u57fa\u56e0\u7ec4\u6709\u4e09\u5341\u4ebf\u4e2a\u78b1\u57fa\u5bf9\uff0c\u4e00\u4e2a\u57fa\u56e0\u7684\u7f16\u7801\u533a\u5f88\u5c11\u8d85\u8fc7\u4e00\u4e07\u4e2a\u78b1\u57fa\u5bf9\uff0c\u800c\u81f4\u75c5\u7684\u7a81\u53d8\u53ef\u80fd\u53ea\u7275\u6d89\u5176\u4e2d\u6570\u4e2a\u3001\u751a\u81f3\u5355\u4e2a\u7684\u78b1\u57fa\u5bf9\u3002\u8981\u5b9a\u4f4d\u4e09\u5341\u4ebf\u5206\u4e4b\u4e00\u7684\u4f4d\u70b9\uff0c\u96be\u5ea6\u663e\u800c\u6613\u89c1\u3002\u5176\u6b21\uff0c\u57fa\u56e0\u7ec4\u4e0d\u662f\u4e00\u6bb5\u88f8\u9732\u7684DNA\u3002\u6211\u4eec\u77e5\u9053DNA\u5177\u6709\u53cc\u87ba\u65cb\u7ed3\u6784\u3002\u87ba\u65cb\u5916\u4fa7\u7531\u7cd6\u7c7b\u548c\u78f7\u9178\u6784\u6210\u9aa8\u67b6\uff0c\u5185\u4fa7\u7684\u78b1\u57fa\u4e92\u8865\u914d\u5bf9\uff0c\u7f16\u7801\u9057\u4f20\u4fe1\u606f\u3002\u53cc\u87ba\u65cb\u7f20\u7ed5\u5728\u86cb\u767d\u8d28\u6784\u6210\u7684\u6838\u5c0f\u4f53\u4e0a\uff0c\u770b\u8d77\u6765\u50cf\u4e00\u4e32\u5ff5\u73e0\u3002\u8fd9\u4e32\u5ff5\u73e0\u53c8\u7ee7\u7eed\u6298\u53e0\u6210\u66f4\u590d\u6742\u7684\u4e09\u7ef4\u7ed3\u6784\uff0c\u6211\u4eec\u79f0\u5176\u4e3a\u67d3\u8272\u8d28\u3002\u8981\u7f16\u8f91\u67d0\u4e2a\u7279\u5b9a\u7684\u78b1\u57fa\uff0c\u9996\u5148\u8981\u770b\u67d3\u8272\u8d28\u662f\u4e0d\u662f\u6709\u677e\u6563\u5f00\u6765\u7684\u65f6\u523b\uff0c\u53ea\u6709\u66b4\u9732\u7684DNA\u624d\u662f\u53ef\u7f16\u8f91\u7684\u3002\u7136\u540e\u8981\u628a\u53cc\u87ba\u65cb\u5c40\u90e8\u62c6\u6563\uff0c\u5728\u6b63\u786e\u7684\u4f4d\u7f6e\u4e0a\u5207\u4e00\u201c\u5200\u201d\u3002 \u80fd\u5728\u5206\u5b50\u6c34\u5e73\u64cd\u7eb5DNA\u7684\uff0c\u81ea\u8eab\u8981\u5c0f\u5230\u7eb3\u7c73\u7ea7\u522b\u3002\u6211\u4eec\u9700\u8981\u7684\uff0c\u662f\u4e00\u79cd\u80fd\u81ea\u4e3b\u5b9e\u73b0\u76ee\u6807\u5b9a\u4f4d\u3001\u89e3\u5f00\u53cc\u87ba\u65cb\u3001\u5207\u65adDNA\u9aa8\u67b6\u7684\u6838\u9178\u9176\u3002<\/p>\n<p>\u8fc4\u4eca\u4e3a\u6b62\uff0c\u79d1\u5b66\u5bb6\u53d1\u660e\u7684\u57fa\u56e0\u7ec4\u7f16\u8f91\u5de5\u5177\u6709\u4e09\u79cd\uff1a ZFN(zinc-finger nucleases), TALEN\uff08 transcription activator-like effector nucleases\uff09\uff0c \u548cCRISPR( Clustered Regularly Interspaced Short Palindromic Repeats)\u3002\u9650\u4e8e\u7bc7\u5e45\uff0c\u8fd9\u91cc\u4e0d\u4ecb\u7ecdZFN\u548cTALEN\uff0c\u800c\u628a\u91cd\u70b9\u653e\u5728\u8fd9\u6b21\u57fa\u56e0\u7f16\u8f91\u5a74\u513f\u4e8b\u4ef6\u4e2d\u4e07\u4f17\u77a9\u76ee\u7684CRISPR\u4e0a\u3002<\/p>\n<p>CRISPR\u7684\u57fa\u672c\u7ec4\u4ef6\u6709\u4e24\u4e2a\uff1a\u4e00\u4e2a\u53eb\u505aCAS\u7684\u6838\u9178\u9176\uff0c\u548c\u4e00\u6bb5\u957f\u7ea6100\u4e2a\u78b1\u57fa\u7684RNA\u5206\u5b50\uff0c\u66f4\u786e\u5207\u5f97\u8bb2\uff0c\u662f\u5f15\u5bfcRNA\u5206\u5b50\uff08guide RNA\uff0cgRNA\uff09\u3002gRNA\u4e2d\u95f4\u4e00\u822c\u670920\u4e2a\u78b1\u57fa\uff0c\u6839\u636e\u78b1\u57fa\u4e92\u8865\u8bc6\u522b\u539f\u5219\u8bc6\u522b\u76ee\u6807\u57fa\u56e0\u4e2d\u7684\u4e00\u5c0f\u6bb5\u5e8f\u5217\u3002gRNA\u5269\u4f59\u7684\u90e8\u5206\uff0c\u4e0eCAS\u7ed1\u5b9a\u5728\u4e00\u8d77\u3002CAS\u548cgRNA\u53cc\u5251\u5408\u74a7\uff0c\u53ef\u4ee5\u628a\u5c40\u90e8\u7684\u53cc\u87ba\u65cb\u89e3\u5f00\u3002\u7136\u540eCAS\u5c31\u5728DNA\u53cc\u94fe\u4e0a\u5207\u51fa\u4e00\u4e2a\u53e3\u5b50\u3002\u672c\u6765\u7ec6\u80de\u5bf9DNA\u53cc\u94fe\u51fa\u73b0\u5207\u53e3\u8fd9\u79cd\u60c5\u51b5\u662f\u6709\u7d27\u6025\u9884\u6848\u7684\uff0c\u8d1f\u8d23DNA\u4fee\u590d\u7684\u9176\u4f1a\u8d76\u8fc7\u6765\u5728CAS\u5236\u9020\u7684\u5207\u53e3\u4e0a\u6253\u4e2a\u8865\u4e01\u3002\u95ee\u9898\u662f\uff0c\u4fee\u590d\u9176\u7684\u5de5\u4f5c\u4e0d\u5927\u7cbe\u7ec6\uff0c\u7ecf\u5e38\u53ea\u7ba1\u628a\u65ad\u88c2\u7684\u53cc\u94fe\u91cd\u65b0\u8fde\u63a5\u8d77\u6765\uff0c\u5374\u4e0d\u987e\u672c\u6765\u7684\u5e8f\u5217\u662f\u4ec0\u4e48\u6837\u5b50\uff0c\u80e1\u4e71\u5220\u9664\u6216\u6dfb\u52a0\u82e5\u5e72\u78b1\u57fa\u8fdb\u53bb\u3002\u5728DNA\u7f16\u8bd1\u86cb\u767d\u8d28\u7684\u5bc6\u7801\u672c\u4e0a\uff0c\u4e09\u4e2a\u78b1\u57fa\u5bf9\u5e94\u4e00\u4e2a\u6c28\u57fa\u9178\u3002\u5982\u679c\u589e\u5220\u7684\u78b1\u57fa\u4e0d\u662f\u4e09\u7684\u6574\u500d\u6570\uff0c\u5c31\u4f1a\u9020\u6210\u79fb\u7801\u7a81\u53d8\uff0c\u540e\u7eed\u6c28\u57fa\u9178\u5e8f\u5217\u5b8c\u5168\u9519\u4e71\uff0c\u751a\u81f3\u63d0\u524d\u7ec8\u6b62\u3002\u8fd9\u6837\u539f\u672c\u5b8c\u597d\u7684\u57fa\u56e0\u5728\u529f\u80fd\u5c42\u9762\u7b49\u4e8e\u88ab\u5220\u9664\u4e86\u3002<\/p>\n<p>2013\u5e74\uff0c\u6765\u81ea\u7f8e\u56fd\u9ebb\u7701\u7406\u5de5\u7684\u79d1\u5b66\u5bb6\u5f20\u950b\u3001\u4ece\u4e50\u7b49\u4eba\u7387\u5148\u62a5\u9053\u4e86\u7528CRISPR\u7f16\u8f91\u54fa\u4e73\u7ec6\u80de\u57fa\u56e0\u7ec4\u7684\u5de5\u4f5c\u3002\u4e0e\u6b64\u540c\u65f6\uff0c\u54c8\u4f5b\u533b\u5b66\u9662\u7684George Church\u56e2\u961f\u3001\u7ebd\u7ea6\u6d1b\u514b\u83f2\u52d2\u5927\u5b66\u7684Luciano Marraffini\u56e2\u961f\u4e5f\u53d1\u8868\u4e86\u7c7b\u4f3c\u7684\u7ed3\u679c\u3002CRISPR\u6700\u5927\u7684\u4f18\u52bf\u5728\u4e8e\u6a21\u5757\u5316\uff1a\u53ea\u8981\u6539\u53d8gRNA\u7684\u5e8f\u5217\uff0c\u5c31\u53ef\u4ee5\u6307\u5411\u4e0d\u540c\u7684\u57fa\u56e0\u3002\u5982\u679c\u540c\u65f6\u5411\u7ec6\u80de\u4e2d\u5bfc\u5165\u591a\u4e2agRNA\uff0c\u751a\u81f3\u53ef\u4ee5\u540c\u65f6\u7f16\u8f91\u591a\u4e2a\u57fa\u56e0\u3002\u76f8\u6bd4\u4e4b\u4e0b\uff0c\u64cd\u7eb5ZFN\u548cTALEN\u9700\u8981\u5927\u91cf\u7684\u7ecf\u9a8c\u4f18\u5316\u3002\u540e\u7eed\u7814\u7a76\u8fd8\u53d1\u73b0\uff0cCRISPR\u53ef\u4ee5\u5728\u7ec6\u83cc\u3001\u771f\u83cc\u3001\u690d\u7269\u3001\u52a8\u7269\u7b49\u5404\u79cd\u7ec6\u80de\u548c\u751f\u7269\u6d3b\u4f53\u4e2d\u5de5\u4f5c\u3002\u4ece2013\u81f3\u4eca\uff0c\u79d1\u5b66\u5bb6\u5bf9CRISPR\/CAS\u8fdb\u884c\u4e86\u4e00\u7cfb\u5217\u6539\u9020\uff0c\u8ba9\u8fd9\u628a\u57fa\u56e0\u526a\u5200\u7684\u529f\u80fd\u6108\u53d1\u5f3a\u5927\u3002CRISPR1.0\u4e3b\u8981\u7528\u4e8e\u5236\u9020\u201c\u4e71\u7801\u201d\uff0c\u800c\u4eca\u5929\u7684\u5347\u7ea7\u7248CRISPR\u5df2\u7ecf\u53ef\u4ee5\u7cbe\u786e\u4fee\u6539\u5355\u4e2a\u78b1\u57fa\uff0c\u4e43\u81f3\u5220\u9664\u6216\u6dfb\u52a0\u7279\u5b9a\u7684\u5e8f\u5217\u3002CRISPR\u7684\u51fa\u73b0\uff0c\u8ba9\u57fa\u56e0\u7ec4\u7f16\u8f91\u4ece\u4e00\u4e2a\u8017\u65f6\u8017\u529b\u3001\u9700\u8981\u4e30\u5bcc\u7ecf\u9a8c\u7684\u96be\u9898\u53d8\u6210\u4e86\u672c\u79d1\u751f\u90fd\u53ef\u4ee5\u8f7b\u677e\u5b8c\u6210\u7684\u57fa\u672c\u5b9e\u9a8c\uff01CRISPR\u88ab\u8a89\u4e3a\u8fd1\u5341\u5e74\u5185\u751f\u7269\u6280\u672f\u9886\u57df\u6700\u5927\u7684\u7a81\u7834\uff0c\u5f53\u4e4b\u65e0\u6127\u3002<\/p>\n<p>\u73b0\u5728\u6709\u4e86CRISPR\u8fd9\u4e2a\u5229\u5668\uff0c\u533b\u5b66\u5de5\u4f5c\u8005\u662f\u4e0d\u662f\u53ef\u4ee5\u8f7b\u677e\u6cbb\u7597\u9057\u4f20\u75be\u75c5\u4e86\uff1f \u4e14\u6162\uff0c\u6211\u4eec\u5230\u73b0\u5728\u8fd8\u6ca1\u6709\u8ba8\u8bba\u4e00\u4e2a\u95ee\u9898\uff1a\u6211\u4eec\u5982\u4f55\u77e5\u9053\u54ea\u4e2a\u57fa\u56e0\u4e0a\u7684\u67d0\u4e2a\u7a81\u53d8\u662f\u81f4\u75c5\u7684\u539f\u56e0\uff1f\u6211\u4eec\u53c8\u5982\u4f55\u77e5\u9053\u4fee\u6539\u8fd9\u4e2a\u7a81\u53d8\u53ef\u4ee5\u51cf\u8f7b\u75c5\u75db\uff1f<\/p>\n<p>\u57282018\u5e7411\u6708\u7684\u6700\u540e\u4e00\u5468\uff0c \u5982\u679c\u6ca1\u6709\u57fa\u56e0\u7f16\u8f91\u5a74\u513f\u4e8b\u4ef6\uff0c\u751f\u7269\u533b\u836f\u9886\u57df\u6700\u5927\u7684\u65b0\u95fb\u672c\u5e94\u8be5\u662f\u7f8e\u56fdFDA\u9996\u6b21\u6279\u51c6\u4e00\u9879\u57fa\u4e8eCRISPR\u7684\u57fa\u56e0\u7597\u6cd5\u8fdb\u5165\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u800c\u7533\u8bf7\u8fd9\u4e2a\u4e34\u5e8a\u8bd5\u9a8c\u7684\uff0c\u6b63\u662f\u5f20\u950b\u521b\u5efa\u7684Editas\u3002\u8fd9\u9879\u7597\u6cd5\u9488\u5bf9\u7684\u662f\u4e00\u79cd\u53eb\u505aLeber\u5148\u5929\u6027\u9ed1\u6726\uff08 Leber&#8217;s congenital amaurosis\uff0c LCA\uff09\u7684\u7f55\u89c1\u75be\u75c5\u3002\u5341\u4e5d\u4e16\u7eaa\u4e03\u5341\u5e74\u4ee3\uff0c\u5fb7\u56fd\u7684\u4e00\u4f4d\u773c\u79d1\u533b\u751fTheodor Leber\u9996\u6b21\u63cf\u8ff0\u4e86\u8fd9\u79cd\u4ece\u5a74\u513f\u671f\u5f00\u59cb\u53d1\u75c5\u3001\u60a3\u8005\u9010\u6e10\u5931\u53bb\u89c6\u529b\u4e43\u81f3\u5931\u660e\u7684\u9057\u4f20\u75c5\u3002\u8fd9\u79cd\u75c5\u5728\u6bcf\u5341\u4e07\u4e2a\u65b0\u751f\u513f\u4e2d\u4ec5\u6709\u4e8c\u4e09\u4f8b\uff0c\u8fdb\u4e00\u6b65\u7684\u79d1\u5b66\u7814\u7a76\u53d1\u73b0\uff0c\u6839\u636e\u81f4\u75c5\u57fa\u56e0\u5206\u7c7b\uff0cLCA\u7adf\u7136\u5305\u62ec\u81f3\u5c11\u5341\u4e03\u79cd\u4e9a\u578b\uff01\u800cEditas\u8fd9\u6b21\u7533\u8bf7\u7684\u4e34\u5e8a\u8bd5\u9a8c\u8fdb\u9488\u5bf9\u5176\u4e2d\u7b2c\u5341\u79cd\u4e9a\u578b\uff08LCA-10)\uff0c\u5360LCA\u768420%\u3002\u4e5f\u5c31\u662f\u8bf4\uff0c\u8fd9\u6b21\u4e34\u5e8a\u8bd5\u9a8c\u5b9e\u9645\u9488\u5bf9\u7684\uff0c\u662f\u4e00\u79cd\u53d1\u75c5\u7387\u4f4e\u81f3\u767e\u4e07\u5206\u4e4b\u51e0\u7684\u9057\u4f20\u75c5\uff0c\u5728\u6b27\u7f8e\u56fd\u5bb6\uff0c\u60a3\u8005\u603b\u6570\u53ef\u80fd\u4ec5\u6709\u6570\u5343\u4eba\u3002LCA-10\u7684\u81f4\u75c5\u57fa\u56e0\u53eb\u505aCEP290\u3002\u6211\u4eec\u4e4b\u6240\u4ee5\u6709\u89c6\u89c9\uff0c\u662f\u56e0\u4e3a\u773c\u775b\u4e2d\u6709\u611f\u5149\u7ec6\u80de\u3002\u611f\u5149\u7ec6\u80de\u957f\u51fa\u50cf\u5929\u7ebf\u4e00\u6837\u7684\u7ea4\u6bdb\uff0c\u7ea4\u6bdb\u4e2d\u6392\u6ee1\u4e86\u611f\u5149\u86cb\u767d\u3002\u5f53CEP290\u51fa\u73b0\u7f3a\u9677\u65f6\uff0c\u611f\u5149\u7ec6\u80de\u4e0d\u80fd\u5f62\u6210\u6b63\u5e38\u7684\u7ea4\u6bdb\uff0c\u63a2\u6d4b\u5149\u4fe1\u53f7\u7684\u80fd\u529b\u56e0\u6b64\u53d7\u635f\u3002<\/p>\n<p>\u79d1\u5b66\u5bb6\u6700\u521d\u662f\u600e\u4e48\u4ece\u4e24\u4e07\u4e2a\u57fa\u56e0\u4e2d\u63ea\u51faCEP290\u7684\u5462\uff1f\u5bf9\u7814\u7a76\u7f55\u89c1\u75c5\u6700\u6709\u5e2e\u52a9\u7684\uff0c\u662f\u90a3\u79cd\u660e\u663e\u8868\u73b0\u51fa\u5bb6\u65cf\u5185\u805a\u96c6\u6027\u7684\u60a3\u8005\u3002CEP290\u7a81\u53d8\u6b63\u662f\u4ece\u8fde\u7eed\u51fa\u73b0LCA\u7684\u9b41\u5317\u514b\u5bb6\u5ead\u91cc\u9274\u5b9a\u51fa\u6765\u7684\u3002\u5230\u8fd9\u4e00\u6b65\uff0cCEP290\u7a81\u53d8\u53ea\u80fd\u7b97\u5f97\u4e0a\u201c\u5acc\u72af\u201d\u3002\u4e0b\u4e00\u6b65\uff0c\u79d1\u5b66\u5bb6\u8fd8\u505a\u4e86\u4e00\u7cfb\u5217\u5b9e\u9a8c\uff0c\u8bc1\u660eCEP290\u7684\u7a81\u53d8\u7684\u786e\u53ef\u4ee5\u9020\u6210LCA\uff1a\u4e00\uff0c\u4ece\u60a3\u8005\u8eab\u4e0a\u5206\u79bb\u57f9\u517b\u89c6\u89c9\u7ec6\u80de\uff0c\u5e76\u8f6c\u5165\u6b63\u5e38\u7684CEP290\u57fa\u56e0\uff0c\u53d1\u73b0\u7ea4\u6bdb\u7684\u5f62\u6001\u6062\u590d\u6b63\u5e38\uff1b\u4e8c\uff0c\u7834\u574f\u5c0f\u9f20\u7684CEP290\u57fa\u56e0\uff0c\u89c2\u5bdf\u5230\u4e0e\u4eba\u7c7b\u60a3\u8005\u76f8\u4f3c\u7684\u75c7\u72b6\uff1b\u4e09\uff0c\u7528\u9488\u5bf9CEP290\u7684\u57fa\u56e0\u6280\u672f\u4fee\u590d\u5c0f\u9f20\u7684\u89c6\u529b\u3002\u81f3\u6b64\u6211\u4eec\u53ef\u4ee5\u5408\u7406\u63a8\u6d4b\uff0c\u5982\u679c\u80fd\u4fee\u590d\u4eba\u7c7b\u60a3\u8005\u7684CEP290\uff0c\u5c31\u6709\u53ef\u80fd\u6062\u590d\u4ed6\u4eec\u7684\u89c6\u529b\u3002<\/p>\n<p>\u975e\u4e13\u4e1a\u4eba\u58eb\u53ef\u80fd\u4f1a\u5bf9Editas\u7684\u65b0\u7597\u6cd5\u62b1\u6709\u5f88\u9ad8\u7684\u671f\u5f85\uff0c\u800c\u6709\u7ecf\u9a8c\u7684\u751f\u7269\u533b\u5b66\u5de5\u4f5c\u8005\u5728\u8fd9\u4e2a\u9636\u6bb5\u53ea\u4f1a\u8868\u793a\u201c\u8c28\u614e\u7684\u4e50\u89c2\u201d\u3002\u5176\u5b9e\u4efb\u4f55\u80fd\u8fdb\u5165\u4e34\u5e8a\u8bd5\u9a8c\u9636\u6bb5\u7684\u65b0\u7597\u6cd5\uff0c\u5176\u57fa\u672c\u539f\u7406\u5fc5\u7136\u5df2\u7ecf\u901a\u8fc7\u4e86\u8bba\u8bc1\uff0c\u7136\u800c\u5927\u90e8\u5206\u8bd5\u9a8c\u8fd8\u662f\u6b7b\u4e8e\u7ec6\u8282\u3002CRISPR\u867d\u7136\u662f\u6709\u53f2\u4ee5\u6765\u5a01\u529b\u6700\u5927\u7684\u57fa\u56e0\u7f16\u8f91\u5de5\u5177\uff0c\u4f46\u5176\u51c6\u786e\u6027\u548c\u6210\u529f\u7387\u8fdc\u6ca1\u6709\u63a5\u8fd1\u767e\u5206\u4e4b\u767e\u3002\u5bf9\u5b9e\u9a8c\u5ba4\u4e2d\u7684\u79d1\u5b66\u5bb6\uff0c\u8fd9\u5f88\u5bb9\u6613\u514b\u670d\uff1a\u5148\u7f16\u8f91\uff0c\u518d\u7b5b\u9009\uff0c\u7f16\u8f91\u9519\u8bef\u7684\u7a81\u53d8\u4f53\u76f4\u63a5\u9500\u6bc1\u3002CRISPR\u5728\u76ee\u6807\u57fa\u56e0\u4e4b\u5916\u8fd8\u53ef\u80fd\u9020\u6210\u989d\u5916\u7684\u7a81\u53d8\uff0c\u4e5f\u5c31\u662f\u6240\u8c13\u7684\u8131\u9776\u95ee\u9898\uff0c\u8fd9\u5bf9\u57fa\u7840\u7814\u7a76\u5e76\u4e0d\u662f\u4e25\u91cd\u969c\u788d\uff0c\u4f46\u5bf9\u4e34\u5e8a\u5e94\u7528\u5c31\u662f\u96be\u4ee5\u5bb9\u5fcd\u7684\u3002\u5728Editas\u7684\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\uff0cCRISPR\u7684\u4e24\u4e2a\u7ec4\u4ef6CAS\u548cgRNA\u9700\u8981\u7528\u6211\u4eec\u4e4b\u524d\u63d0\u8d77\u7684\u817a\u75c5\u6bd2\u8f7d\u4f53\u8fd0\u5165\u611f\u5149\u7ec6\u80de\u4e2d\u3002\u867d\u7136\u540c\u6837\u7528\u5230\u817a\u75c5\u6bd2\u8f7d\u4f53\u7684Luxturna\u5df2\u7ecf\u6210\u529f\uff0c\u4f46\u5177\u4f53\u5230\u6bcf\u4e00\u4e2a\u6cbb\u7597\u65b9\u6848\u4e2d\uff0c\u9700\u8981\u7684\u75c5\u6bd2\u8f7d\u4f53\u6570\u91cf\u53ef\u80fd\u662f\u4e0d\u540c\u7684\uff0c\u5bf9\u514d\u75ab\u53cd\u5e94\u7684\u6d4b\u8bd5\u5fc5\u987b\u53e6\u8d77\u7089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immune deficiency syndrome\uff0cAIDS\uff09\uff0c\u4e5f\u5c31\u662f\u6211\u4eec\u4eca\u5929\u719f\u77e5\u7684\u827e\u6ecb\u75c5\u3002 \u533b\u5b66\u5de5\u4f5c\u8005\u5f88\u5feb\u53d1\u73b0\u75c5\u56e0\u662f\u4e00\u79cd\u901a\u8fc7\u4f53\u6db2\u4f20\u64ad\u7684\u75c5\u6bd2\uff0c\u4efb\u4f55\u4eba\u90fd\u53ef\u80fd\u901a\u8fc7\u8f93\u8840\u3001\u6027\u4ea4\u3001\u5171\u7528\u9488\u5934\u3001\u6bcd\u5a74\u4f20\u64ad\u7b49\u65b9\u5f0f\u611f\u67d3\u8fd9\u79cd\u4e25\u91cd\u7684\u514d\u75ab\u75be\u75c5\u3002 \u8fd9\u79cd\u75be\u75c5\u5728\u516c\u4f17\u4e2d\u9020\u6210\u4e86\u6781\u5927\u7684\u6050\u614c\u3002\u6709\u4e24\u4e2a\u7fa4\u4f53\u906d\u53d7\u4e86\u6700\u6c89\u91cd\u7684\u6253\u51fb\uff1a\u9700\u8981\u8f93\u5165\u51dd\u8840\u56e0\u5b50\u7ef4\u6301\u751f\u547d\u7684\u8840\u53cb\u75c5\u60a3\u8005\uff1b\u96c6\u4e2d\u5728\u52a0\u5dde\u548c\u7ebd\u7ea6\u7684\u540c\u6027\u604b\u7fa4\u4f53\u3002<\/p>\n<p>\u524d\u9762\u8bf4\u8fc7\uff0c\u5bf9\u7406\u89e3\u4eba\u7c7b\u7684\u75be\u75c5\u5e2e\u52a9\u6700\u5927\u7684\uff0c\u5e38\u5e38\u662f\u643a\u5e26\u7f55\u89c1\u57fa\u56e0\u7684\u4e2a\u4f53\u30021994\u5e74\uff0c\u7ebd\u7ea6\u6d1b\u514b\u83f2\u52d2\u5927\u5b66\u827e\u4f26\u6234\u8499\u5fb7\u827e\u6ecb\u75c5\u7814\u7a76\u4e2d\u5fc3\uff08Aaron Diamond AIDS Research Center\uff09\u7684\u4e00\u4f4d\u5e74\u8f7b\u533b\u751fWilliam Paxton\u5192\u51fa\u4e86\u8fd9\u4e48\u4e00\u4e2a\u60f3\u6cd5\uff1a\u5982\u679c\u80fd\u627e\u5230\u9ad8\u5371\u7fa4\u4f53\u4e2d\u5177\u6709\u201c\u8d85\u7ea7\u62b5\u6297\u529b\u201d\u7684\u4e2a\u4f53\uff0c\u7814\u7a76\u4ed6\u4eec\u7684\u57fa\u56e0\u7ec4\uff0c\u6709\u6ca1\u6709\u53ef\u80fd\u66f4\u597d\u5730\u7406\u89e3\u827e\u6ecb\u75c5\u7684\u673a\u5236\u5462\uff1f\u5f53\u65f6\u7ebd\u7ea6\u6709\u4e00\u4f4d\u53ebStephen Crohn\u7684\u7537\u540c\u6027\u604b\uff0c\u4ece80\u5e74\u4ee3\u523090\u5e74\u4ee3\u521d\uff0c\u4ed6\u8eab\u8fb9\u7684\u670b\u53cb\uff0c\u5305\u62ec\u4ed6\u7684\u4f34\u4fa3\uff0c\u9646\u7eed\u88ab\u827e\u6ecb\u75c5\u593a\u53bb\u4e86\u751f\u547d\u3002\u7136\u800c 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