{"id":17742,"date":"2018-05-01T00:00:10","date_gmt":"2018-05-01T07:00:10","guid":{"rendered":"https:\/\/vermont.salk.edu\/?post_type=disclosure&#038;p=17742"},"modified":"2018-05-18T13:17:40","modified_gmt":"2018-05-18T20:17:40","slug":"single-injection-treats-hemophilia-b-for-life-in-proof-of-concept-study","status":"publish","type":"disclosure","link":"https:\/\/www.salk.edu\/zh\/news-release\/single-injection-treats-hemophilia-b-for-life-in-proof-of-concept-study\/","title":{"rendered":"\u5355\u6b21\u6ce8\u5c04\u6cbb\u6108B\u578b\u8840\u53cb\u75c5\uff0c\u6982\u5ff5\u9a8c\u8bc1\u7814\u7a76"},"content":{"rendered":"<p>\u62c9\u970d\u4e9a\u2014\u5bf9\u4e8e\u5927\u591a\u6570\u8840\u53cb\u75c5B\u60a3\u8005\u6765\u8bf4\uff0c\u4ed6\u4eec\u7684\u8eab\u4f53\u65e0\u6cd5\u6b63\u5e38\u5f62\u6210\u8840\u6813\uff0c\u56e0\u6b64\u9700\u8981\u6301\u7eed\u6ce8\u5c04\u6765\u8865\u5145\u51dd\u8840\u56e0\u5b50\uff0c\u8fd9\u5df2\u6210\u4e3a\u4ed6\u4eec\u751f\u6d3b\u7684\u4e00\u90e8\u5206\u3002\u4f46\u73b0\u5728\uff0c\u7d22\u5c14\u514b\u7814\u7a76\u6240\u7684\u7814\u7a76\u4eba\u5458\u5728\u5c0f\u9f20\u8eab\u4e0a\u8fdb\u884c\u7684\u5b9e\u9a8c\u8868\u660e\uff0c\u901a\u8fc7\u4e00\u6b21\u6027\u7684\u6ce8\u5c04\u542b\u6709\u80fd\u591f\u4ea7\u751f\u7f3a\u5931\u51dd\u8840\u56e0\u5b50\u7684\u65e0\u75c5\u809d\u7ec6\u80de\uff0c\u5c31\u53ef\u4ee5\u7ec8\u8eab\u6cbb\u7597\u8840\u53cb\u75c5B\u3002\u8fd9\u9879\u53d1\u73b0\u53d1\u8868\u5728\u300a <a href=\"#\" target=\"_blank\" rel=\"noopener\"><em>\u7ec6\u80de\u62a5\u544a<\/em><\/a> 2018\u5e745\u67081\u65e5\uff0c\u8fd9\u9879\u7814\u7a76\u53ef\u80fd\u5f7b\u5e95\u6539\u53d8\u8840\u53cb\u75c5B\u7684\u8bca\u65ad\u610f\u4e49\uff0c\u5e76\u4e3a\u6cbb\u7597\u5176\u4ed6\u76f8\u5173\u9057\u4f20\u6027\u75be\u75c5\u94fa\u5e73\u9053\u8def\u3002.<\/p>\n<figure id=\"attachment_17743\"  class=\"wp-caption alignright\"><a href=\"https:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction.jpg\"><img loading=\"lazy\" decoding=\"async\" width=\"458\" height=\"136\" class=\"img-responsive wp-image-17743 size-col-md-5\" src=\"https:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction-458x136.jpg\" alt=\"Left two panels: Hepatocyte like cells (HLCs) differentiated from the stem cells of patients with hemophilia B show very low levels of clotting factor IX, or FIX, shown in white. Right two panels: After treating these same cell lines with a gene correction tool, however, FIX increased to healthy levels.\" srcset=\"https:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction-458x136.jpg 458w, https:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction-300x89.jpg 300w, https:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction-768x229.jpg 768w, https:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction-1024x305.jpg 1024w, https:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction-147x44.jpg 147w, https:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction-585x174.jpg 585w, https:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction-553x165.jpg 553w, https:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction-750x223.jpg 750w, https:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction-767x228.jpg 767w, https:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction-945x281.jpg 945w, https:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction.jpg 1499w\" sizes=\"auto, (max-width: 458px) 100vw, 458px\" \/><\/a><figcaption class=\"wp-caption-text\">\u5de6\u4fa7\u4e24\u5e45\u56fe\uff1a\u6e90\u81ea\u8840\u53cb\u75c5B\u60a3\u8005\u5e72\u7ec6\u80de\u5206\u5316\u7684\u809d\u7ec6\u80de\u6837\u7ec6\u80de\uff08HLCs\uff09\u4ec5\u663e\u793a\u975e\u5e38\u4f4e\u6c34\u5e73\u7684\u51dd\u8840\u56e0\u5b50IX\uff08FIX\uff09\uff0c\u56fe\u4e2d\u4ee5\u767d\u8272\u6807\u793a\u3002\u53f3\u4fa7\u4e24\u5e45\u56fe\uff1a\u7528\u57fa\u56e0\u7ea0\u6b63\u5de5\u5177\u5904\u7406\u76f8\u540c\u7684\u7ec6\u80de\u7cfb\u540e\uff0cFIX\u6c34\u5e73\u5347\u9ad8\u81f3\u5065\u5eb7\u6c34\u5e73\u3002. <\/p>\n<p> <a href=\"https:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction.jpg\">\u70b9\u51fb\u6b64\u5904<\/a> \u7528\u4e8e\u9ad8\u5206\u8fa8\u7387\u56fe\u50cf\u3002. <\/p>\n<p> \u7248\u6743\uff1a\u8428\u514b\u7814\u7a76\u6240<\/figcaption><\/figure>\n<p>B\u578b\u8840\u53cb\u75c5\u662f\u7531\u7f16\u7801\u51dd\u8840\u56e0\u5b50IX\uff08FIX\uff09\u7684\u57fa\u56e0\u7f3a\u9677\u5f15\u8d77\u7684\u3002\u8840\u53cb\u75c5\u60a3\u8005\u53ef\u80fd\u4ea7\u751f\u8be5\u86cb\u767d\u7684\u91cf\u51cf\u5c11\uff0c\u6216\u8005\u5b8c\u5168\u7f3a\u4e4f\u529f\u80fd\u6027\u7248\u672c\uff0c\u5bfc\u81f4\u5371\u53ca\u751f\u547d\u7684\u8840\u6db2\u51dd\u56fa\u5ef6\u8fdf\u3002\u76ee\u524d\uff0c\u60a3\u8005\u7684\u6cbb\u7597\u65b9\u6cd5\u662f\u6ce8\u5c04\u2014\u2014\u6bcf\u5468\u53ef\u80fd\u591a\u8fbe\u6570\u6b21\u2014\u2014\u542b\u6709\u5728\u52a8\u7269\u7ec6\u80de\u4e2d\u5236\u9020\u7136\u540e\u7eaf\u5316\u7684FIX\u3002\u4f46\u8fd9\u79cd\u65b9\u6cd5\u6210\u672c\u9ad8\u6602\u3001\u8017\u65f6\uff0c\u5e76\u4e14\u968f\u7740\u65f6\u95f4\u7684\u63a8\u79fb\u6548\u679c\u53ef\u80fd\u4f1a\u51cf\u5f31\u3002.<\/p>\n<p>\u6700\u8fd1\uff0c\u7d22\u5c14\u514b\u7814\u7a76\u6240\u7684\u79d1\u5b66\u5bb6\u4eec\u5f00\u53d1\u4e86\u4e00\u79cd\u65b0\u65b9\u6cd5\uff0c\u7528 <a href=\"https:\/\/www.salk.edu\/zh\/news-release\/dont-kill-messenger-rna\/\">\u7f16\u7801 FIX \u57fa\u56e0\u7684\u4fe1\u4f7f RNA \u94fe<\/a>. \u7136\u800c\uff0c\u4e0e\u6807\u51c6\u6cbb\u7597\u4e00\u6837\uff0c\u8fd9\u9700\u8981\u6bcf\u6b21\u4fe1\u4f7f RNA \u6c34\u5e73\u4e0b\u964d\u65f6\u91cd\u590d\u6ce8\u5c04\u3002\u56e0\u6b64\uff0c\u79d1\u5b66\u5bb6\u4eec\u5e0c\u671b\u5c1d\u8bd5\u4e00\u79cd\u66f4\u6301\u4e45\u7684\u65b9\u6cd5\uff1a\u5c06\u80fd\u591f\u4ea7\u751f FIX \u7684\u5065\u5eb7\u809d\u7ec6\u80de\u79fb\u690d\u5230\u60a3\u8005\u4f53\u5185\u3002.<\/p>\n<p>\u201c\u57fa\u4e8e\u7ec6\u80de\u7684\u65b9\u6cd5\u7684\u5438\u5f15\u529b\u5728\u4e8e\uff0c\u60a8\u53ef\u4ee5\u6700\u5927\u9650\u5ea6\u5730\u51cf\u5c11\u60a3\u8005\u6240\u9700\u7684\u6cbb\u7597\u6b21\u6570\uff0c\u201dSalk \u524d\u7814\u7a76\u5458\u3001\u8be5\u8bba\u6587\u7b2c\u4e00\u4f5c\u8005 Suvasini Ramaswamy \u8bf4\u9053\u3002\u201c\u4e0e\u5176\u53cd\u590d\u6ce8\u5c04\uff0c\u4e0d\u5982\u4e00\u6b21\u5b8c\u6210\u3002\u201d<\/p>\n<p>\u7531\u4e8e\u6350\u8d60\u809d\u810f\u4f9b\u5e94\u77ed\u7f3a\uff0c\u7814\u7a76\u4eba\u5458\u8f6c\u800c\u91c7\u53d6\u4e86\u5e72\u7ec6\u80de\u7b56\u7565\u6765\u751f\u4ea7\u5065\u5eb7\u7684\u809d\u810f\u7ec6\u80de\u3002\u4ed6\u4eec\u6536\u96c6\u4e86\u60a3\u6709\u4e25\u91cdB\u578b\u8840\u53cb\u75c5\u7684\u4e24\u4f4d\u4eba\u7c7b\u60a3\u8005\u7684\u8840\u6db2\u6837\u672c\uff0c\u8fd9\u4e9b\u60a3\u8005\u65e0\u6cd5\u4ea7\u751fFIX\u3002\u7136\u540e\uff0c\u5728\u5b9e\u9a8c\u5ba4\u4e2d\uff0c\u4ed6\u4eec\u5c06\u8fd9\u4e9b\u7ec6\u80de\u91cd\u7f16\u7a0b\u4e3a\u8bf1\u5bfc\u591a\u80fd\u5e72\u7ec6\u80de\uff08iPSCs\uff09\uff0ciPSCs\u6709\u80fd\u529b\u5206\u5316\u6210\u8bb8\u591a\u5176\u4ed6\u7ec6\u80de\u7c7b\u578b\uff0c\u5305\u62ec\u809d\u810f\u7ec6\u80de\u3002\u63a5\u7740\uff0c\u4ed6\u4eec\u4f7f\u7528\u4e00\u79cd\u80fd\u591f\u6539\u53d8\u57fa\u56e0\u7684\u5de5\u5177CRISPR\/Cas9\uff0c\u4fee\u590d\u4e86\u6bcf\u4f4d\u60a3\u8005FIX\u57fa\u56e0\u4e2d\u7684\u7a81\u53d8\u3002\u6700\u540e\uff0c\u4ed6\u4eec\u8bf1\u5bfc\u8fd9\u4e9b\u4fee\u590d\u540e\u7684\u7ec6\u80de\u53d1\u80b2\u6210\u809d\u810f\u524d\u4f53\u7ec6\u80de\uff0c\u79f0\u4e3a\u7c7b\u809d\u7ec6\u80de\uff08HLCs\uff09\uff0c\u5e76\u5c06\u5b83\u4eec\u79fb\u690d\u5230\u60a3\u6709B\u578b\u8840\u53cb\u75c5\u7684\u5c0f\u9f20\u4f53\u5185\u3002.<\/p>\n<p>\u4e0e\u5176\u5bf9\u60a3\u6709\u8840\u53cb\u75c5\u7684 the mice \u8fdb\u884c\u624b\u672f\u2014\u2014\u8fd9\u662f\u4e00\u9879\u5177\u6709\u98ce\u9669\u7684\u64cd\u4f5c\uff0c\u56e0\u4e3a\u5b83\u4eec\u7684\u8840\u6db2\u6709\u65f6\u65e0\u6cd5\u51dd\u56fa\u2014\u2014\u8be5\u56e2\u961f\u901a\u8fc7\u813e\u810f\u79fb\u690d\u4e86 the HLCs\uff0c\u4ee5\u5c06\u7ec6\u80de\u5747\u5300\u5730\u5206\u5e03\u5728\u809d\u810f\u4e2d\u3002.<\/p>\n<p>\u65b0\u7684 HLC \u4e0d\u4ec5\u4ea7\u751f\u4e86 FIX\uff0c\u800c\u4e14\u4ea7\u751f\u4e86\u8db3\u591f\u7684\u86cb\u767d\u8d28\uff0c\u4f7f\u5c0f\u9f20\u80fd\u591f\u5f62\u6210\u6b63\u5e38\u7684\u8840\u6813\uff0c\u5e76\u4e14\u7ec6\u80de\u5728\u79fb\u690d\u540e\u81f3\u5c11\u4e00\u5e74\u7ee7\u7eed\u5b58\u6d3b\u5e76\u4ea7\u751f FIX\u3002.<\/p>\n<p>\u5bf9\u4e8e\u8840\u53cb\u75c5\u60a3\u8005\uff0c\u5229\u7528\u81ea\u8eab\u7ec6\u80de\u751f\u6210\u5065\u5eb7\u7684\u809d\u810f\u7ec6\u80de\uff08HLCs\uff09\uff0c\u7136\u540e\u5c06\u5176\u79fb\u690d\u56de\u4f53\u5185\uff0c\u53ef\u80fd\u6709\u52a9\u4e8e\u907f\u514d\u7ec6\u80de\u7597\u6cd5\u5e38\u4f34\u968f\u7684\u514d\u75ab\u5e76\u53d1\u75c7\u3002\u4f46\u8981\u5c06\u8fd9\u4e9b\u53d1\u73b0\u8f6c\u5316\u4e3a\u4e34\u5e8a\u5e94\u7528\uff0c\u4ecd\u9700\u66f4\u591a\u52aa\u529b\u3002.<\/p>\n<p>\u201c\u5728\u80fd\u591f\u8fdb\u5165\u4eba\u4f53\u4e4b\u524d\uff0c\u5f88\u591a\u4e8b\u60c5\u90fd\u5fc5\u987b\u53d1\u751f\uff0c\u201d\u62c9\u9a6c\u65af\u74e6\u7c73\u8bf4\u3002.<\/p>\n<p>\u5979\u8865\u5145\u8bf4\uff0c\u8fd9\u9879\u5de5\u4f5c\u5df2\u7ecf\u8bc1\u660e\u4e86\u7ed3\u5408\u5e72\u7ec6\u80de\u91cd\u7f16\u7a0b\u548c\u65b0\u7684\u57fa\u56e0\u6539\u826f\u65b9\u6cd5\u6765\u6cbb\u7597\u9057\u4f20\u6027\u75be\u75c5\u7684\u4ef7\u503c\u3002.<\/p>\n<p>\u8fd9\u9879\u5de5\u4f5c\u548c\u53c2\u4e0e\u7814\u7a76\u7684\u79d1\u7814\u4eba\u5458\u5f97\u5230\u4e86\u4ee5\u4e0b\u673a\u6784\u7684\u8d44\u52a9\uff1a\u7f8e\u56fd\u56fd\u7acb\u536b\u751f\u7814\u7a76\u9662\u3001\u7f8e\u56fd\u56fd\u5bb6\u764c\u75c7\u7814\u7a76\u6240\u3001\u7f8e\u56fd\u56fd\u5bb6\u795e\u7ecf\u75be\u75c5\u4e0e\u5352\u4e2d\u7814\u7a76\u6240\u3001\u97e6\u7279\u57fa\u91d1\u4f1a\u3001\u76ca\u666e\u751f\u516c\u53f8\u3001H.N.\u6350\u8d60\u53caFrances C.\u4f2f\u6770\u57fa\u91d1\u4f1a\u3001\u683c\u4f26\u8870\u8001\u7814\u7a76\u4e2d\u5fc3\u3001\u5229\u5965\u5a1c\u00b7M.\u548c\u54c8\u91cc\u00b7B.\u6d77\u59c6\u65af\u5229\u6148\u5584\u4fe1\u6258\u4ee5\u53ca\u52a0\u5dde\u518d\u751f\u533b\u5b66\u7814\u7a76\u6240\u3002.<\/p>","protected":false},"featured_media":0,"template":"","faculty":[115],"disease-research":[146],"class_list":["post-17742","disclosure","type-disclosure","status-publish","hentry","faculty-inder-verma","disease-research-aging-and-regenerative-medicine"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.3 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Single injection treats hemophilia B for life, in proof-of-concept study - Salk Institute for Biological Studies<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.salk.edu\/zh\/news-release\/single-injection-treats-hemophilia-b-for-life-in-proof-of-concept-study\/\" \/>\n<meta property=\"og:locale\" content=\"zh_CN\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Single injection treats hemophilia B for life, in proof-of-concept study - Salk Institute for Biological Studies\" \/>\n<meta property=\"og:description\" content=\"LA JOLLA\u2014For most people with hemophilia B, whose bodies can\u2019t properly form blood clots, constant injections to replenish their clotting factors are a way of life. But now, Salk researchers have demonstrated in mice that hemophilia B can be treated for life with one single injection containing disease-free liver cells that can produce their missing clotting factor. The finding, published in the journal Cell Reports on May 1, 2018, could drastically change what it means to be diagnosed with hemophilia B, and could pave the way toward similar treatments for other, related genetic disorders.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.salk.edu\/zh\/news-release\/single-injection-treats-hemophilia-b-for-life-in-proof-of-concept-study\/\" \/>\n<meta property=\"og:site_name\" content=\"Salk Institute for Biological Studies\" \/>\n<meta property=\"article:modified_time\" content=\"2018-05-18T20:17:40+00:00\" \/>\n<meta property=\"og:image\" content=\"http:\/\/www.salk.edu\/wp-content\/uploads\/2018\/04\/F9-before-and-after-correction-458x136.jpg\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data1\" content=\"3 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/www.salk.edu\\\/news-release\\\/single-injection-treats-hemophilia-b-for-life-in-proof-of-concept-study\\\/\",\"url\":\"https:\\\/\\\/www.salk.edu\\\/news-release\\\/single-injection-treats-hemophilia-b-for-life-in-proof-of-concept-study\\\/\",\"name\":\"Single injection treats hemophilia B for life, in proof-of-concept study - 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