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Goldberg\u535a\u58eb\u8aaa\uff0c\u4ed6\u5011\u7528\u5f9e\u5c4d\u9ad4\u4e0a\u63d0\u53d6\u7684\u89d2\u819c\u4f5c\u70ba\u201c\u6bcd\u201d\u7d30\u80de\u57f9\u990a\u7522\u751f\u8a31\u591a\u65b0\u7684\u5e74\u8f15\u7684\u7d30\u80de\u3002\u4ed6\u5011\u7684\u5275\u65b0\u6280\u8853\u662f\u4f7f\u7528\u76f4\u5f91\u53ea\u670950\u7d0d\u7c73\u7684\u78c1\u6027\u9846\u7c92\uff08\u8a3b\uff1a\u4eba\u985e\u982d\u9aee\u7684\u76f4\u5f91\u662f75000\u7d0d\u7c73\uff09\u628a\u65b0\u57f9\u990a\u7684\u7d30\u80de\u78c1\u5316\uff1b\u7528\u6ce8\u5c04\u5668\u628a\u9019\u4e9b\u78c1\u5316\u7684\u7d30\u80de\u6ce8\u5165\u773c\u775b\uff1b\u7136\u5f8c\uff0c\u901a\u904e\u5728\u773c\u7403\u5916\u7684\u8cbc\u7247\u4e0a\u65bd\u52a0\u96fb\u78c1\u529b\uff0c\u5c07\u7d30\u80de\u62c9\u5230\u773c\u7403\u4e2d\u592e\u7684\u89d2\u819c\u5f8c\u90e8\uff1b\u96a8\u5f8c\uff0c\u78c1\u6027\u9846\u7c92\u5f9e\u7d30\u80de\u4e0a\u812b\u843d\uff0c\u96a8\u60a3\u8005\u7684\u5c3f\u6db2\u6392\u51fa\u9ad4\u5916\u3002\u9019\u7a2e\u65b9\u6cd5\u63d0\u4f9b\u4e00\u7a2e\u4ee4\u4eba\u8208\u596e\u7684\u53ef\u80fd\u6027\uff0c\u5c31\u662f\u5f9e\u4e00\u500b\u4f9b\u9ad4\u7372\u5f97\u7684\u89d2\u819c\u53ef\u4ee5\u6cbb\u7642\u5e7e\u5341\u500b\u751a\u81f3\u5e7e\u767e\u500b\u60a3\u8005\uff0c\u800c\u4e14\u53ef\u671b\u6cbb\u7642\u5176\u5b83\u8996\u7db2\u819c\u548c\u8996\u795e\u7d93\u75be\u75c5\uff0c\u5305\u62ec\u9752\u5149\u773c\u3002 Stanford\u5927\u5b78\u7814\u7a76\u5c0f\u7d44\u7684\u79fb\u690d\u8207\u5176\u5b83\u89d2\u819c\u79fb\u690d\u624b\u8853\u4e0d\u540c\u4e4b\u8655\u662f\uff0c\u5b83\u4e26\u4e0d\u9700\u8981\u6350\u8d08\u8005\u548c\u63a5\u53d7\u8005\u4e4b\u9593\u5b8c\u7f8e\u7684\u201c\u5339\u914d\u201d\uff0c\u800c\u53ea\u9700\u8981\u5c40\u90e8\u6ef4\u773c\u6db2\u5c31\u53ef\u4ee5\u9810\u9632\u6392\u65a5\u53cd\u61c9\u3002Goldberg\u535a\u58eb\u8aaa\uff0c\u4e0d\u50c5\u624b\u8853\u662f\u5b89\u5168\u7684\uff0c\u800c\u4e14\u770b\u5230\u4e86\u521d\u6b65\u7642\u6548\u7684\u8de1\u8c61\u3002\u201c\u5c0d\u6b64\u6211\u5011\u611f\u5230\u975e\u5e38\u8208\u596e\uff0c\u6211\u5011\u6301\u8b39\u614e\u6a02\u89c0\u614b\u5ea6\u3002\u201d\u4ed6\u9810\u6e2c\uff0c\u9019\u7a2e\u65b9\u6cd5\u6700\u7d42\u5c07\u53d6\u4ee3\u5927\u7d0480%\u7684\u89d2\u819c\u79fb\u690d\u3002Stanford\u5927\u5b78\u7684\u9019\u9805\u7814\u7a76\u5f97\u5230\u4e86\u7f8e\u570b\u773c\u79d1\u5b78\u6703\uff08American Academy of Ophthalmology\uff09\u7684\u652f\u6301\u3002\u7f8e\u570b\u773c\u79d1\u5b78\u6703\u81e8\u5e8a\u767c\u8a00\u4ebaPhilip R. Rizzuto\u535a\u58eb\u8aaa\uff0c\u8a72\u5b78\u6703\u652f\u6301\u5275\u65b0\u7684\u81e8\u5e8a\u7814\u7a76\u3002\u201c\u5982\u679c\u9019\u9805\u65e9\u671f\u7814\u7a76\u88ab\u8b49\u660e\u662f\u5b89\u5168\u6709\u6548\u7684\uff0c\u90a3\u9ebc\u9019\u9805\u6280\u8853\u53ef\u80fd\u6703\u5e6b\u52a9\u4e00\u4e9b\u60a3\u8005\u907f\u514d\u89d2\u819c\u79fb\u690d\u3002\u201d \u57fa\u56e0\u7642\u6cd5\u5df2\u7d93\u670920\u591a\u5e74\u7684\u6b77\u53f2\u4e86\u3002\u4f46\u662f\uff0c1999\u5e74\u5728\u7d66\u60a3\u6709\u907a\u50b3\u6027\u809d\u75c5\u7684\u4e9e\u5229\u6851\u90a3\u5dde\u5c11\u5e74Jesse Gelsinger\u690d\u5165\u77ef\u6b63\u57fa\u56e0\u7684\u6642\u5019\uff0c\u60a3\u8005\u5c0d\u4f7f\u7528\u7684\u75c5\u6bd2\u7522\u751f\u4e86\u56b4\u91cd\u53cd\u61c9\uff0c\u5c0e\u81f4\u6b7b\u4ea1\u3002\u9019\u4f7f\u57fa\u56e0\u7642\u6cd5\u906d\u5230\u4e86\u91cd\u5927\u6253\u64ca\u3002\u73fe\u5728\u4f7f\u7528\u7684\u6539\u9032\u4e86\u7684\u57fa\u56e0\u7642\u6cd5\uff0c\u5373\u7a31\u70baCRISPR\u7684\u65b0\u6280\u8853\uff0c\u5c31\u50cf\u4e00\u628a\u986f\u5fae\u93e1\u4e0b\u7684\u624b\u8853\u5200\uff0c\u4ee5\u4eba\u5011\u96e3\u4ee5\u60f3\u8c61\u7684\u7cbe\u5ea6\u3001\u6548\u7387\u548c\u5b89\u5168\u6027\u9032\u884c\u57fa\u56e0\u91cd\u7d44\u624b\u8853\u3002 Stanford\u5927\u5b78\u753151\u6b72\u7684Matthew Porteus\u535a\u58eb\u9818\u5c0e\u7684\u5718\u968a\u767c\u73fe\uff0c\u4eba\u985e\u6709\u8d85\u904e10000\u7a2e\u770b\u4e0a\u53bb\u975e\u5e38\u4e0d\u540c\u7684\u75be\u75c5\u537b\u6709\u4e00\u500b\u5171\u540c\u9ede\u2014\u2014\u5b83\u5011\u90fd\u662f\u7531\u55ae\u4e00\u7d30\u80de\u985e\u578b\u7684\u55ae\u500b\u57fa\u56e0\u7684\u55ae\u500b\u7a81\u8b8a\u5f15\u8d77\u7684\u3002\u9019\u500b\u5718\u968a\u4f7f\u7528CRISPR-Cas9\u6280\u8853\uff0c\u5229\u7528Stanford\u6821\u5712\u88e1\u7684\u7b2c\u4e00\u500b\u7d30\u80de\u88fd\u9020\u5de5\u5ee0\u7684\u7522\u54c1\u9032\u884c\u201c\u57fa\u56e0\u7de8\u8f2f\u201d\uff0c\u5c31\u662f\u5c07\u58de\u7684\u57fa\u56e0\u5207\u9664\uff0c\u7136\u5f8c\u5f15\u5165\u4e00\u500b\u597d\u7684\u57fa\u56e0\u3002\u9019\u7a2e\u65b9\u6cd5\u5df2\u7d93\u5728\u767d\u9f20\u8eab\u4e0a\u6210\u529f\u6cbb\u7642\u4e86\u4e00\u4e9b\u8840\u6db2\u75c5\uff0c\u5982\u942e\u72c0\u7d30\u80de\u8ca7\u8840\uff08sickle cell anemia\uff09\u548c\u5730\u4e2d\u6d77\u8ca7\u8840\uff08beta thalassemia\uff09\u4ee5\u53ca\u808c\u71df\u990a\u4e0d\u826f\u7b49\u5e7e\u7a2e\u7834\u58de\u514d\u75ab\u7cfb\u7d71\u7684\u75be\u75c5\u3002 \u76ee\u524d\uff0c\u4ed6\u5011\u7684\u76ee\u6a19\u662f\u81e8\u5e8a\u8a66\u9a57\u6cbb\u7642\u5169\u7a2e\u56b4\u91cd\u7684\u8840\u6db2\u75be\u75c5\u2014\u2014\u942e\u72c0\u7d30\u80de\u8ca7\u8840\u548c\u5730\u4e2d\u6d77\u8ca7\u8840\u3002\u81e8\u5e8a\u8a66\u9a57\u5206\u70ba\u5169\u500b\u968e\u6bb5\uff0c\u7b2c\u4e00\u968e\u6bb5\u4e3b\u8981\u662f\u5b89\u5168\u6027\u8a66\u9a57\uff0c\u7b2c\u4e8c\u968e\u6bb5\u91cd\u9ede\u662f\u770b\u6cbb\u7642\u6548\u679c\u3002\u4ed6\u5011\u5f9e\u60a3\u8005\u7684\u9aa8\u9ad3\u4e2d\u63d0\u53d6\u51fa\u4e00\u4e9b\u60a3\u75c5\u7684\u5e79\u7d30\u80de\uff08stem cells\uff0c\u80fd\u5920\u7522\u751f\u8840\u6db2\u6216\u514d\u75ab\u7cfb\u7d71\u7d30\u80de\uff09\u3002\u7136\u5f8c\uff0c\u60a3\u8005\u5c07\u63a5\u53d7\u9ad8\u5f37\u5ea6\u5316\u7642\uff0c\u6bba\u6b7b\u5269\u9918\u7684\u60a3\u75c5\u5e79\u7d30\u80de\uff0c\u4e26\u628a\u65b0\u7684\u3001\u5065\u5eb7\u7684\u5e79\u7d30\u80de\u6ce8\u5165\u60a3\u8005\u9ad4\u5167\u3002\u4e00\u65e6\u5065\u5eb7\u7684\u5e79\u7d30\u80de\u5230\u4f4d\uff0c\u5c31\u6703\u7522\u751f\u66f4\u591a\u5065\u5eb7\u7684\u7d30\u80de\u4f86\u53d6\u4ee3\u751f\u75c5\u7684\u7d30\u80de\u2014\u2014\u9019\u6a23\u60a3\u8005\u5c31\u6703\u75ca\u7652\u3002\u6240\u4ee5\uff0c\u4e26\u4e0d\u662f\u6240\u6709\u7684\u58de\u7d30\u80de\u90fd\u5fc5\u9808\u4fee\u5fa9\u624d\u884c\u3002 Stanford\u5927\u5b78\u5e79\u7d30\u80de\u751f\u7269\u5b78\u548c\u518d\u751f\u91ab\u5b78\u7814\u7a76\u6240\uff08Institute for Stem Cell Biology and Regenerative Medicine\uff09\u526f\u6240\u9577Maria Grazia Roncarolo\u535a\u58eb\u8aaa\uff1a\u201c\u5982\u679c\u81e8\u5e8a\u8a66\u9a57\u6210\u529f\uff0c\u5c31\u70ba\u6cbb\u7642\u8a31\u591a\u514d\u75ab\u7cfb\u7d71\u548c\u8840\u6db2\u7cfb\u7d71\u75be\u75c5\u6253\u958b\u4e86\u5927\u9580\u3002\u91ab\u5b78\u754c\u90fd\u70ba\u9019\u6a23\u7684\u6cbb\u7642\u524d\u666f\u8208\u596e\u4e0d\u5df2\uff0c\u5c24\u5176\u662f\u5728\u9019\u7a2e\u65b0\u6280\u8853\u50f9\u683c\u731b\u8dcc\u3001\u53ef\u53ca\u7bc4\u570d\u64f4\u5927\u4e4b\u969b\u3002\u201d Stanford\u5927\u5b78\u91ab\u5b78\u9662\u9662\u9577Lloyd B. Minor\u535a\u58eb\u8aaa\uff1a\u201c\u9019\u4e9b\u9032\u5c55\u8aaa\u660e\u6211\u5011\u6b63\u5728\u9032\u5165\u91ab\u5b78\u7684\u4e00\u500b\u65b0\u6642\u4ee3\u3002\u201d \u00a0 \u8a18\u8005 \u6e6f\u6bc5\u5805","rel":"","context":"In &quot;\u5065\u5eb7\u990a\u751f&quot;","block_context":{"text":"\u5065\u5eb7\u990a\u751f","link":"https:\/\/newsforchinese.com\/v3\/category\/health\/"},"img":{"alt_text":"","src":"https:\/\/i0.wp.com\/newsforchinese.com\/v3\/wp-content\/uploads\/2019\/01\/Stanford%E5%AF%A6%E9%A9%97%E5%AE%A4%E9%87%8C%E4%BB%A4%E4%BA%BA%E8%88%88%E5%A5%AE%E7%9A%84%E4%BA%8B-small.jpg?resize=350%2C200&ssl=1","width":350,"height":200,"srcset":"https:\/\/i0.wp.com\/newsforchinese.com\/v3\/wp-content\/uploads\/2019\/01\/Stanford%E5%AF%A6%E9%A9%97%E5%AE%A4%E9%87%8C%E4%BB%A4%E4%BA%BA%E8%88%88%E5%A5%AE%E7%9A%84%E4%BA%8B-small.jpg?resize=350%2C200&ssl=1 1x, https:\/\/i0.wp.com\/newsforchinese.com\/v3\/wp-content\/uploads\/2019\/01\/Stanford%E5%AF%A6%E9%A9%97%E5%AE%A4%E9%87%8C%E4%BB%A4%E4%BA%BA%E8%88%88%E5%A5%AE%E7%9A%84%E4%BA%8B-small.jpg?resize=525%2C300&ssl=1 1.5x"},"classes":[]},{"id":141070,"url":"https:\/\/newsforchinese.com\/v3\/2017\/07\/12\/%e5%af%a6%e9%a9%97%e6%80%a7%e5%a2%9e%e5%bc%b7%e5%8a%91%e8%97%a5%e7%89%a9%e5%8f%af%e4%bf%83%e7%94%a8%e6%b2%bb%e7%99%82%e8%87%b4%e5%91%bd%e7%9a%84%e5%82%b3%e7%b5%b1%e7%99%82%e6%b3%95\/","url_meta":{"origin":143451,"position":4},"title":"\u5be6\u9a57\u6027\u201c\u589e\u5f37\u5291\u201d\u85e5\u7269\u53ef\u4fc3\u7528\u6cbb\u7642\u81f4\u547d\u7684\u50b3\u7d71\u7642\u6cd5","author":"Raymond","date":"July 12, 2017","format":false,"excerpt":"\u5be6\u9a57\u5ba4\u7814\u7a76\u63d0\u793a\uff0c\u4e00\u7a2e\u5df2\u7d93\u9032\u5165\u65e9\u671f\u81e8\u5e8a\u8a66\u9a57\u7684\u7528\u65bc\u6cbb\u7642\u591a\u7a2e\u6210\u5e74\u4eba\u764c\u75c7\u7684\u5be6\u9a57\u6027\u85e5\u7269\u53ef\u80fd\u589e\u5f37\u5c0d\u5169\u7a2e\u76ee\u524d\u57fa\u672c\u4e0a\u7e3d\u662f\u81f4\u547d\u7684\u7ae5\u5e74\u671f\u8166\u764c\u7684\u653e\u7642\u548c\u5316\u7642\u6548\u679c\u3002 \u5728\u95dc\u65bc\u7f8e\u570b\u7d04\u7ff0\u00b7\u970d\u666e\u91d1\u65af\u57fa\u6885\u723e\u764c\u75c7\u4e2d\u5fc3\u7684\u79d1\u7814\u4eba\u54e1\u9032\u884c\u7684\u5169\u9805\u7814\u7a76\u7684\u5831\u544a\u4e2d\uff0c\u9019\u7a2e\u7a31\u70baTAK228\u7684\u85e5\u7269\u6291\u5236\u4e86\u5728\u5be6\u9a57\u5ba4\u57f9\u990a\u7684\u4eba\u985e\u764c\u7d30\u80de\u7684\u751f\u9577\uff0c\u4e26\u4e14\u986f\u8457\u5ef6\u9577\u4e86\u79fb\u690d\u4e86\u4f86\u81ea\u7030\u6f2b\u6027\u771f\u6027\u8166\u6a4b\u795e\u7d93\u81a0\u8cea\u7624\uff08DIPG\uff09\u548c\u975e\u5178\u578b\u7578\u80ce\u7624\u6a23\/\u6a6b\u7d0b\u808c\u6a23\u7624\uff08AT\/RTs\uff09\u9019\u5169\u7a2e\u764c\u75c7\u7684\u7d30\u80de\u7684\u5c0f\u9f20\u7684\u58fd\u547d\u3002 \u7030\u6f2b\u6027\u771f\u6027\u8166\u6a4b\u795e\u7d93\u81a0\u8cea\u7624\uff08DIPG\uff09\u2014\u2014\u9019\u662f\u4e00\u7a2e\u8166\u5e79\u7684\u764c\u75c7\uff0c\u6bcf\u5e74\u5f71\u97ff\u5168\u4e16\u754c\u5927\u7d043000\u540d\u5152\u7ae5\u2014\u2014\u5373\u4fbf\u4f7f\u7528\u5305\u62ec\u5916\u79d1\u624b\u8853\u3001\u5316\u7642\u548c\u653e\u7642\u5728\u5167\u7684\u73fe\u6709\u53ef\u7528\u7684\u7642\u6cd5\u52a0\u4ee5\u6cbb\u7642\uff0c\u4e5f\u662f\u666e\u904d\u81f4\u547d\u7684\u3002\u6700\u5e38\u898b\u7684\u65b0\u751f\u5152\u8166\u764c\u2014\u2014\u975e\u5178\u578b\u7578\u80ce\u7624\u6a23\/\u6a6b\u7d0b\u808c\u6a23\u7624\uff08AT\/RTs\uff09\u6709\u985e\u4f3c\u7684\u4e0d\u826f\u9810\u5f8c\uff0c\u591a\u6578\u75c5\u4eba\u5728\u8a3a\u65b7\u5f8c\u5b58\u6d3b6\u523011\u500b\u6708\u3002 \u7531\u7d04\u7ff0\u00b7\u970d\u666e\u91d1\u65af\u57fa\u6885\u723e\u764c\u75c7\u4e2d\u5fc3\u7684\u816b\u7624\u5b78\u52a9\u7406\u6559\u6388\u3001\u75c5\u7406\u5b78\u8b1b\u5e2b\u3001\u91ab\u5b78\u535a\u58eb\u3001\u7406\u5b78\u535a\u58ebEric H. 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