{"id":379703,"date":"2025-11-07T08:00:00","date_gmt":"2025-11-07T00:00:00","guid":{"rendered":"https:\/\/pingtungtimes.com.tw\/?p=379703"},"modified":"2025-11-07T08:00:00","modified_gmt":"2025-11-07T00:00:00","slug":"%e9%87%8c%e7%a8%8b%e7%a2%91%e7%aa%81%e7%a0%b4%ef%bc%81%e5%85%a8%e7%90%83%e9%a6%96%e5%80%8bapoc3%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e8%87%a8%e5%ba%8a%e6%b2%bb%e7%99%82%e9%ab%98%e8%a1%80%e8%84%82","status":"publish","type":"post","link":"https:\/\/pingtungtimes.com.tw\/?p=379703","title":{"rendered":"\u91cc\u7a0b\u7891\u7a81\u7834\uff01\u5168\u7403\u9996\u500bAPOC3\u57fa\u56e0\u7de8\u8f2f\u81e8\u5e8a\u6cbb\u7642\u9ad8\u8840\u8102\u7372\u5f97\u6210\u529f"},"content":{"rendered":"<div>\n<p class=\"prntac\"><b>\u6b63\u5e8f\u751f\u7269<\/b><b>CS-121<\/b><b>\u9996\u4f8b\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u60a3\u8005\u5b8c\u6210\u7d66\u85e5\uff0c\u986f\u793a\u826f\u597d\u5b89\u5168\u6027\u53ca<\/b><b>\u986f\u8457\u7642\u6548<\/b><\/p>\n<p><span class=\"legendSpanClass\">\u4e0a\u6d77<\/span><span class=\"legendSpanClass\">2025\u5e7411\u67087\u65e5<\/span> \/\u7f8e\u901a\u793e\/ &#8212; \u7576\u5730\u6642\u95932025\u5e7411\u67086\u65e5\uff0c\u5c08\u6ce8\u65bc\u65b0\u578b\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u3001\u8655\u65bc\u81e8\u5e8a\u968e\u6bb5\u7684\u5275\u65b0\u751f\u7269\u91ab\u85e5\u79d1\u6280\u4f01\u696d\u6b63\u5e8f\u751f\u7269\uff08\u4e0a\u6d77\uff09\u5ba3\u4f48\uff0c\u8207\u5b89\u5fbd\u91ab\u79d1\u5927\u5b78\u7b2c\u4e00\u9644\u5c6c\u91ab\u9662\u5408\u4f5c\u958b\u5c55\u7684\u91dd\u5c0d<b>\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7<\/b><b>\/<\/b><b>\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7<\/b>\u7684\u9776\u5411<i>APOC3<\/i>\u7684\u9e7c\u57fa\u7de8\u8f2f\u85e5\u7269CS-121\u6ce8\u5c04\u6db2\u7684\u7814\u7a76\u8005\u767c\u8d77\u7684\u81e8\u5e8a\u7814\u7a76\uff08Investigator Initiated Trial, IIT\uff09\u6cbb\u7642\u7684\u9996\u4f4d\u60a3\u8005\u5df2\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662\u3002\u60a3\u8005\u60a3\u6709\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\uff0c\u6b77\u53f2\u7a7a\u8179\u7518\u6cb9\u4e09\u916f\uff08TG\uff09\u6c34\u6e96&gt;12.5 mmol\/L\uff0c\u591a\u6b21\u8a98\u767c\u6025\u6027\u80f0\u817a\u708e\u3002\u6b64\u6b21IIT\u8a66\u9a57\u70ba\u5291\u91cf\u722c\u5761\u8a66\u9a57\uff0c\u60a3\u8005\u5728\u55ae\u6b21\u6700\u4f4e\u5291\u91cf\u7d66\u85e5\u5f8c\u4e09\u5929\u5167\uff0c\u7a7a\u8179TG\u6c34\u6e96\u986f\u8457\u4e0b\u964d\uff0c\u4e14\u672a\u767c\u751f\u4e0d\u826f\u53cd\u61c9\u3002\u9019\u662f\u5168\u7403\u9996\u6b21\u901a\u904e\u9776\u5411\u9e7c\u57fa\u7de8\u8f2f<i>APOC3<\/i>\u57fa\u56e0\u6cbb\u7642\u9ad8\u8840\u8102\u60a3\u8005\u7684\u6848\u4f8b\u3002<\/p>\n<div class=\"PRN_ImbeddedAssetReference\" id=\"DivAssetPlaceHolder3861\">\n<p style=\"TEXT-ALIGN: center; WIDTH: 100%\"><span>\u5168\u7403\u9996\u4f8b\u63a5\u53d7APOC3\u57fa\u56e0\u7de8\u8f2f\u6cbb\u7642\uff08\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\uff09\u7684\u9ad8\u8840\u8102\u60a3\u8005\uff08\u53f35\uff09\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662<\/span><\/p>\n<\/p><\/div>\n<p><span id=\"spanHghltc90f\">\u5168\u7403\u9996\u4f8b\u63a5\u53d7<i>APOC3<\/i>\u57fa\u56e0\u7de8\u8f2f\u6cbb\u7642\uff08\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\uff09\u7684\u9ad8\u8840\u8102\u60a3\u8005\uff08\u53f35\uff09\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662<\/span><\/p>\n<p>\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u662f\u4e00\u7a2e\u56e0\u8840\u6db2\u4e2d\u4e73\u7cdc\u5fae\u7c92\u7570\u5e38\u5347\u9ad8\u5c0e\u81f4\u7684\u4ee3\u8b1d\u6027\u75be\u75c5\uff0c\u5e38\u8207\u8102\u8cea\u4ee3\u8b1d\u7d0a\u4e82\u76f8\u95dc\uff0c\u4e3b\u8981\u8868\u73fe\u70ba\u7a7a\u8179\u7518\u6cb9\u4e09\u916f\uff08TG\uff09\u6c34\u6e96\u6975\u5ea6\u5347\u9ad8\uff0c\u53ef\u80fd\u5f15\u767c\u4e0d\u53ef\u9810\u6e2c\u4e14\u6f5b\u5728\u81f4\u547d\u7684\u6025\u6027\u80f0\u817a\u708e\u7b49\u56b4\u91cd\u4f75\u767c\u75c7\uff0c\u662f<b>\u56b4\u91cd\u9ad8\u7518\u6cb9\u4e09\u8102\u8840\u75c7\uff08<\/b><b>Severe Hypertriglyceridemia<\/b><b>\uff0c<\/b><b>sHTG<\/b><b>\uff09<\/b>\u6700\u56b4\u91cd\u7684\u4e00\u7a2e\u4e9e\u578b\u3002\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u5305\u542b<b>\u5bb6\u65cf\u6027\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7d9c\u5408\u5f81\uff08<\/b><b>Familial Chylomicronemia Syndrome<\/b><b>\uff0c<\/b><b>FCS<\/b><b>\uff09<\/b>\u548c<b>\u591a\u56e0\u7d20\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7d9c\u5408\u5f81\uff08<\/b><b>Multifactorial Chylomicronemia Syndrome<\/b><b>\uff0c<\/b><b>MCS<\/b><b>\uff09<\/b>\u3002\u5176\u4e2d<b>FCS<\/b>\u662f\u4e00\u7a2e\u7f55\u898b\u7684\u5e38\u67d3\u8272\u9ad4\u96b1\u6027\u907a\u50b3\u75c5\uff0c\u7531\u65bc\u8102\u86cb\u767d\u8102\u80aa\u9176\uff08Lipoprotein lipase\uff0cLPL\uff09\u57fa\u56e0\u6216\u53c3\u8207\u8abf\u63a7LPL\u6d3b\u6027\u7684\u95dc\u9375\u57fa\u56e0\u767c\u751f<b>\u96d9\u7b49\u4f4d\u57fa\u56e0\u7a81\u8b8a<\/b>\uff0c\u5c0e\u81f4\u8102\u86cb\u767d\u8102\u80aa\u9176\u529f\u80fd\u53d7\u640d\uff0c\u4f7f\u7518\u6cb9\u4e09\u8102\u7684\u5206\u89e3\u4ee3\u8b1d\u7522\u751f\u969c\u7919\uff0c\u5f9e\u800c\u9020\u6210\u9ad4\u5167\u4e73\u7cdc\u5fae\u7c92\u7684\u84c4\u7a4d\uff0c\u901a\u5e38\u60a3\u8005\u7684\u7a7a\u8179TG\u6c34\u6e96\u226510 mmol\/L (885 mg\/dL)\uff0c\u5168\u7403\u767c\u75c5\u7387\u5728\u5341\u842c\u5206\u4e4b\u4e00\u5230\u767e\u842c\u5206\u4e4b\u4e00\u4e4b\u9593\uff1b\u800c<b>MCS<\/b>\u5247\u7531<b>\u591a\u7a2e\u56e0\u7d20<\/b>\u5f15\u8d77\u7684\u75c5\u7406\u72c0\u614b\uff0c\u5176\u767c\u75c5\u53d7\u907a\u50b3\u80cc\u666f\u3001\u751f\u6d3b\u7fd2\u6163\u6216\u5176\u4ed6\u4ee3\u8b1d\u75be\u75c5\u5171\u540c\u5f71\u97ff\uff0c\u5168\u7403\u767c\u75c5\u7387\u9ad8\u9054<b>1\/600<\/b>\u3002<\/p>\n<p>\u76ee\u524d\uff0c\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7684\u4e3b\u8981\u6cbb\u7642\u7b56\u7565\u662f\u5c07\u60a3\u8005\u7684\u7a7a\u8179\u7518\u6cb9\u4e09\u8102\uff08TG\uff09\u6c34\u6e96\u63a7\u5236\u5728\u6025\u6027\u80f0\u817a\u708e\u98a8\u96aa\u95be\u503c\u4ee5\u4e0b\uff08\u901a\u5e38\u9700&lt;5.7 mmol\/L\uff0c\u5373\u00a0500 mg\/dL\uff09\uff0c\u7136\u800c\u76ee\u524d\u5df2\u4e0a\u5e02\u7684\u5404\u985e\u964d\u7518\u6cb9\u4e09\u8102\u7684\u85e5\u7269\u5c0d\u9019\u985e\u60a3\u8005\u7684\u6548\u679c\u5fae\u4e4e\u5176\u5fae\uff0c\u60a3\u8005\u4ecd\u7136\u9762\u81e8\u60a3\u6025\u6027\u80f0\u817a\u708e\u7b49\u4f75\u767c\u75c7\u7684\u98a8\u96aa\uff1b\u56b4\u683c\u7684\u98f2\u98df\u63a7\u5236\u5118\u7ba1\u53ef\u4ee5\u901a\u904e\u6bcf\u65e5\u6975\u4f4e\u8102\u80aa\u7684\u98f2\u98df\u4f86\u5be6\u73fe\u4e73\u7cdc\u5fae\u7c92\u7684\u964d\u4f4e\uff0c\u4f46\u662f\u60a3\u8005\u5f80\u5f80\u5f88\u96e3\u9577\u671f\u5805\u6301\uff0c\u4f9d\u7136\u6301\u7e8c\u9762\u81e8\u56b4\u91cd\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u5e36\u4f86\u7684\u98a8\u96aa\u3002\u7814\u7a76\u767c\u73fe\uff0c\u4e00\u7a2e\u4e3b\u8981\u5728\u809d\u81df\u4e2d\u7522\u751f\u7684\u86cb\u767d\u8cea<b>APOC3<\/b>\u5728\u8abf\u7bc0\u8840\u6f3f\u7518\u6cb9\u4e09\u916f\u65b9\u9762\u767c\u63ee\u8457\u91cd\u8981\u4f5c\u7528\uff0c\u5c0d\u5927\u898f\u6a21\u4eba\u7fa4\u7684\u6aa2\u6e2c\u7814\u7a76\u767c\u73fe\uff0c\u651c\u5e36\u5929\u7136<b>APOC3<\/b><b>\u529f\u80fd\u7f3a\u5931\u7a81\u8b8a<\/b>\u7684\u4eba\u7fa4\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u542b\u91cf\u8f03\u4f4e\u4e14\u4e0d\u8868\u73fe\u5176\u4ed6\u81e8\u5e8a\u75c7\u72c0\u3002\u96a8\u8457\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u7684\u767c\u5c55\uff0c\u901a\u904e\u5728\u57fa\u56e0\u5c64\u9762<b>\u8abf\u63a7<\/b><b>APOC3<\/b><b>\u7684\u8868\u9054\u5f9e\u800c\u964d\u4f4e\u7518\u6cb9\u4e09\u916f\u6c34\u6e96<\/b>\u7684\u6cbb\u7642\u7b56\u7565\u5f97\u4ee5\u5be6\u73fe\uff0c\u9019\u5c07\u70ba\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u60a3\u8005\u5e36\u4f86\u6cbb\u7652\u7684\u5e0c\u671b\u3002<\/p>\n<p>\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\u662f\u5229\u7528\u4e0a\u6d77\u79d1\u6280\u5927\u5b78\u81ea\u4e3b\u7814\u767c\u7684<b>\u9ad8\u7cbe\u51c6\u8b8a\u5f62\u5f0f\u9e7c\u57fa\u7de8\u8f2f\u5668<\/b><b>tBE<\/b><b>\uff08<\/b><b>transformer Base Editor<\/b><b>\uff09\uff08<\/b><b>Wang <i>et al.<\/i><\/b><b>\uff0c<\/b><b><i>Nat Cell Biol<\/i><\/b><b>\uff0c<\/b><b>2021<\/b><b>\uff09<\/b>\u958b\u767c\u7684\u91dd\u5c0d<b>\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7<\/b><b>\/<\/b><b>\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7<\/b>\u7684<b>\u9996\u6b3e\u9ad4\u5167\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269<\/b>\u3002tBE\u7531\u8102\u8cea\u7d0d\u7c73\u7c92\uff08Lipid Nanoparticle\uff0cLNP\uff09\u5305\u88dd\uff0c\u901a\u904e\u975c\u8108\u6ce8\u5c04\u7d66\u85e5\u905e\u9001\u81f3\u60a3\u8005\u809d\u81df\uff0c\u5c0d\u9776\u6a19\u57fa\u56e0<i>APOC3<\/i>\u9032\u884c\u7cbe\u51c6\u9e7c\u57fa\u7de8\u8f2f\uff0c\u6a21\u64ec\u5929\u7136APOC3\u529f\u80fd\u7f3a\u5931\u4eba\u7fa4\u4e2d\u7684\u6709\u76ca\u7a81\u8b8a\uff0c\u4e0b\u8abfAPOC3\u7684\u57fa\u56e0\u8868\u9054\uff0c\u9032\u800c\u9054\u5230\u964d\u4f4e\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u7684\u76ee\u7684\u3002\u901a\u904e\u57fa\u56e0\u6c34\u6e96\u7684\u7de8\u8f2f\uff0c\u8a72\u7642\u6cd5\u6709\u671b\u5be6\u73fe&#8221;\u4e00\u6b21\u7d66\u85e5\uff0c\u7d42\u8eab\u6709\u6548&#8221;\u7684\u964d\u7518\u6cb9\u4e09\u8102\u7684\u76ee\u6a19\u3002tBE\u5c6c\u65bc\u4e0b\u4e00\u4ee3\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\uff0c\u4e0d\u9700\u8981\u7834\u58deDNA\u96d9\u93c8\u5373\u53ef\u5be6\u73fe\u5c0d\u55ae\u9e7c\u57fa\u7a81\u8b8a\u9032\u884c\u7cbe\u51c6\u6821\u6b63\uff0c\u4e0d\u6703\u4fc3\u767cp53\u901a\u8def\u555f\u52d5\u548c\u67d3\u8272\u9ad4\u7570\u5e38\u7b49\u5b89\u5168\u98a8\u96aa\u3002\u76f8\u6bd4\u8f03\u5176\u4ed6\u57fa\u65bcCRISPR\u6280\u8853\u7684\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5\uff0cCS-121\u5b89\u5168\u6027\u66f4\u9ad8\uff0c\u4e0d\u6703\u5f15\u767c\u60a3\u8005\u57fa\u56e0\u7d44DNA\u5927\u7247\u6bb5\u7f3a\u5931\u3001\u67d3\u8272\u9ad4\u7a81\u8b8a\u3001\u812b\u9776\u7a81\u8b8a\u7b49\u98a8\u96aa\uff0c\u66f4\u4e0d\u6703\u5e36\u4f86\u7531CRISPR\/Cas9\u5207\u65b7DNA\u96d9\u93c8\u800c\u555f\u52d5p53\u901a\u8def\u5c0e\u81f4\u7684\u809d\u6bd2\u6027\u7b49\u526f\u53cd\u61c9\u3002\u5728\u524d\u671f\u7684\u52d5\u7269\u8a66\u9a57\u4e2d\uff0cCS-121\u7684\u5b89\u5168\u6027\u548c\u9577\u671f\u6709\u6548\u6027\u5df2\u7d93\u7372\u5f97\u9a57\u8b49\uff0c\u5728\u809d\u81df\u3001\u80ba\u3001\u808c\u8089\u3001\u813e\u81df\u3001\u5375\u5de2\u3001\u5fc3\u81df\u3001\u814e\u81df\u7b49\u5404\u7a2e\u7d44\u7e54\u4e2d\u5747\u6c92\u6709\u6aa2\u6e2c\u5230\u812b\u9776\u7de8\u8f2f\u3002<\/p>\n<p>\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\u6cbb\u7642\u7684\u9996\u4f4d\u60a3\u8005\u662f\u4e00\u540d\u60a3\u6709\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u768463\u6b72\u7537\u6027\u3002\u60a3\u8005\u57282025\u5e7410\u670818\u65e5\u63a5\u53d7CS-121\u6700\u4f4e\u5291\u91cf\u55ae\u6b21\u7d66\u85e5\uff0c\u7d66\u85e5\u5f8c\u4e09\u5929\u5167\uff0c\u7a7a\u8179TG\u6c34\u6e96\u986f\u8457\u4e0b\u964d\uff0c\u4e26\u65bc\u7d66\u85e5\u5f8c\u7b2c3\u5929\u9806\u5229\u51fa\u9662\u3002\u622a\u81f3\u5831\u5c0e\u65e5\u671f\uff0c\u672a\u51fa\u73fe\u7522\u54c1\u76f8\u95dc\u7684\u4e0d\u826f\u4e8b\u4ef6\u3002<\/p>\n<p>\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2IIT\u5c08\u6848\u7684\u4e3b\u8981\u7814\u7a76\u8005\u70ba\u5b89\u5fbd\u91ab\u79d1\u5927\u5b78\u7b2c\u4e00\u9644\u5c6c\u91ab\u9662\u5468\u7165\u6559\u6388\uff0c\u5433\u5fd7\u9e97\u4e3b\u4efb\u3002<\/p>\n<p>\u6b63\u5e8f\u751f\u7269\u662f\u4e00\u5bb6\u5df2\u7d93\u9032\u5165IND\u81e8\u5e8a\u968e\u6bb5\u7684\u5275\u65b0\u751f\u7269\u91ab\u85e5\u79d1\u6280\u516c\u53f8\uff0c\u5176\u958b\u767c\u7684\u9996\u6b3e\u9ad4\u5916\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269CS-101\u6ce8\u5c04\u6db2\u5df2\u7d93\u6210\u529f\u6cbb\u7652\u6578\u5341\u4f8b\u03b2-\u5730\u4e2d\u6d77\u8ca7\u8840\u548c\u942e\u5200\u578b\u7d30\u80de\u8ca7\u8840\u75c5\u60a3\u8005\u3002\u6b63\u5e8f\u751f\u7269\u6b63\u5728\u52a0\u901f\u63a8\u9032\u9996\u6b3e\u9ad4\u5167\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269CS-121\u6ce8\u5c04\u6db2\u9032\u5165IND\u81e8\u5e8a\u53ca\u4e0a\u5e02\uff0c\u6709\u671b\u70ba\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7b49\u4ee3\u8b1d\u985e\u75be\u75c5\u60a3\u8005\u5e36\u4f86&#8221;<b>\u4e00\u6b21\u6cbb\u7642\uff0c\u7d42\u8eab\u6709\u6548<\/b>&#8220;\u7684\u985b\u8986\u6027\u6cbb\u7642\u65b9\u6848\u3002<\/p>\n<p class=\"prntac\">\uff08\u7279\u5225\u611f\u8b1d\u5b89\u5fbd\u91ab\u79d1\u5927\u5b78\u7b2c\u4e00\u9644\u5c6c\u91ab\u9662\u3001\u4e0a\u6d77\u79d1\u6280\u5927\u5b78\u53ca\u4e0a\u6d77\u81e8\u5e8a\u7814\u7a76\u4e2d\u5fc3\uff09<\/p>\n<p><b>\u95dc\u65bc\u6b63\u5e8f\u751f\u7269<\/b><\/p>\n<p>\u6b63\u5e8f\u751f\u7269\uff08CorrectSequence Therapeutics\uff09\u662f\u4e00\u5bb6\u5c08\u6ce8\u65bc\u65b0\u578b\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u3001\u8655\u65bc\u81e8\u5e8a\u968e\u6bb5\u7684\u751f\u7269\u91ab\u85e5\u79d1\u6280\u516c\u53f8\uff0c\u5b75\u5316\u81ea\u4e0a\u6d77\u79d1\u6280\u5927\u5b78\uff0c\u81f4\u529b\u65bc\u5229\u7528\u81ea\u4e3b\u77e5\u8b58\u7522\u6b0a\u7684\u9e7c\u57fa\u7de8\u8f2f\u9ad4\u7cfb\uff0c\u958b\u767c\u7a81\u7834\u6027\u7cbe\u51c6\u7642\u6cd5\uff0c\u9020\u798f\u4eba\u985e\u5065\u5eb7\u3002\u516c\u53f8\u57fa\u65bc\u4ee5\u8b8a\u5f62\u5f0f\u9e7c\u57fa\u7de8\u8f2f\u5668tBE\uff08transformer Base Editor\uff09\u70ba\u4ee3\u8868\u7684\u81ea\u4e3bIP\u9e7c\u57fa\u7de8\u8f2f\u7cfb\u7d71\u5275\u5efa\u4e86\u591a\u7a2e\u7cbe\u51c6\u7642\u6cd5\uff0c\u91dd\u5c0d\u907a\u50b3\u75be\u75c5\u3001\u4ee3\u8b1d\u75be\u75c5\u3001\u5fc3\u8840\u7ba1\u75be\u75c5\u7b49\u4f48\u5c40\u4e86\u591a\u689d\u7ba1\u7dda\uff0c\u9996\u689d\u7ba1\u7dda\u5373\u5c07\u9032\u5165\u95dc\u9375\u6027\u81e8\u5e8a\u8a66\u9a57\uff08\u2161\/\u2162\u671f\u81e8\u5e8a\u8a66\u9a57\uff09\u968e\u6bb5\u3002<\/p>\n<p>\u6b32\u77ad\u89e3\u66f4\u591a\u8cc7\u8a0a\uff0c\u8acb\u9ede\u64ca\u9032\u5165\u5b98\u7db2\u00a0<a href=\"http:\/\/www.correctsequence.com\/\" target=\"_blank\" rel=\"nofollow\" style=\"color: #0000FF\">www.correctsequence.com<\/a><\/p>\n<p><b>\u5a92\u9ad4\u806f\u7cfb\u4eba\uff1a<\/b><br \/>\u5546\u52d9\u5408\u4f5c\uff1a<a href=\"mailto:BD@correctsequence.com\" target=\"_blank\" rel=\"nofollow\" style=\"color: #0000FF\">BD@correctsequence.com<\/a><br \/>\u81e8\u5e8a\u8a66\u9a57\uff1a<a href=\"mailto:CT@correctsequence.com\" target=\"_blank\" rel=\"nofollow\" style=\"color: #0000FF\">CT@correctsequence.com<\/a><\/p>\n<div class=\"PRN_ImbeddedAssetReference\" id=\"DivAssetPlaceHolder0\">  <\/div>\n<\/div>\n","protected":false},"excerpt":{"rendered":"<p>\u6b63\u5e8f\u751f\u7269CS-121\u9996\u4f8b\u9ad8\u4e73\u7cdc&#8230;<\/p>\n","protected":false},"author":16,"featured_media":379704,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"fifu_image_url":"","fifu_image_alt":"","footnotes":""},"categories":[40567],"tags":[],"class_list":["post-379703","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-40567"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>\u91cc\u7a0b\u7891\u7a81\u7834\uff01\u5168\u7403\u9996\u500bAPOC3\u57fa\u56e0\u7de8\u8f2f\u81e8\u5e8a\u6cbb\u7642\u9ad8\u8840\u8102\u7372\u5f97\u6210\u529f - \u5c4f\u6771\u6642\u5831\u96fb\u5b50\u5831<\/title>\n<meta name=\"description\" content=\"\u6b63\u5e8f\u751f\u7269CS-121\u9996\u4f8b\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u60a3\u8005\u5b8c\u6210\u7d66\u85e5\uff0c\u986f\u793a\u826f\u597d\u5b89\u5168\u6027\u53ca\u986f\u8457\u7642\u6548 \u4e0a\u6d772025\u5e7411\u67087\u65e5 \/\u7f8e\u901a\u793e\/ -- \u7576\u5730\u6642\u95932025\u5e7411\u67086\u65e5\uff0c\u5c08\u6ce8\u65bc\u65b0\u578b\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u3001\u8655\u65bc\u81e8\u5e8a\u968e\u6bb5\u7684\u5275\u65b0\u751f\u7269\u91ab\u85e5\u79d1\u6280\u4f01\u696d\u6b63\u5e8f\u751f\u7269\uff08\u4e0a\u6d77\uff09\u5ba3\u4f48\uff0c\u8207\u5b89\u5fbd\u91ab\u79d1\u5927\u5b78\u7b2c\u4e00\u9644\u5c6c\u91ab\u9662\u5408\u4f5c\u958b\u5c55\u7684\u91dd\u5c0d\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7684\u9776\u5411APOC3\u7684\u9e7c\u57fa\u7de8\u8f2f\u85e5\u7269CS-121\u6ce8\u5c04\u6db2\u7684\u7814\u7a76\u8005\u767c\u8d77\u7684\u81e8\u5e8a\u7814\u7a76\uff08Investigator Initiated Trial, IIT\uff09\u6cbb\u7642\u7684\u9996\u4f4d\u60a3\u8005\u5df2\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662\u3002\u60a3\u8005\u60a3\u6709\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\uff0c\u6b77\u53f2\u7a7a\u8179\u7518\u6cb9\u4e09\u916f\uff08TG\uff09\u6c34\u6e96&gt;12.5 mmol\/L\uff0c\u591a\u6b21\u8a98\u767c\u6025\u6027\u80f0\u817a\u708e\u3002\u6b64\u6b21IIT\u8a66\u9a57\u70ba\u5291\u91cf\u722c\u5761\u8a66\u9a57\uff0c\u60a3\u8005\u5728\u55ae\u6b21\u6700\u4f4e\u5291\u91cf\u7d66\u85e5\u5f8c\u4e09\u5929\u5167\uff0c\u7a7a\u8179TG\u6c34\u6e96\u986f\u8457\u4e0b\u964d\uff0c\u4e14\u672a\u767c\u751f\u4e0d\u826f\u53cd\u61c9\u3002\u9019\u662f\u5168\u7403\u9996\u6b21\u901a\u904e\u9776\u5411\u9e7c\u57fa\u7de8\u8f2fAPOC3\u57fa\u56e0\u6cbb\u7642\u9ad8\u8840\u8102\u60a3\u8005\u7684\u6848\u4f8b\u3002   \u5168\u7403\u9996\u4f8b\u63a5\u53d7APOC3\u57fa\u56e0\u7de8\u8f2f\u6cbb\u7642\uff08\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\uff09\u7684\u9ad8\u8840\u8102\u60a3\u8005\uff08\u53f35\uff09\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662  \u5168\u7403\u9996\u4f8b\u63a5\u53d7APOC3\u57fa\u56e0\u7de8\u8f2f\u6cbb\u7642\uff08\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\uff09\u7684\u9ad8\u8840\u8102\u60a3\u8005\uff08\u53f35\uff09\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662 \u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u662f\u4e00\u7a2e\u56e0\u8840\u6db2\u4e2d\u4e73\u7cdc\u5fae\u7c92\u7570\u5e38\u5347\u9ad8\u5c0e\u81f4\u7684\u4ee3\u8b1d\u6027\u75be\u75c5\uff0c\u5e38\u8207\u8102\u8cea\u4ee3\u8b1d\u7d0a\u4e82\u76f8\u95dc\uff0c\u4e3b\u8981\u8868\u73fe\u70ba\u7a7a\u8179\u7518\u6cb9\u4e09\u916f\uff08TG\uff09\u6c34\u6e96\u6975\u5ea6\u5347\u9ad8\uff0c\u53ef\u80fd\u5f15\u767c\u4e0d\u53ef\u9810\u6e2c\u4e14\u6f5b\u5728\u81f4\u547d\u7684\u6025\u6027\u80f0\u817a\u708e\u7b49\u56b4\u91cd\u4f75\u767c\u75c7\uff0c\u662f\u56b4\u91cd\u9ad8\u7518\u6cb9\u4e09\u8102\u8840\u75c7\uff08Severe Hypertriglyceridemia\uff0csHTG\uff09\u6700\u56b4\u91cd\u7684\u4e00\u7a2e\u4e9e\u578b\u3002\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u5305\u542b\u5bb6\u65cf\u6027\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7d9c\u5408\u5f81\uff08Familial Chylomicronemia Syndrome\uff0cFCS\uff09\u548c\u591a\u56e0\u7d20\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7d9c\u5408\u5f81\uff08Multifactorial Chylomicronemia Syndrome\uff0cMCS\uff09\u3002\u5176\u4e2dFCS\u662f\u4e00\u7a2e\u7f55\u898b\u7684\u5e38\u67d3\u8272\u9ad4\u96b1\u6027\u907a\u50b3\u75c5\uff0c\u7531\u65bc\u8102\u86cb\u767d\u8102\u80aa\u9176\uff08Lipoprotein lipase\uff0cLPL\uff09\u57fa\u56e0\u6216\u53c3\u8207\u8abf\u63a7LPL\u6d3b\u6027\u7684\u95dc\u9375\u57fa\u56e0\u767c\u751f\u96d9\u7b49\u4f4d\u57fa\u56e0\u7a81\u8b8a\uff0c\u5c0e\u81f4\u8102\u86cb\u767d\u8102\u80aa\u9176\u529f\u80fd\u53d7\u640d\uff0c\u4f7f\u7518\u6cb9\u4e09\u8102\u7684\u5206\u89e3\u4ee3\u8b1d\u7522\u751f\u969c\u7919\uff0c\u5f9e\u800c\u9020\u6210\u9ad4\u5167\u4e73\u7cdc\u5fae\u7c92\u7684\u84c4\u7a4d\uff0c\u901a\u5e38\u60a3\u8005\u7684\u7a7a\u8179TG\u6c34\u6e96\u226510 mmol\/L (885 mg\/dL)\uff0c\u5168\u7403\u767c\u75c5\u7387\u5728\u5341\u842c\u5206\u4e4b\u4e00\u5230\u767e\u842c\u5206\u4e4b\u4e00\u4e4b\u9593\uff1b\u800cMCS\u5247\u7531\u591a\u7a2e\u56e0\u7d20\u5f15\u8d77\u7684\u75c5\u7406\u72c0\u614b\uff0c\u5176\u767c\u75c5\u53d7\u907a\u50b3\u80cc\u666f\u3001\u751f\u6d3b\u7fd2\u6163\u6216\u5176\u4ed6\u4ee3\u8b1d\u75be\u75c5\u5171\u540c\u5f71\u97ff\uff0c\u5168\u7403\u767c\u75c5\u7387\u9ad8\u90541\/600\u3002 \u76ee\u524d\uff0c\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7684\u4e3b\u8981\u6cbb\u7642\u7b56\u7565\u662f\u5c07\u60a3\u8005\u7684\u7a7a\u8179\u7518\u6cb9\u4e09\u8102\uff08TG\uff09\u6c34\u6e96\u63a7\u5236\u5728\u6025\u6027\u80f0\u817a\u708e\u98a8\u96aa\u95be\u503c\u4ee5\u4e0b\uff08\u901a\u5e38\u9700&lt;5.7 mmol\/L\uff0c\u5373\u00a0500 mg\/dL\uff09\uff0c\u7136\u800c\u76ee\u524d\u5df2\u4e0a\u5e02\u7684\u5404\u985e\u964d\u7518\u6cb9\u4e09\u8102\u7684\u85e5\u7269\u5c0d\u9019\u985e\u60a3\u8005\u7684\u6548\u679c\u5fae\u4e4e\u5176\u5fae\uff0c\u60a3\u8005\u4ecd\u7136\u9762\u81e8\u60a3\u6025\u6027\u80f0\u817a\u708e\u7b49\u4f75\u767c\u75c7\u7684\u98a8\u96aa\uff1b\u56b4\u683c\u7684\u98f2\u98df\u63a7\u5236\u5118\u7ba1\u53ef\u4ee5\u901a\u904e\u6bcf\u65e5\u6975\u4f4e\u8102\u80aa\u7684\u98f2\u98df\u4f86\u5be6\u73fe\u4e73\u7cdc\u5fae\u7c92\u7684\u964d\u4f4e\uff0c\u4f46\u662f\u60a3\u8005\u5f80\u5f80\u5f88\u96e3\u9577\u671f\u5805\u6301\uff0c\u4f9d\u7136\u6301\u7e8c\u9762\u81e8\u56b4\u91cd\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u5e36\u4f86\u7684\u98a8\u96aa\u3002\u7814\u7a76\u767c\u73fe\uff0c\u4e00\u7a2e\u4e3b\u8981\u5728\u809d\u81df\u4e2d\u7522\u751f\u7684\u86cb\u767d\u8ceaAPOC3\u5728\u8abf\u7bc0\u8840\u6f3f\u7518\u6cb9\u4e09\u916f\u65b9\u9762\u767c\u63ee\u8457\u91cd\u8981\u4f5c\u7528\uff0c\u5c0d\u5927\u898f\u6a21\u4eba\u7fa4\u7684\u6aa2\u6e2c\u7814\u7a76\u767c\u73fe\uff0c\u651c\u5e36\u5929\u7136APOC3\u529f\u80fd\u7f3a\u5931\u7a81\u8b8a\u7684\u4eba\u7fa4\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u542b\u91cf\u8f03\u4f4e\u4e14\u4e0d\u8868\u73fe\u5176\u4ed6\u81e8\u5e8a\u75c7\u72c0\u3002\u96a8\u8457\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u7684\u767c\u5c55\uff0c\u901a\u904e\u5728\u57fa\u56e0\u5c64\u9762\u8abf\u63a7APOC3\u7684\u8868\u9054\u5f9e\u800c\u964d\u4f4e\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u7684\u6cbb\u7642\u7b56\u7565\u5f97\u4ee5\u5be6\u73fe\uff0c\u9019\u5c07\u70ba\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u60a3\u8005\u5e36\u4f86\u6cbb\u7652\u7684\u5e0c\u671b\u3002 \u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\u662f\u5229\u7528\u4e0a\u6d77\u79d1\u6280\u5927\u5b78\u81ea\u4e3b\u7814\u767c\u7684\u9ad8\u7cbe\u51c6\u8b8a\u5f62\u5f0f\u9e7c\u57fa\u7de8\u8f2f\u5668tBE\uff08transformer Base Editor\uff09\uff08Wang et al.\uff0cNat Cell Biol\uff0c2021\uff09\u958b\u767c\u7684\u91dd\u5c0d\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7684\u9996\u6b3e\u9ad4\u5167\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269\u3002tBE\u7531\u8102\u8cea\u7d0d\u7c73\u7c92\uff08Lipid Nanoparticle\uff0cLNP\uff09\u5305\u88dd\uff0c\u901a\u904e\u975c\u8108\u6ce8\u5c04\u7d66\u85e5\u905e\u9001\u81f3\u60a3\u8005\u809d\u81df\uff0c\u5c0d\u9776\u6a19\u57fa\u56e0APOC3\u9032\u884c\u7cbe\u51c6\u9e7c\u57fa\u7de8\u8f2f\uff0c\u6a21\u64ec\u5929\u7136APOC3\u529f\u80fd\u7f3a\u5931\u4eba\u7fa4\u4e2d\u7684\u6709\u76ca\u7a81\u8b8a\uff0c\u4e0b\u8abfAPOC3\u7684\u57fa\u56e0\u8868\u9054\uff0c\u9032\u800c\u9054\u5230\u964d\u4f4e\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u7684\u76ee\u7684\u3002\u901a\u904e\u57fa\u56e0\u6c34\u6e96\u7684\u7de8\u8f2f\uff0c\u8a72\u7642\u6cd5\u6709\u671b\u5be6\u73fe&quot;\u4e00\u6b21\u7d66\u85e5\uff0c\u7d42\u8eab\u6709\u6548&quot;\u7684\u964d\u7518\u6cb9\u4e09\u8102\u7684\u76ee\u6a19\u3002tBE\u5c6c\u65bc\u4e0b\u4e00\u4ee3\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\uff0c\u4e0d\u9700\u8981\u7834\u58deDNA\u96d9\u93c8\u5373\u53ef\u5be6\u73fe\u5c0d\u55ae\u9e7c\u57fa\u7a81\u8b8a\u9032\u884c\u7cbe\u51c6\u6821\u6b63\uff0c\u4e0d\u6703\u4fc3\u767cp53\u901a\u8def\u555f\u52d5\u548c\u67d3\u8272\u9ad4\u7570\u5e38\u7b49\u5b89\u5168\u98a8\u96aa\u3002\u76f8\u6bd4\u8f03\u5176\u4ed6\u57fa\u65bcCRISPR\u6280\u8853\u7684\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5\uff0cCS-121\u5b89\u5168\u6027\u66f4\u9ad8\uff0c\u4e0d\u6703\u5f15\u767c\u60a3\u8005\u57fa\u56e0\u7d44DNA\u5927\u7247\u6bb5\u7f3a\u5931\u3001\u67d3\u8272\u9ad4\u7a81\u8b8a\u3001\u812b\u9776\u7a81\u8b8a\u7b49\u98a8\u96aa\uff0c\u66f4\u4e0d\u6703\u5e36\u4f86\u7531CRISPR\/Cas9\u5207\u65b7DNA\u96d9\u93c8\u800c\u555f\u52d5p53\u901a\u8def\u5c0e\u81f4\u7684\u809d\u6bd2\u6027\u7b49\u526f\u53cd\u61c9\u3002\u5728\u524d\u671f\u7684\u52d5\u7269\u8a66\u9a57\u4e2d\uff0cCS-121\u7684\u5b89\u5168\u6027\u548c\u9577\u671f\u6709\u6548\u6027\u5df2\u7d93\u7372\u5f97\u9a57\u8b49\uff0c\u5728\u809d\u81df\u3001\u80ba\u3001\u808c\u8089\u3001\u813e\u81df\u3001\u5375\u5de2\u3001\u5fc3\u81df\u3001\u814e\u81df\u7b49\u5404\u7a2e\u7d44\u7e54\u4e2d\u5747\u6c92\u6709\u6aa2\u6e2c\u5230\u812b\u9776\u7de8\u8f2f\u3002 \u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\u6cbb\u7642\u7684\u9996\u4f4d\u60a3\u8005\u662f\u4e00\u540d\u60a3\u6709\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u768463\u6b72\u7537\u6027\u3002\u60a3\u8005\u57282025\u5e7410\u670818\u65e5\u63a5\u53d7CS-121\u6700\u4f4e\u5291\u91cf\u55ae\u6b21\u7d66\u85e5\uff0c\u7d66\u85e5\u5f8c\u4e09\u5929\u5167\uff0c\u7a7a\u8179TG\u6c34\u6e96\u986f\u8457\u4e0b\u964d\uff0c\u4e26\u65bc\u7d66\u85e5\u5f8c\u7b2c3\u5929\u9806\u5229\u51fa\u9662\u3002\u622a\u81f3\u5831\u5c0e\u65e5\u671f\uff0c\u672a\u51fa\u73fe\u7522\u54c1\u76f8\u95dc\u7684\u4e0d\u826f\u4e8b\u4ef6\u3002 \u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2IIT\u5c08\u6848\u7684\u4e3b\u8981\u7814\u7a76\u8005\u70ba\u5b89\u5fbd\u91ab\u79d1\u5927\u5b78\u7b2c\u4e00\u9644\u5c6c\u91ab\u9662\u5468\u7165\u6559\u6388\uff0c\u5433\u5fd7\u9e97\u4e3b\u4efb\u3002 \u6b63\u5e8f\u751f\u7269\u662f\u4e00\u5bb6\u5df2\u7d93\u9032\u5165IND\u81e8\u5e8a\u968e\u6bb5\u7684\u5275\u65b0\u751f\u7269\u91ab\u85e5\u79d1\u6280\u516c\u53f8\uff0c\u5176\u958b\u767c\u7684\u9996\u6b3e\u9ad4\u5916\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269CS-101\u6ce8\u5c04\u6db2\u5df2\u7d93\u6210\u529f\u6cbb\u7652\u6578\u5341\u4f8b\u03b2-\u5730\u4e2d\u6d77\u8ca7\u8840\u548c\u942e\u5200\u578b\u7d30\u80de\u8ca7\u8840\u75c5\u60a3\u8005\u3002\u6b63\u5e8f\u751f\u7269\u6b63\u5728\u52a0\u901f\u63a8\u9032\u9996\u6b3e\u9ad4\u5167\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269CS-121\u6ce8\u5c04\u6db2\u9032\u5165IND\u81e8\u5e8a\u53ca\u4e0a\u5e02\uff0c\u6709\u671b\u70ba\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7b49\u4ee3\u8b1d\u985e\u75be\u75c5\u60a3\u8005\u5e36\u4f86&quot;\u4e00\u6b21\u6cbb\u7642\uff0c\u7d42\u8eab\u6709\u6548&quot;\u7684\u985b\u8986\u6027\u6cbb\u7642\u65b9\u6848\u3002 \uff08\u7279\u5225\u611f\u8b1d\u5b89\u5fbd\u91ab\u79d1\u5927\u5b78\u7b2c\u4e00\u9644\u5c6c\u91ab\u9662\u3001\u4e0a\u6d77\u79d1\u6280\u5927\u5b78\u53ca\u4e0a\u6d77\u81e8\u5e8a\u7814\u7a76\u4e2d\u5fc3\uff09 \u95dc\u65bc\u6b63\u5e8f\u751f\u7269 \u6b63\u5e8f\u751f\u7269\uff08CorrectSequence Therapeutics\uff09\u662f\u4e00\u5bb6\u5c08\u6ce8\u65bc\u65b0\u578b\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u3001\u8655\u65bc\u81e8\u5e8a\u968e\u6bb5\u7684\u751f\u7269\u91ab\u85e5\u79d1\u6280\u516c\u53f8\uff0c\u5b75\u5316\u81ea\u4e0a\u6d77\u79d1\u6280\u5927\u5b78\uff0c\u81f4\u529b\u65bc\u5229\u7528\u81ea\u4e3b\u77e5\u8b58\u7522\u6b0a\u7684\u9e7c\u57fa\u7de8\u8f2f\u9ad4\u7cfb\uff0c\u958b\u767c\u7a81\u7834\u6027\u7cbe\u51c6\u7642\u6cd5\uff0c\u9020\u798f\u4eba\u985e\u5065\u5eb7\u3002\u516c\u53f8\u57fa\u65bc\u4ee5\u8b8a\u5f62\u5f0f\u9e7c\u57fa\u7de8\u8f2f\u5668tBE\uff08transformer Base Editor\uff09\u70ba\u4ee3\u8868\u7684\u81ea\u4e3bIP\u9e7c\u57fa\u7de8\u8f2f\u7cfb\u7d71\u5275\u5efa\u4e86\u591a\u7a2e\u7cbe\u51c6\u7642\u6cd5\uff0c\u91dd\u5c0d\u907a\u50b3\u75be\u75c5\u3001\u4ee3\u8b1d\u75be\u75c5\u3001\u5fc3\u8840\u7ba1\u75be\u75c5\u7b49\u4f48\u5c40\u4e86\u591a\u689d\u7ba1\u7dda\uff0c\u9996\u689d\u7ba1\u7dda\u5373\u5c07\u9032\u5165\u95dc\u9375\u6027\u81e8\u5e8a\u8a66\u9a57\uff08\u2161\/\u2162\u671f\u81e8\u5e8a\u8a66\u9a57\uff09\u968e\u6bb5\u3002 \u6b32\u77ad\u89e3\u66f4\u591a\u8cc7\u8a0a\uff0c\u8acb\u9ede\u64ca\u9032\u5165\u5b98\u7db2\u00a0www.correctsequence.com \u5a92\u9ad4\u806f\u7cfb\u4eba\uff1a\u5546\u52d9\u5408\u4f5c\uff1aBD@correctsequence.com\u81e8\u5e8a\u8a66\u9a57\uff1aCT@correctsequence.com\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/pingtungtimes.com.tw\/?p=379703\" \/>\n<meta property=\"og:locale\" content=\"zh_TW\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"\u91cc\u7a0b\u7891\u7a81\u7834\uff01\u5168\u7403\u9996\u500bAPOC3\u57fa\u56e0\u7de8\u8f2f\u81e8\u5e8a\u6cbb\u7642\u9ad8\u8840\u8102\u7372\u5f97\u6210\u529f - \u5c4f\u6771\u6642\u5831\u96fb\u5b50\u5831\" \/>\n<meta property=\"og:description\" content=\"\u6b63\u5e8f\u751f\u7269CS-121\u9996\u4f8b\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u60a3\u8005\u5b8c\u6210\u7d66\u85e5\uff0c\u986f\u793a\u826f\u597d\u5b89\u5168\u6027\u53ca\u986f\u8457\u7642\u6548 \u4e0a\u6d772025\u5e7411\u67087\u65e5 \/\u7f8e\u901a\u793e\/ -- \u7576\u5730\u6642\u95932025\u5e7411\u67086\u65e5\uff0c\u5c08\u6ce8\u65bc\u65b0\u578b\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u3001\u8655\u65bc\u81e8\u5e8a\u968e\u6bb5\u7684\u5275\u65b0\u751f\u7269\u91ab\u85e5\u79d1\u6280\u4f01\u696d\u6b63\u5e8f\u751f\u7269\uff08\u4e0a\u6d77\uff09\u5ba3\u4f48\uff0c\u8207\u5b89\u5fbd\u91ab\u79d1\u5927\u5b78\u7b2c\u4e00\u9644\u5c6c\u91ab\u9662\u5408\u4f5c\u958b\u5c55\u7684\u91dd\u5c0d\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7684\u9776\u5411APOC3\u7684\u9e7c\u57fa\u7de8\u8f2f\u85e5\u7269CS-121\u6ce8\u5c04\u6db2\u7684\u7814\u7a76\u8005\u767c\u8d77\u7684\u81e8\u5e8a\u7814\u7a76\uff08Investigator Initiated Trial, IIT\uff09\u6cbb\u7642\u7684\u9996\u4f4d\u60a3\u8005\u5df2\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662\u3002\u60a3\u8005\u60a3\u6709\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\uff0c\u6b77\u53f2\u7a7a\u8179\u7518\u6cb9\u4e09\u916f\uff08TG\uff09\u6c34\u6e96&gt;12.5 mmol\/L\uff0c\u591a\u6b21\u8a98\u767c\u6025\u6027\u80f0\u817a\u708e\u3002\u6b64\u6b21IIT\u8a66\u9a57\u70ba\u5291\u91cf\u722c\u5761\u8a66\u9a57\uff0c\u60a3\u8005\u5728\u55ae\u6b21\u6700\u4f4e\u5291\u91cf\u7d66\u85e5\u5f8c\u4e09\u5929\u5167\uff0c\u7a7a\u8179TG\u6c34\u6e96\u986f\u8457\u4e0b\u964d\uff0c\u4e14\u672a\u767c\u751f\u4e0d\u826f\u53cd\u61c9\u3002\u9019\u662f\u5168\u7403\u9996\u6b21\u901a\u904e\u9776\u5411\u9e7c\u57fa\u7de8\u8f2fAPOC3\u57fa\u56e0\u6cbb\u7642\u9ad8\u8840\u8102\u60a3\u8005\u7684\u6848\u4f8b\u3002   \u5168\u7403\u9996\u4f8b\u63a5\u53d7APOC3\u57fa\u56e0\u7de8\u8f2f\u6cbb\u7642\uff08\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\uff09\u7684\u9ad8\u8840\u8102\u60a3\u8005\uff08\u53f35\uff09\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662  \u5168\u7403\u9996\u4f8b\u63a5\u53d7APOC3\u57fa\u56e0\u7de8\u8f2f\u6cbb\u7642\uff08\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\uff09\u7684\u9ad8\u8840\u8102\u60a3\u8005\uff08\u53f35\uff09\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662 \u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u662f\u4e00\u7a2e\u56e0\u8840\u6db2\u4e2d\u4e73\u7cdc\u5fae\u7c92\u7570\u5e38\u5347\u9ad8\u5c0e\u81f4\u7684\u4ee3\u8b1d\u6027\u75be\u75c5\uff0c\u5e38\u8207\u8102\u8cea\u4ee3\u8b1d\u7d0a\u4e82\u76f8\u95dc\uff0c\u4e3b\u8981\u8868\u73fe\u70ba\u7a7a\u8179\u7518\u6cb9\u4e09\u916f\uff08TG\uff09\u6c34\u6e96\u6975\u5ea6\u5347\u9ad8\uff0c\u53ef\u80fd\u5f15\u767c\u4e0d\u53ef\u9810\u6e2c\u4e14\u6f5b\u5728\u81f4\u547d\u7684\u6025\u6027\u80f0\u817a\u708e\u7b49\u56b4\u91cd\u4f75\u767c\u75c7\uff0c\u662f\u56b4\u91cd\u9ad8\u7518\u6cb9\u4e09\u8102\u8840\u75c7\uff08Severe Hypertriglyceridemia\uff0csHTG\uff09\u6700\u56b4\u91cd\u7684\u4e00\u7a2e\u4e9e\u578b\u3002\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u5305\u542b\u5bb6\u65cf\u6027\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7d9c\u5408\u5f81\uff08Familial Chylomicronemia Syndrome\uff0cFCS\uff09\u548c\u591a\u56e0\u7d20\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7d9c\u5408\u5f81\uff08Multifactorial Chylomicronemia Syndrome\uff0cMCS\uff09\u3002\u5176\u4e2dFCS\u662f\u4e00\u7a2e\u7f55\u898b\u7684\u5e38\u67d3\u8272\u9ad4\u96b1\u6027\u907a\u50b3\u75c5\uff0c\u7531\u65bc\u8102\u86cb\u767d\u8102\u80aa\u9176\uff08Lipoprotein lipase\uff0cLPL\uff09\u57fa\u56e0\u6216\u53c3\u8207\u8abf\u63a7LPL\u6d3b\u6027\u7684\u95dc\u9375\u57fa\u56e0\u767c\u751f\u96d9\u7b49\u4f4d\u57fa\u56e0\u7a81\u8b8a\uff0c\u5c0e\u81f4\u8102\u86cb\u767d\u8102\u80aa\u9176\u529f\u80fd\u53d7\u640d\uff0c\u4f7f\u7518\u6cb9\u4e09\u8102\u7684\u5206\u89e3\u4ee3\u8b1d\u7522\u751f\u969c\u7919\uff0c\u5f9e\u800c\u9020\u6210\u9ad4\u5167\u4e73\u7cdc\u5fae\u7c92\u7684\u84c4\u7a4d\uff0c\u901a\u5e38\u60a3\u8005\u7684\u7a7a\u8179TG\u6c34\u6e96\u226510 mmol\/L (885 mg\/dL)\uff0c\u5168\u7403\u767c\u75c5\u7387\u5728\u5341\u842c\u5206\u4e4b\u4e00\u5230\u767e\u842c\u5206\u4e4b\u4e00\u4e4b\u9593\uff1b\u800cMCS\u5247\u7531\u591a\u7a2e\u56e0\u7d20\u5f15\u8d77\u7684\u75c5\u7406\u72c0\u614b\uff0c\u5176\u767c\u75c5\u53d7\u907a\u50b3\u80cc\u666f\u3001\u751f\u6d3b\u7fd2\u6163\u6216\u5176\u4ed6\u4ee3\u8b1d\u75be\u75c5\u5171\u540c\u5f71\u97ff\uff0c\u5168\u7403\u767c\u75c5\u7387\u9ad8\u90541\/600\u3002 \u76ee\u524d\uff0c\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7684\u4e3b\u8981\u6cbb\u7642\u7b56\u7565\u662f\u5c07\u60a3\u8005\u7684\u7a7a\u8179\u7518\u6cb9\u4e09\u8102\uff08TG\uff09\u6c34\u6e96\u63a7\u5236\u5728\u6025\u6027\u80f0\u817a\u708e\u98a8\u96aa\u95be\u503c\u4ee5\u4e0b\uff08\u901a\u5e38\u9700&lt;5.7 mmol\/L\uff0c\u5373\u00a0500 mg\/dL\uff09\uff0c\u7136\u800c\u76ee\u524d\u5df2\u4e0a\u5e02\u7684\u5404\u985e\u964d\u7518\u6cb9\u4e09\u8102\u7684\u85e5\u7269\u5c0d\u9019\u985e\u60a3\u8005\u7684\u6548\u679c\u5fae\u4e4e\u5176\u5fae\uff0c\u60a3\u8005\u4ecd\u7136\u9762\u81e8\u60a3\u6025\u6027\u80f0\u817a\u708e\u7b49\u4f75\u767c\u75c7\u7684\u98a8\u96aa\uff1b\u56b4\u683c\u7684\u98f2\u98df\u63a7\u5236\u5118\u7ba1\u53ef\u4ee5\u901a\u904e\u6bcf\u65e5\u6975\u4f4e\u8102\u80aa\u7684\u98f2\u98df\u4f86\u5be6\u73fe\u4e73\u7cdc\u5fae\u7c92\u7684\u964d\u4f4e\uff0c\u4f46\u662f\u60a3\u8005\u5f80\u5f80\u5f88\u96e3\u9577\u671f\u5805\u6301\uff0c\u4f9d\u7136\u6301\u7e8c\u9762\u81e8\u56b4\u91cd\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u5e36\u4f86\u7684\u98a8\u96aa\u3002\u7814\u7a76\u767c\u73fe\uff0c\u4e00\u7a2e\u4e3b\u8981\u5728\u809d\u81df\u4e2d\u7522\u751f\u7684\u86cb\u767d\u8ceaAPOC3\u5728\u8abf\u7bc0\u8840\u6f3f\u7518\u6cb9\u4e09\u916f\u65b9\u9762\u767c\u63ee\u8457\u91cd\u8981\u4f5c\u7528\uff0c\u5c0d\u5927\u898f\u6a21\u4eba\u7fa4\u7684\u6aa2\u6e2c\u7814\u7a76\u767c\u73fe\uff0c\u651c\u5e36\u5929\u7136APOC3\u529f\u80fd\u7f3a\u5931\u7a81\u8b8a\u7684\u4eba\u7fa4\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u542b\u91cf\u8f03\u4f4e\u4e14\u4e0d\u8868\u73fe\u5176\u4ed6\u81e8\u5e8a\u75c7\u72c0\u3002\u96a8\u8457\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u7684\u767c\u5c55\uff0c\u901a\u904e\u5728\u57fa\u56e0\u5c64\u9762\u8abf\u63a7APOC3\u7684\u8868\u9054\u5f9e\u800c\u964d\u4f4e\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u7684\u6cbb\u7642\u7b56\u7565\u5f97\u4ee5\u5be6\u73fe\uff0c\u9019\u5c07\u70ba\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u60a3\u8005\u5e36\u4f86\u6cbb\u7652\u7684\u5e0c\u671b\u3002 \u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\u662f\u5229\u7528\u4e0a\u6d77\u79d1\u6280\u5927\u5b78\u81ea\u4e3b\u7814\u767c\u7684\u9ad8\u7cbe\u51c6\u8b8a\u5f62\u5f0f\u9e7c\u57fa\u7de8\u8f2f\u5668tBE\uff08transformer Base Editor\uff09\uff08Wang et al.\uff0cNat Cell Biol\uff0c2021\uff09\u958b\u767c\u7684\u91dd\u5c0d\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7684\u9996\u6b3e\u9ad4\u5167\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269\u3002tBE\u7531\u8102\u8cea\u7d0d\u7c73\u7c92\uff08Lipid 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\u7576\u5730\u6642\u95932025\u5e7411\u67086\u65e5\uff0c\u5c08\u6ce8\u65bc\u65b0\u578b\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u3001\u8655\u65bc\u81e8\u5e8a\u968e\u6bb5\u7684\u5275\u65b0\u751f\u7269\u91ab\u85e5\u79d1\u6280\u4f01\u696d\u6b63\u5e8f\u751f\u7269\uff08\u4e0a\u6d77\uff09\u5ba3\u4f48\uff0c\u8207\u5b89\u5fbd\u91ab\u79d1\u5927\u5b78\u7b2c\u4e00\u9644\u5c6c\u91ab\u9662\u5408\u4f5c\u958b\u5c55\u7684\u91dd\u5c0d\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\\\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7684\u9776\u5411APOC3\u7684\u9e7c\u57fa\u7de8\u8f2f\u85e5\u7269CS-121\u6ce8\u5c04\u6db2\u7684\u7814\u7a76\u8005\u767c\u8d77\u7684\u81e8\u5e8a\u7814\u7a76\uff08Investigator Initiated Trial, IIT\uff09\u6cbb\u7642\u7684\u9996\u4f4d\u60a3\u8005\u5df2\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662\u3002\u60a3\u8005\u60a3\u6709\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\uff0c\u6b77\u53f2\u7a7a\u8179\u7518\u6cb9\u4e09\u916f\uff08TG\uff09\u6c34\u6e96&gt;12.5 mmol\\\/L\uff0c\u591a\u6b21\u8a98\u767c\u6025\u6027\u80f0\u817a\u708e\u3002\u6b64\u6b21IIT\u8a66\u9a57\u70ba\u5291\u91cf\u722c\u5761\u8a66\u9a57\uff0c\u60a3\u8005\u5728\u55ae\u6b21\u6700\u4f4e\u5291\u91cf\u7d66\u85e5\u5f8c\u4e09\u5929\u5167\uff0c\u7a7a\u8179TG\u6c34\u6e96\u986f\u8457\u4e0b\u964d\uff0c\u4e14\u672a\u767c\u751f\u4e0d\u826f\u53cd\u61c9\u3002\u9019\u662f\u5168\u7403\u9996\u6b21\u901a\u904e\u9776\u5411\u9e7c\u57fa\u7de8\u8f2fAPOC3\u57fa\u56e0\u6cbb\u7642\u9ad8\u8840\u8102\u60a3\u8005\u7684\u6848\u4f8b\u3002 \u5168\u7403\u9996\u4f8b\u63a5\u53d7APOC3\u57fa\u56e0\u7de8\u8f2f\u6cbb\u7642\uff08\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\uff09\u7684\u9ad8\u8840\u8102\u60a3\u8005\uff08\u53f35\uff09\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662 \u5168\u7403\u9996\u4f8b\u63a5\u53d7APOC3\u57fa\u56e0\u7de8\u8f2f\u6cbb\u7642\uff08\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\uff09\u7684\u9ad8\u8840\u8102\u60a3\u8005\uff08\u53f35\uff09\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662 \u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u662f\u4e00\u7a2e\u56e0\u8840\u6db2\u4e2d\u4e73\u7cdc\u5fae\u7c92\u7570\u5e38\u5347\u9ad8\u5c0e\u81f4\u7684\u4ee3\u8b1d\u6027\u75be\u75c5\uff0c\u5e38\u8207\u8102\u8cea\u4ee3\u8b1d\u7d0a\u4e82\u76f8\u95dc\uff0c\u4e3b\u8981\u8868\u73fe\u70ba\u7a7a\u8179\u7518\u6cb9\u4e09\u916f\uff08TG\uff09\u6c34\u6e96\u6975\u5ea6\u5347\u9ad8\uff0c\u53ef\u80fd\u5f15\u767c\u4e0d\u53ef\u9810\u6e2c\u4e14\u6f5b\u5728\u81f4\u547d\u7684\u6025\u6027\u80f0\u817a\u708e\u7b49\u56b4\u91cd\u4f75\u767c\u75c7\uff0c\u662f\u56b4\u91cd\u9ad8\u7518\u6cb9\u4e09\u8102\u8840\u75c7\uff08Severe Hypertriglyceridemia\uff0csHTG\uff09\u6700\u56b4\u91cd\u7684\u4e00\u7a2e\u4e9e\u578b\u3002\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u5305\u542b\u5bb6\u65cf\u6027\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7d9c\u5408\u5f81\uff08Familial Chylomicronemia Syndrome\uff0cFCS\uff09\u548c\u591a\u56e0\u7d20\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7d9c\u5408\u5f81\uff08Multifactorial Chylomicronemia Syndrome\uff0cMCS\uff09\u3002\u5176\u4e2dFCS\u662f\u4e00\u7a2e\u7f55\u898b\u7684\u5e38\u67d3\u8272\u9ad4\u96b1\u6027\u907a\u50b3\u75c5\uff0c\u7531\u65bc\u8102\u86cb\u767d\u8102\u80aa\u9176\uff08Lipoprotein lipase\uff0cLPL\uff09\u57fa\u56e0\u6216\u53c3\u8207\u8abf\u63a7LPL\u6d3b\u6027\u7684\u95dc\u9375\u57fa\u56e0\u767c\u751f\u96d9\u7b49\u4f4d\u57fa\u56e0\u7a81\u8b8a\uff0c\u5c0e\u81f4\u8102\u86cb\u767d\u8102\u80aa\u9176\u529f\u80fd\u53d7\u640d\uff0c\u4f7f\u7518\u6cb9\u4e09\u8102\u7684\u5206\u89e3\u4ee3\u8b1d\u7522\u751f\u969c\u7919\uff0c\u5f9e\u800c\u9020\u6210\u9ad4\u5167\u4e73\u7cdc\u5fae\u7c92\u7684\u84c4\u7a4d\uff0c\u901a\u5e38\u60a3\u8005\u7684\u7a7a\u8179TG\u6c34\u6e96\u226510 mmol\\\/L (885 mg\\\/dL)\uff0c\u5168\u7403\u767c\u75c5\u7387\u5728\u5341\u842c\u5206\u4e4b\u4e00\u5230\u767e\u842c\u5206\u4e4b\u4e00\u4e4b\u9593\uff1b\u800cMCS\u5247\u7531\u591a\u7a2e\u56e0\u7d20\u5f15\u8d77\u7684\u75c5\u7406\u72c0\u614b\uff0c\u5176\u767c\u75c5\u53d7\u907a\u50b3\u80cc\u666f\u3001\u751f\u6d3b\u7fd2\u6163\u6216\u5176\u4ed6\u4ee3\u8b1d\u75be\u75c5\u5171\u540c\u5f71\u97ff\uff0c\u5168\u7403\u767c\u75c5\u7387\u9ad8\u90541\\\/600\u3002 \u76ee\u524d\uff0c\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7684\u4e3b\u8981\u6cbb\u7642\u7b56\u7565\u662f\u5c07\u60a3\u8005\u7684\u7a7a\u8179\u7518\u6cb9\u4e09\u8102\uff08TG\uff09\u6c34\u6e96\u63a7\u5236\u5728\u6025\u6027\u80f0\u817a\u708e\u98a8\u96aa\u95be\u503c\u4ee5\u4e0b\uff08\u901a\u5e38\u9700&lt;5.7 mmol\\\/L\uff0c\u5373\u00a0500 mg\\\/dL\uff09\uff0c\u7136\u800c\u76ee\u524d\u5df2\u4e0a\u5e02\u7684\u5404\u985e\u964d\u7518\u6cb9\u4e09\u8102\u7684\u85e5\u7269\u5c0d\u9019\u985e\u60a3\u8005\u7684\u6548\u679c\u5fae\u4e4e\u5176\u5fae\uff0c\u60a3\u8005\u4ecd\u7136\u9762\u81e8\u60a3\u6025\u6027\u80f0\u817a\u708e\u7b49\u4f75\u767c\u75c7\u7684\u98a8\u96aa\uff1b\u56b4\u683c\u7684\u98f2\u98df\u63a7\u5236\u5118\u7ba1\u53ef\u4ee5\u901a\u904e\u6bcf\u65e5\u6975\u4f4e\u8102\u80aa\u7684\u98f2\u98df\u4f86\u5be6\u73fe\u4e73\u7cdc\u5fae\u7c92\u7684\u964d\u4f4e\uff0c\u4f46\u662f\u60a3\u8005\u5f80\u5f80\u5f88\u96e3\u9577\u671f\u5805\u6301\uff0c\u4f9d\u7136\u6301\u7e8c\u9762\u81e8\u56b4\u91cd\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u5e36\u4f86\u7684\u98a8\u96aa\u3002\u7814\u7a76\u767c\u73fe\uff0c\u4e00\u7a2e\u4e3b\u8981\u5728\u809d\u81df\u4e2d\u7522\u751f\u7684\u86cb\u767d\u8ceaAPOC3\u5728\u8abf\u7bc0\u8840\u6f3f\u7518\u6cb9\u4e09\u916f\u65b9\u9762\u767c\u63ee\u8457\u91cd\u8981\u4f5c\u7528\uff0c\u5c0d\u5927\u898f\u6a21\u4eba\u7fa4\u7684\u6aa2\u6e2c\u7814\u7a76\u767c\u73fe\uff0c\u651c\u5e36\u5929\u7136APOC3\u529f\u80fd\u7f3a\u5931\u7a81\u8b8a\u7684\u4eba\u7fa4\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u542b\u91cf\u8f03\u4f4e\u4e14\u4e0d\u8868\u73fe\u5176\u4ed6\u81e8\u5e8a\u75c7\u72c0\u3002\u96a8\u8457\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u7684\u767c\u5c55\uff0c\u901a\u904e\u5728\u57fa\u56e0\u5c64\u9762\u8abf\u63a7APOC3\u7684\u8868\u9054\u5f9e\u800c\u964d\u4f4e\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u7684\u6cbb\u7642\u7b56\u7565\u5f97\u4ee5\u5be6\u73fe\uff0c\u9019\u5c07\u70ba\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\\\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u60a3\u8005\u5e36\u4f86\u6cbb\u7652\u7684\u5e0c\u671b\u3002 \u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\u662f\u5229\u7528\u4e0a\u6d77\u79d1\u6280\u5927\u5b78\u81ea\u4e3b\u7814\u767c\u7684\u9ad8\u7cbe\u51c6\u8b8a\u5f62\u5f0f\u9e7c\u57fa\u7de8\u8f2f\u5668tBE\uff08transformer Base Editor\uff09\uff08Wang et al.\uff0cNat Cell Biol\uff0c2021\uff09\u958b\u767c\u7684\u91dd\u5c0d\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\\\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7684\u9996\u6b3e\u9ad4\u5167\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269\u3002tBE\u7531\u8102\u8cea\u7d0d\u7c73\u7c92\uff08Lipid Nanoparticle\uff0cLNP\uff09\u5305\u88dd\uff0c\u901a\u904e\u975c\u8108\u6ce8\u5c04\u7d66\u85e5\u905e\u9001\u81f3\u60a3\u8005\u809d\u81df\uff0c\u5c0d\u9776\u6a19\u57fa\u56e0APOC3\u9032\u884c\u7cbe\u51c6\u9e7c\u57fa\u7de8\u8f2f\uff0c\u6a21\u64ec\u5929\u7136APOC3\u529f\u80fd\u7f3a\u5931\u4eba\u7fa4\u4e2d\u7684\u6709\u76ca\u7a81\u8b8a\uff0c\u4e0b\u8abfAPOC3\u7684\u57fa\u56e0\u8868\u9054\uff0c\u9032\u800c\u9054\u5230\u964d\u4f4e\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u7684\u76ee\u7684\u3002\u901a\u904e\u57fa\u56e0\u6c34\u6e96\u7684\u7de8\u8f2f\uff0c\u8a72\u7642\u6cd5\u6709\u671b\u5be6\u73fe\\\"\u4e00\u6b21\u7d66\u85e5\uff0c\u7d42\u8eab\u6709\u6548\\\"\u7684\u964d\u7518\u6cb9\u4e09\u8102\u7684\u76ee\u6a19\u3002tBE\u5c6c\u65bc\u4e0b\u4e00\u4ee3\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\uff0c\u4e0d\u9700\u8981\u7834\u58deDNA\u96d9\u93c8\u5373\u53ef\u5be6\u73fe\u5c0d\u55ae\u9e7c\u57fa\u7a81\u8b8a\u9032\u884c\u7cbe\u51c6\u6821\u6b63\uff0c\u4e0d\u6703\u4fc3\u767cp53\u901a\u8def\u555f\u52d5\u548c\u67d3\u8272\u9ad4\u7570\u5e38\u7b49\u5b89\u5168\u98a8\u96aa\u3002\u76f8\u6bd4\u8f03\u5176\u4ed6\u57fa\u65bcCRISPR\u6280\u8853\u7684\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5\uff0cCS-121\u5b89\u5168\u6027\u66f4\u9ad8\uff0c\u4e0d\u6703\u5f15\u767c\u60a3\u8005\u57fa\u56e0\u7d44DNA\u5927\u7247\u6bb5\u7f3a\u5931\u3001\u67d3\u8272\u9ad4\u7a81\u8b8a\u3001\u812b\u9776\u7a81\u8b8a\u7b49\u98a8\u96aa\uff0c\u66f4\u4e0d\u6703\u5e36\u4f86\u7531CRISPR\\\/Cas9\u5207\u65b7DNA\u96d9\u93c8\u800c\u555f\u52d5p53\u901a\u8def\u5c0e\u81f4\u7684\u809d\u6bd2\u6027\u7b49\u526f\u53cd\u61c9\u3002\u5728\u524d\u671f\u7684\u52d5\u7269\u8a66\u9a57\u4e2d\uff0cCS-121\u7684\u5b89\u5168\u6027\u548c\u9577\u671f\u6709\u6548\u6027\u5df2\u7d93\u7372\u5f97\u9a57\u8b49\uff0c\u5728\u809d\u81df\u3001\u80ba\u3001\u808c\u8089\u3001\u813e\u81df\u3001\u5375\u5de2\u3001\u5fc3\u81df\u3001\u814e\u81df\u7b49\u5404\u7a2e\u7d44\u7e54\u4e2d\u5747\u6c92\u6709\u6aa2\u6e2c\u5230\u812b\u9776\u7de8\u8f2f\u3002 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lipase\uff0cLPL\uff09\u57fa\u56e0\u6216\u53c3\u8207\u8abf\u63a7LPL\u6d3b\u6027\u7684\u95dc\u9375\u57fa\u56e0\u767c\u751f\u96d9\u7b49\u4f4d\u57fa\u56e0\u7a81\u8b8a\uff0c\u5c0e\u81f4\u8102\u86cb\u767d\u8102\u80aa\u9176\u529f\u80fd\u53d7\u640d\uff0c\u4f7f\u7518\u6cb9\u4e09\u8102\u7684\u5206\u89e3\u4ee3\u8b1d\u7522\u751f\u969c\u7919\uff0c\u5f9e\u800c\u9020\u6210\u9ad4\u5167\u4e73\u7cdc\u5fae\u7c92\u7684\u84c4\u7a4d\uff0c\u901a\u5e38\u60a3\u8005\u7684\u7a7a\u8179TG\u6c34\u6e96\u226510 mmol\/L (885 mg\/dL)\uff0c\u5168\u7403\u767c\u75c5\u7387\u5728\u5341\u842c\u5206\u4e4b\u4e00\u5230\u767e\u842c\u5206\u4e4b\u4e00\u4e4b\u9593\uff1b\u800cMCS\u5247\u7531\u591a\u7a2e\u56e0\u7d20\u5f15\u8d77\u7684\u75c5\u7406\u72c0\u614b\uff0c\u5176\u767c\u75c5\u53d7\u907a\u50b3\u80cc\u666f\u3001\u751f\u6d3b\u7fd2\u6163\u6216\u5176\u4ed6\u4ee3\u8b1d\u75be\u75c5\u5171\u540c\u5f71\u97ff\uff0c\u5168\u7403\u767c\u75c5\u7387\u9ad8\u90541\/600\u3002 \u76ee\u524d\uff0c\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7684\u4e3b\u8981\u6cbb\u7642\u7b56\u7565\u662f\u5c07\u60a3\u8005\u7684\u7a7a\u8179\u7518\u6cb9\u4e09\u8102\uff08TG\uff09\u6c34\u6e96\u63a7\u5236\u5728\u6025\u6027\u80f0\u817a\u708e\u98a8\u96aa\u95be\u503c\u4ee5\u4e0b\uff08\u901a\u5e38\u9700&lt;5.7 mmol\/L\uff0c\u5373\u00a0500 mg\/dL\uff09\uff0c\u7136\u800c\u76ee\u524d\u5df2\u4e0a\u5e02\u7684\u5404\u985e\u964d\u7518\u6cb9\u4e09\u8102\u7684\u85e5\u7269\u5c0d\u9019\u985e\u60a3\u8005\u7684\u6548\u679c\u5fae\u4e4e\u5176\u5fae\uff0c\u60a3\u8005\u4ecd\u7136\u9762\u81e8\u60a3\u6025\u6027\u80f0\u817a\u708e\u7b49\u4f75\u767c\u75c7\u7684\u98a8\u96aa\uff1b\u56b4\u683c\u7684\u98f2\u98df\u63a7\u5236\u5118\u7ba1\u53ef\u4ee5\u901a\u904e\u6bcf\u65e5\u6975\u4f4e\u8102\u80aa\u7684\u98f2\u98df\u4f86\u5be6\u73fe\u4e73\u7cdc\u5fae\u7c92\u7684\u964d\u4f4e\uff0c\u4f46\u662f\u60a3\u8005\u5f80\u5f80\u5f88\u96e3\u9577\u671f\u5805\u6301\uff0c\u4f9d\u7136\u6301\u7e8c\u9762\u81e8\u56b4\u91cd\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u5e36\u4f86\u7684\u98a8\u96aa\u3002\u7814\u7a76\u767c\u73fe\uff0c\u4e00\u7a2e\u4e3b\u8981\u5728\u809d\u81df\u4e2d\u7522\u751f\u7684\u86cb\u767d\u8ceaAPOC3\u5728\u8abf\u7bc0\u8840\u6f3f\u7518\u6cb9\u4e09\u916f\u65b9\u9762\u767c\u63ee\u8457\u91cd\u8981\u4f5c\u7528\uff0c\u5c0d\u5927\u898f\u6a21\u4eba\u7fa4\u7684\u6aa2\u6e2c\u7814\u7a76\u767c\u73fe\uff0c\u651c\u5e36\u5929\u7136APOC3\u529f\u80fd\u7f3a\u5931\u7a81\u8b8a\u7684\u4eba\u7fa4\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u542b\u91cf\u8f03\u4f4e\u4e14\u4e0d\u8868\u73fe\u5176\u4ed6\u81e8\u5e8a\u75c7\u72c0\u3002\u96a8\u8457\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u7684\u767c\u5c55\uff0c\u901a\u904e\u5728\u57fa\u56e0\u5c64\u9762\u8abf\u63a7APOC3\u7684\u8868\u9054\u5f9e\u800c\u964d\u4f4e\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u7684\u6cbb\u7642\u7b56\u7565\u5f97\u4ee5\u5be6\u73fe\uff0c\u9019\u5c07\u70ba\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u60a3\u8005\u5e36\u4f86\u6cbb\u7652\u7684\u5e0c\u671b\u3002 \u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\u662f\u5229\u7528\u4e0a\u6d77\u79d1\u6280\u5927\u5b78\u81ea\u4e3b\u7814\u767c\u7684\u9ad8\u7cbe\u51c6\u8b8a\u5f62\u5f0f\u9e7c\u57fa\u7de8\u8f2f\u5668tBE\uff08transformer Base Editor\uff09\uff08Wang et al.\uff0cNat Cell Biol\uff0c2021\uff09\u958b\u767c\u7684\u91dd\u5c0d\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7684\u9996\u6b3e\u9ad4\u5167\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269\u3002tBE\u7531\u8102\u8cea\u7d0d\u7c73\u7c92\uff08Lipid 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Editor\uff09\u70ba\u4ee3\u8868\u7684\u81ea\u4e3bIP\u9e7c\u57fa\u7de8\u8f2f\u7cfb\u7d71\u5275\u5efa\u4e86\u591a\u7a2e\u7cbe\u51c6\u7642\u6cd5\uff0c\u91dd\u5c0d\u907a\u50b3\u75be\u75c5\u3001\u4ee3\u8b1d\u75be\u75c5\u3001\u5fc3\u8840\u7ba1\u75be\u75c5\u7b49\u4f48\u5c40\u4e86\u591a\u689d\u7ba1\u7dda\uff0c\u9996\u689d\u7ba1\u7dda\u5373\u5c07\u9032\u5165\u95dc\u9375\u6027\u81e8\u5e8a\u8a66\u9a57\uff08\u2161\/\u2162\u671f\u81e8\u5e8a\u8a66\u9a57\uff09\u968e\u6bb5\u3002 \u6b32\u77ad\u89e3\u66f4\u591a\u8cc7\u8a0a\uff0c\u8acb\u9ede\u64ca\u9032\u5165\u5b98\u7db2\u00a0www.correctsequence.com \u5a92\u9ad4\u806f\u7cfb\u4eba\uff1a\u5546\u52d9\u5408\u4f5c\uff1aBD@correctsequence.com\u81e8\u5e8a\u8a66\u9a57\uff1aCT@correctsequence.com","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/pingtungtimes.com.tw\/?p=379703","og_locale":"zh_TW","og_type":"article","og_title":"\u91cc\u7a0b\u7891\u7a81\u7834\uff01\u5168\u7403\u9996\u500bAPOC3\u57fa\u56e0\u7de8\u8f2f\u81e8\u5e8a\u6cbb\u7642\u9ad8\u8840\u8102\u7372\u5f97\u6210\u529f - \u5c4f\u6771\u6642\u5831\u96fb\u5b50\u5831","og_description":"\u6b63\u5e8f\u751f\u7269CS-121\u9996\u4f8b\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u60a3\u8005\u5b8c\u6210\u7d66\u85e5\uff0c\u986f\u793a\u826f\u597d\u5b89\u5168\u6027\u53ca\u986f\u8457\u7642\u6548 \u4e0a\u6d772025\u5e7411\u67087\u65e5 \/\u7f8e\u901a\u793e\/ -- \u7576\u5730\u6642\u95932025\u5e7411\u67086\u65e5\uff0c\u5c08\u6ce8\u65bc\u65b0\u578b\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u3001\u8655\u65bc\u81e8\u5e8a\u968e\u6bb5\u7684\u5275\u65b0\u751f\u7269\u91ab\u85e5\u79d1\u6280\u4f01\u696d\u6b63\u5e8f\u751f\u7269\uff08\u4e0a\u6d77\uff09\u5ba3\u4f48\uff0c\u8207\u5b89\u5fbd\u91ab\u79d1\u5927\u5b78\u7b2c\u4e00\u9644\u5c6c\u91ab\u9662\u5408\u4f5c\u958b\u5c55\u7684\u91dd\u5c0d\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7684\u9776\u5411APOC3\u7684\u9e7c\u57fa\u7de8\u8f2f\u85e5\u7269CS-121\u6ce8\u5c04\u6db2\u7684\u7814\u7a76\u8005\u767c\u8d77\u7684\u81e8\u5e8a\u7814\u7a76\uff08Investigator Initiated Trial, IIT\uff09\u6cbb\u7642\u7684\u9996\u4f4d\u60a3\u8005\u5df2\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662\u3002\u60a3\u8005\u60a3\u6709\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\uff0c\u6b77\u53f2\u7a7a\u8179\u7518\u6cb9\u4e09\u916f\uff08TG\uff09\u6c34\u6e96&gt;12.5 mmol\/L\uff0c\u591a\u6b21\u8a98\u767c\u6025\u6027\u80f0\u817a\u708e\u3002\u6b64\u6b21IIT\u8a66\u9a57\u70ba\u5291\u91cf\u722c\u5761\u8a66\u9a57\uff0c\u60a3\u8005\u5728\u55ae\u6b21\u6700\u4f4e\u5291\u91cf\u7d66\u85e5\u5f8c\u4e09\u5929\u5167\uff0c\u7a7a\u8179TG\u6c34\u6e96\u986f\u8457\u4e0b\u964d\uff0c\u4e14\u672a\u767c\u751f\u4e0d\u826f\u53cd\u61c9\u3002\u9019\u662f\u5168\u7403\u9996\u6b21\u901a\u904e\u9776\u5411\u9e7c\u57fa\u7de8\u8f2fAPOC3\u57fa\u56e0\u6cbb\u7642\u9ad8\u8840\u8102\u60a3\u8005\u7684\u6848\u4f8b\u3002   \u5168\u7403\u9996\u4f8b\u63a5\u53d7APOC3\u57fa\u56e0\u7de8\u8f2f\u6cbb\u7642\uff08\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\uff09\u7684\u9ad8\u8840\u8102\u60a3\u8005\uff08\u53f35\uff09\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662  \u5168\u7403\u9996\u4f8b\u63a5\u53d7APOC3\u57fa\u56e0\u7de8\u8f2f\u6cbb\u7642\uff08\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\uff09\u7684\u9ad8\u8840\u8102\u60a3\u8005\uff08\u53f35\uff09\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662 \u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u662f\u4e00\u7a2e\u56e0\u8840\u6db2\u4e2d\u4e73\u7cdc\u5fae\u7c92\u7570\u5e38\u5347\u9ad8\u5c0e\u81f4\u7684\u4ee3\u8b1d\u6027\u75be\u75c5\uff0c\u5e38\u8207\u8102\u8cea\u4ee3\u8b1d\u7d0a\u4e82\u76f8\u95dc\uff0c\u4e3b\u8981\u8868\u73fe\u70ba\u7a7a\u8179\u7518\u6cb9\u4e09\u916f\uff08TG\uff09\u6c34\u6e96\u6975\u5ea6\u5347\u9ad8\uff0c\u53ef\u80fd\u5f15\u767c\u4e0d\u53ef\u9810\u6e2c\u4e14\u6f5b\u5728\u81f4\u547d\u7684\u6025\u6027\u80f0\u817a\u708e\u7b49\u56b4\u91cd\u4f75\u767c\u75c7\uff0c\u662f\u56b4\u91cd\u9ad8\u7518\u6cb9\u4e09\u8102\u8840\u75c7\uff08Severe Hypertriglyceridemia\uff0csHTG\uff09\u6700\u56b4\u91cd\u7684\u4e00\u7a2e\u4e9e\u578b\u3002\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u5305\u542b\u5bb6\u65cf\u6027\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7d9c\u5408\u5f81\uff08Familial Chylomicronemia Syndrome\uff0cFCS\uff09\u548c\u591a\u56e0\u7d20\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7d9c\u5408\u5f81\uff08Multifactorial Chylomicronemia Syndrome\uff0cMCS\uff09\u3002\u5176\u4e2dFCS\u662f\u4e00\u7a2e\u7f55\u898b\u7684\u5e38\u67d3\u8272\u9ad4\u96b1\u6027\u907a\u50b3\u75c5\uff0c\u7531\u65bc\u8102\u86cb\u767d\u8102\u80aa\u9176\uff08Lipoprotein lipase\uff0cLPL\uff09\u57fa\u56e0\u6216\u53c3\u8207\u8abf\u63a7LPL\u6d3b\u6027\u7684\u95dc\u9375\u57fa\u56e0\u767c\u751f\u96d9\u7b49\u4f4d\u57fa\u56e0\u7a81\u8b8a\uff0c\u5c0e\u81f4\u8102\u86cb\u767d\u8102\u80aa\u9176\u529f\u80fd\u53d7\u640d\uff0c\u4f7f\u7518\u6cb9\u4e09\u8102\u7684\u5206\u89e3\u4ee3\u8b1d\u7522\u751f\u969c\u7919\uff0c\u5f9e\u800c\u9020\u6210\u9ad4\u5167\u4e73\u7cdc\u5fae\u7c92\u7684\u84c4\u7a4d\uff0c\u901a\u5e38\u60a3\u8005\u7684\u7a7a\u8179TG\u6c34\u6e96\u226510 mmol\/L (885 mg\/dL)\uff0c\u5168\u7403\u767c\u75c5\u7387\u5728\u5341\u842c\u5206\u4e4b\u4e00\u5230\u767e\u842c\u5206\u4e4b\u4e00\u4e4b\u9593\uff1b\u800cMCS\u5247\u7531\u591a\u7a2e\u56e0\u7d20\u5f15\u8d77\u7684\u75c5\u7406\u72c0\u614b\uff0c\u5176\u767c\u75c5\u53d7\u907a\u50b3\u80cc\u666f\u3001\u751f\u6d3b\u7fd2\u6163\u6216\u5176\u4ed6\u4ee3\u8b1d\u75be\u75c5\u5171\u540c\u5f71\u97ff\uff0c\u5168\u7403\u767c\u75c5\u7387\u9ad8\u90541\/600\u3002 \u76ee\u524d\uff0c\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7684\u4e3b\u8981\u6cbb\u7642\u7b56\u7565\u662f\u5c07\u60a3\u8005\u7684\u7a7a\u8179\u7518\u6cb9\u4e09\u8102\uff08TG\uff09\u6c34\u6e96\u63a7\u5236\u5728\u6025\u6027\u80f0\u817a\u708e\u98a8\u96aa\u95be\u503c\u4ee5\u4e0b\uff08\u901a\u5e38\u9700&lt;5.7 mmol\/L\uff0c\u5373\u00a0500 mg\/dL\uff09\uff0c\u7136\u800c\u76ee\u524d\u5df2\u4e0a\u5e02\u7684\u5404\u985e\u964d\u7518\u6cb9\u4e09\u8102\u7684\u85e5\u7269\u5c0d\u9019\u985e\u60a3\u8005\u7684\u6548\u679c\u5fae\u4e4e\u5176\u5fae\uff0c\u60a3\u8005\u4ecd\u7136\u9762\u81e8\u60a3\u6025\u6027\u80f0\u817a\u708e\u7b49\u4f75\u767c\u75c7\u7684\u98a8\u96aa\uff1b\u56b4\u683c\u7684\u98f2\u98df\u63a7\u5236\u5118\u7ba1\u53ef\u4ee5\u901a\u904e\u6bcf\u65e5\u6975\u4f4e\u8102\u80aa\u7684\u98f2\u98df\u4f86\u5be6\u73fe\u4e73\u7cdc\u5fae\u7c92\u7684\u964d\u4f4e\uff0c\u4f46\u662f\u60a3\u8005\u5f80\u5f80\u5f88\u96e3\u9577\u671f\u5805\u6301\uff0c\u4f9d\u7136\u6301\u7e8c\u9762\u81e8\u56b4\u91cd\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u5e36\u4f86\u7684\u98a8\u96aa\u3002\u7814\u7a76\u767c\u73fe\uff0c\u4e00\u7a2e\u4e3b\u8981\u5728\u809d\u81df\u4e2d\u7522\u751f\u7684\u86cb\u767d\u8ceaAPOC3\u5728\u8abf\u7bc0\u8840\u6f3f\u7518\u6cb9\u4e09\u916f\u65b9\u9762\u767c\u63ee\u8457\u91cd\u8981\u4f5c\u7528\uff0c\u5c0d\u5927\u898f\u6a21\u4eba\u7fa4\u7684\u6aa2\u6e2c\u7814\u7a76\u767c\u73fe\uff0c\u651c\u5e36\u5929\u7136APOC3\u529f\u80fd\u7f3a\u5931\u7a81\u8b8a\u7684\u4eba\u7fa4\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u542b\u91cf\u8f03\u4f4e\u4e14\u4e0d\u8868\u73fe\u5176\u4ed6\u81e8\u5e8a\u75c7\u72c0\u3002\u96a8\u8457\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u7684\u767c\u5c55\uff0c\u901a\u904e\u5728\u57fa\u56e0\u5c64\u9762\u8abf\u63a7APOC3\u7684\u8868\u9054\u5f9e\u800c\u964d\u4f4e\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u7684\u6cbb\u7642\u7b56\u7565\u5f97\u4ee5\u5be6\u73fe\uff0c\u9019\u5c07\u70ba\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u60a3\u8005\u5e36\u4f86\u6cbb\u7652\u7684\u5e0c\u671b\u3002 \u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\u662f\u5229\u7528\u4e0a\u6d77\u79d1\u6280\u5927\u5b78\u81ea\u4e3b\u7814\u767c\u7684\u9ad8\u7cbe\u51c6\u8b8a\u5f62\u5f0f\u9e7c\u57fa\u7de8\u8f2f\u5668tBE\uff08transformer Base Editor\uff09\uff08Wang et al.\uff0cNat Cell Biol\uff0c2021\uff09\u958b\u767c\u7684\u91dd\u5c0d\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7684\u9996\u6b3e\u9ad4\u5167\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269\u3002tBE\u7531\u8102\u8cea\u7d0d\u7c73\u7c92\uff08Lipid Nanoparticle\uff0cLNP\uff09\u5305\u88dd\uff0c\u901a\u904e\u975c\u8108\u6ce8\u5c04\u7d66\u85e5\u905e\u9001\u81f3\u60a3\u8005\u809d\u81df\uff0c\u5c0d\u9776\u6a19\u57fa\u56e0APOC3\u9032\u884c\u7cbe\u51c6\u9e7c\u57fa\u7de8\u8f2f\uff0c\u6a21\u64ec\u5929\u7136APOC3\u529f\u80fd\u7f3a\u5931\u4eba\u7fa4\u4e2d\u7684\u6709\u76ca\u7a81\u8b8a\uff0c\u4e0b\u8abfAPOC3\u7684\u57fa\u56e0\u8868\u9054\uff0c\u9032\u800c\u9054\u5230\u964d\u4f4e\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u7684\u76ee\u7684\u3002\u901a\u904e\u57fa\u56e0\u6c34\u6e96\u7684\u7de8\u8f2f\uff0c\u8a72\u7642\u6cd5\u6709\u671b\u5be6\u73fe\"\u4e00\u6b21\u7d66\u85e5\uff0c\u7d42\u8eab\u6709\u6548\"\u7684\u964d\u7518\u6cb9\u4e09\u8102\u7684\u76ee\u6a19\u3002tBE\u5c6c\u65bc\u4e0b\u4e00\u4ee3\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\uff0c\u4e0d\u9700\u8981\u7834\u58deDNA\u96d9\u93c8\u5373\u53ef\u5be6\u73fe\u5c0d\u55ae\u9e7c\u57fa\u7a81\u8b8a\u9032\u884c\u7cbe\u51c6\u6821\u6b63\uff0c\u4e0d\u6703\u4fc3\u767cp53\u901a\u8def\u555f\u52d5\u548c\u67d3\u8272\u9ad4\u7570\u5e38\u7b49\u5b89\u5168\u98a8\u96aa\u3002\u76f8\u6bd4\u8f03\u5176\u4ed6\u57fa\u65bcCRISPR\u6280\u8853\u7684\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5\uff0cCS-121\u5b89\u5168\u6027\u66f4\u9ad8\uff0c\u4e0d\u6703\u5f15\u767c\u60a3\u8005\u57fa\u56e0\u7d44DNA\u5927\u7247\u6bb5\u7f3a\u5931\u3001\u67d3\u8272\u9ad4\u7a81\u8b8a\u3001\u812b\u9776\u7a81\u8b8a\u7b49\u98a8\u96aa\uff0c\u66f4\u4e0d\u6703\u5e36\u4f86\u7531CRISPR\/Cas9\u5207\u65b7DNA\u96d9\u93c8\u800c\u555f\u52d5p53\u901a\u8def\u5c0e\u81f4\u7684\u809d\u6bd2\u6027\u7b49\u526f\u53cd\u61c9\u3002\u5728\u524d\u671f\u7684\u52d5\u7269\u8a66\u9a57\u4e2d\uff0cCS-121\u7684\u5b89\u5168\u6027\u548c\u9577\u671f\u6709\u6548\u6027\u5df2\u7d93\u7372\u5f97\u9a57\u8b49\uff0c\u5728\u809d\u81df\u3001\u80ba\u3001\u808c\u8089\u3001\u813e\u81df\u3001\u5375\u5de2\u3001\u5fc3\u81df\u3001\u814e\u81df\u7b49\u5404\u7a2e\u7d44\u7e54\u4e2d\u5747\u6c92\u6709\u6aa2\u6e2c\u5230\u812b\u9776\u7de8\u8f2f\u3002 \u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\u6cbb\u7642\u7684\u9996\u4f4d\u60a3\u8005\u662f\u4e00\u540d\u60a3\u6709\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u768463\u6b72\u7537\u6027\u3002\u60a3\u8005\u57282025\u5e7410\u670818\u65e5\u63a5\u53d7CS-121\u6700\u4f4e\u5291\u91cf\u55ae\u6b21\u7d66\u85e5\uff0c\u7d66\u85e5\u5f8c\u4e09\u5929\u5167\uff0c\u7a7a\u8179TG\u6c34\u6e96\u986f\u8457\u4e0b\u964d\uff0c\u4e26\u65bc\u7d66\u85e5\u5f8c\u7b2c3\u5929\u9806\u5229\u51fa\u9662\u3002\u622a\u81f3\u5831\u5c0e\u65e5\u671f\uff0c\u672a\u51fa\u73fe\u7522\u54c1\u76f8\u95dc\u7684\u4e0d\u826f\u4e8b\u4ef6\u3002 \u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2IIT\u5c08\u6848\u7684\u4e3b\u8981\u7814\u7a76\u8005\u70ba\u5b89\u5fbd\u91ab\u79d1\u5927\u5b78\u7b2c\u4e00\u9644\u5c6c\u91ab\u9662\u5468\u7165\u6559\u6388\uff0c\u5433\u5fd7\u9e97\u4e3b\u4efb\u3002 \u6b63\u5e8f\u751f\u7269\u662f\u4e00\u5bb6\u5df2\u7d93\u9032\u5165IND\u81e8\u5e8a\u968e\u6bb5\u7684\u5275\u65b0\u751f\u7269\u91ab\u85e5\u79d1\u6280\u516c\u53f8\uff0c\u5176\u958b\u767c\u7684\u9996\u6b3e\u9ad4\u5916\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269CS-101\u6ce8\u5c04\u6db2\u5df2\u7d93\u6210\u529f\u6cbb\u7652\u6578\u5341\u4f8b\u03b2-\u5730\u4e2d\u6d77\u8ca7\u8840\u548c\u942e\u5200\u578b\u7d30\u80de\u8ca7\u8840\u75c5\u60a3\u8005\u3002\u6b63\u5e8f\u751f\u7269\u6b63\u5728\u52a0\u901f\u63a8\u9032\u9996\u6b3e\u9ad4\u5167\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269CS-121\u6ce8\u5c04\u6db2\u9032\u5165IND\u81e8\u5e8a\u53ca\u4e0a\u5e02\uff0c\u6709\u671b\u70ba\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7b49\u4ee3\u8b1d\u985e\u75be\u75c5\u60a3\u8005\u5e36\u4f86\"\u4e00\u6b21\u6cbb\u7642\uff0c\u7d42\u8eab\u6709\u6548\"\u7684\u985b\u8986\u6027\u6cbb\u7642\u65b9\u6848\u3002 \uff08\u7279\u5225\u611f\u8b1d\u5b89\u5fbd\u91ab\u79d1\u5927\u5b78\u7b2c\u4e00\u9644\u5c6c\u91ab\u9662\u3001\u4e0a\u6d77\u79d1\u6280\u5927\u5b78\u53ca\u4e0a\u6d77\u81e8\u5e8a\u7814\u7a76\u4e2d\u5fc3\uff09 \u95dc\u65bc\u6b63\u5e8f\u751f\u7269 \u6b63\u5e8f\u751f\u7269\uff08CorrectSequence Therapeutics\uff09\u662f\u4e00\u5bb6\u5c08\u6ce8\u65bc\u65b0\u578b\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u3001\u8655\u65bc\u81e8\u5e8a\u968e\u6bb5\u7684\u751f\u7269\u91ab\u85e5\u79d1\u6280\u516c\u53f8\uff0c\u5b75\u5316\u81ea\u4e0a\u6d77\u79d1\u6280\u5927\u5b78\uff0c\u81f4\u529b\u65bc\u5229\u7528\u81ea\u4e3b\u77e5\u8b58\u7522\u6b0a\u7684\u9e7c\u57fa\u7de8\u8f2f\u9ad4\u7cfb\uff0c\u958b\u767c\u7a81\u7834\u6027\u7cbe\u51c6\u7642\u6cd5\uff0c\u9020\u798f\u4eba\u985e\u5065\u5eb7\u3002\u516c\u53f8\u57fa\u65bc\u4ee5\u8b8a\u5f62\u5f0f\u9e7c\u57fa\u7de8\u8f2f\u5668tBE\uff08transformer Base 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\u7576\u5730\u6642\u95932025\u5e7411\u67086\u65e5\uff0c\u5c08\u6ce8\u65bc\u65b0\u578b\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u3001\u8655\u65bc\u81e8\u5e8a\u968e\u6bb5\u7684\u5275\u65b0\u751f\u7269\u91ab\u85e5\u79d1\u6280\u4f01\u696d\u6b63\u5e8f\u751f\u7269\uff08\u4e0a\u6d77\uff09\u5ba3\u4f48\uff0c\u8207\u5b89\u5fbd\u91ab\u79d1\u5927\u5b78\u7b2c\u4e00\u9644\u5c6c\u91ab\u9662\u5408\u4f5c\u958b\u5c55\u7684\u91dd\u5c0d\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7684\u9776\u5411APOC3\u7684\u9e7c\u57fa\u7de8\u8f2f\u85e5\u7269CS-121\u6ce8\u5c04\u6db2\u7684\u7814\u7a76\u8005\u767c\u8d77\u7684\u81e8\u5e8a\u7814\u7a76\uff08Investigator Initiated Trial, IIT\uff09\u6cbb\u7642\u7684\u9996\u4f4d\u60a3\u8005\u5df2\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662\u3002\u60a3\u8005\u60a3\u6709\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\uff0c\u6b77\u53f2\u7a7a\u8179\u7518\u6cb9\u4e09\u916f\uff08TG\uff09\u6c34\u6e96&gt;12.5 mmol\/L\uff0c\u591a\u6b21\u8a98\u767c\u6025\u6027\u80f0\u817a\u708e\u3002\u6b64\u6b21IIT\u8a66\u9a57\u70ba\u5291\u91cf\u722c\u5761\u8a66\u9a57\uff0c\u60a3\u8005\u5728\u55ae\u6b21\u6700\u4f4e\u5291\u91cf\u7d66\u85e5\u5f8c\u4e09\u5929\u5167\uff0c\u7a7a\u8179TG\u6c34\u6e96\u986f\u8457\u4e0b\u964d\uff0c\u4e14\u672a\u767c\u751f\u4e0d\u826f\u53cd\u61c9\u3002\u9019\u662f\u5168\u7403\u9996\u6b21\u901a\u904e\u9776\u5411\u9e7c\u57fa\u7de8\u8f2fAPOC3\u57fa\u56e0\u6cbb\u7642\u9ad8\u8840\u8102\u60a3\u8005\u7684\u6848\u4f8b\u3002 \u5168\u7403\u9996\u4f8b\u63a5\u53d7APOC3\u57fa\u56e0\u7de8\u8f2f\u6cbb\u7642\uff08\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\uff09\u7684\u9ad8\u8840\u8102\u60a3\u8005\uff08\u53f35\uff09\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662 \u5168\u7403\u9996\u4f8b\u63a5\u53d7APOC3\u57fa\u56e0\u7de8\u8f2f\u6cbb\u7642\uff08\u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\uff09\u7684\u9ad8\u8840\u8102\u60a3\u8005\uff08\u53f35\uff09\u5b8c\u6210\u7d66\u85e5\u4e26\u9806\u5229\u51fa\u9662 \u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u662f\u4e00\u7a2e\u56e0\u8840\u6db2\u4e2d\u4e73\u7cdc\u5fae\u7c92\u7570\u5e38\u5347\u9ad8\u5c0e\u81f4\u7684\u4ee3\u8b1d\u6027\u75be\u75c5\uff0c\u5e38\u8207\u8102\u8cea\u4ee3\u8b1d\u7d0a\u4e82\u76f8\u95dc\uff0c\u4e3b\u8981\u8868\u73fe\u70ba\u7a7a\u8179\u7518\u6cb9\u4e09\u916f\uff08TG\uff09\u6c34\u6e96\u6975\u5ea6\u5347\u9ad8\uff0c\u53ef\u80fd\u5f15\u767c\u4e0d\u53ef\u9810\u6e2c\u4e14\u6f5b\u5728\u81f4\u547d\u7684\u6025\u6027\u80f0\u817a\u708e\u7b49\u56b4\u91cd\u4f75\u767c\u75c7\uff0c\u662f\u56b4\u91cd\u9ad8\u7518\u6cb9\u4e09\u8102\u8840\u75c7\uff08Severe Hypertriglyceridemia\uff0csHTG\uff09\u6700\u56b4\u91cd\u7684\u4e00\u7a2e\u4e9e\u578b\u3002\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u5305\u542b\u5bb6\u65cf\u6027\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7d9c\u5408\u5f81\uff08Familial Chylomicronemia Syndrome\uff0cFCS\uff09\u548c\u591a\u56e0\u7d20\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7d9c\u5408\u5f81\uff08Multifactorial Chylomicronemia Syndrome\uff0cMCS\uff09\u3002\u5176\u4e2dFCS\u662f\u4e00\u7a2e\u7f55\u898b\u7684\u5e38\u67d3\u8272\u9ad4\u96b1\u6027\u907a\u50b3\u75c5\uff0c\u7531\u65bc\u8102\u86cb\u767d\u8102\u80aa\u9176\uff08Lipoprotein lipase\uff0cLPL\uff09\u57fa\u56e0\u6216\u53c3\u8207\u8abf\u63a7LPL\u6d3b\u6027\u7684\u95dc\u9375\u57fa\u56e0\u767c\u751f\u96d9\u7b49\u4f4d\u57fa\u56e0\u7a81\u8b8a\uff0c\u5c0e\u81f4\u8102\u86cb\u767d\u8102\u80aa\u9176\u529f\u80fd\u53d7\u640d\uff0c\u4f7f\u7518\u6cb9\u4e09\u8102\u7684\u5206\u89e3\u4ee3\u8b1d\u7522\u751f\u969c\u7919\uff0c\u5f9e\u800c\u9020\u6210\u9ad4\u5167\u4e73\u7cdc\u5fae\u7c92\u7684\u84c4\u7a4d\uff0c\u901a\u5e38\u60a3\u8005\u7684\u7a7a\u8179TG\u6c34\u6e96\u226510 mmol\/L (885 mg\/dL)\uff0c\u5168\u7403\u767c\u75c5\u7387\u5728\u5341\u842c\u5206\u4e4b\u4e00\u5230\u767e\u842c\u5206\u4e4b\u4e00\u4e4b\u9593\uff1b\u800cMCS\u5247\u7531\u591a\u7a2e\u56e0\u7d20\u5f15\u8d77\u7684\u75c5\u7406\u72c0\u614b\uff0c\u5176\u767c\u75c5\u53d7\u907a\u50b3\u80cc\u666f\u3001\u751f\u6d3b\u7fd2\u6163\u6216\u5176\u4ed6\u4ee3\u8b1d\u75be\u75c5\u5171\u540c\u5f71\u97ff\uff0c\u5168\u7403\u767c\u75c5\u7387\u9ad8\u90541\/600\u3002 \u76ee\u524d\uff0c\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u7684\u4e3b\u8981\u6cbb\u7642\u7b56\u7565\u662f\u5c07\u60a3\u8005\u7684\u7a7a\u8179\u7518\u6cb9\u4e09\u8102\uff08TG\uff09\u6c34\u6e96\u63a7\u5236\u5728\u6025\u6027\u80f0\u817a\u708e\u98a8\u96aa\u95be\u503c\u4ee5\u4e0b\uff08\u901a\u5e38\u9700&lt;5.7 mmol\/L\uff0c\u5373\u00a0500 mg\/dL\uff09\uff0c\u7136\u800c\u76ee\u524d\u5df2\u4e0a\u5e02\u7684\u5404\u985e\u964d\u7518\u6cb9\u4e09\u8102\u7684\u85e5\u7269\u5c0d\u9019\u985e\u60a3\u8005\u7684\u6548\u679c\u5fae\u4e4e\u5176\u5fae\uff0c\u60a3\u8005\u4ecd\u7136\u9762\u81e8\u60a3\u6025\u6027\u80f0\u817a\u708e\u7b49\u4f75\u767c\u75c7\u7684\u98a8\u96aa\uff1b\u56b4\u683c\u7684\u98f2\u98df\u63a7\u5236\u5118\u7ba1\u53ef\u4ee5\u901a\u904e\u6bcf\u65e5\u6975\u4f4e\u8102\u80aa\u7684\u98f2\u98df\u4f86\u5be6\u73fe\u4e73\u7cdc\u5fae\u7c92\u7684\u964d\u4f4e\uff0c\u4f46\u662f\u60a3\u8005\u5f80\u5f80\u5f88\u96e3\u9577\u671f\u5805\u6301\uff0c\u4f9d\u7136\u6301\u7e8c\u9762\u81e8\u56b4\u91cd\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u5e36\u4f86\u7684\u98a8\u96aa\u3002\u7814\u7a76\u767c\u73fe\uff0c\u4e00\u7a2e\u4e3b\u8981\u5728\u809d\u81df\u4e2d\u7522\u751f\u7684\u86cb\u767d\u8ceaAPOC3\u5728\u8abf\u7bc0\u8840\u6f3f\u7518\u6cb9\u4e09\u916f\u65b9\u9762\u767c\u63ee\u8457\u91cd\u8981\u4f5c\u7528\uff0c\u5c0d\u5927\u898f\u6a21\u4eba\u7fa4\u7684\u6aa2\u6e2c\u7814\u7a76\u767c\u73fe\uff0c\u651c\u5e36\u5929\u7136APOC3\u529f\u80fd\u7f3a\u5931\u7a81\u8b8a\u7684\u4eba\u7fa4\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u542b\u91cf\u8f03\u4f4e\u4e14\u4e0d\u8868\u73fe\u5176\u4ed6\u81e8\u5e8a\u75c7\u72c0\u3002\u96a8\u8457\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\u7684\u767c\u5c55\uff0c\u901a\u904e\u5728\u57fa\u56e0\u5c64\u9762\u8abf\u63a7APOC3\u7684\u8868\u9054\u5f9e\u800c\u964d\u4f4e\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u7684\u6cbb\u7642\u7b56\u7565\u5f97\u4ee5\u5be6\u73fe\uff0c\u9019\u5c07\u70ba\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u60a3\u8005\u5e36\u4f86\u6cbb\u7652\u7684\u5e0c\u671b\u3002 \u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\u662f\u5229\u7528\u4e0a\u6d77\u79d1\u6280\u5927\u5b78\u81ea\u4e3b\u7814\u767c\u7684\u9ad8\u7cbe\u51c6\u8b8a\u5f62\u5f0f\u9e7c\u57fa\u7de8\u8f2f\u5668tBE\uff08transformer Base Editor\uff09\uff08Wang et al.\uff0cNat Cell Biol\uff0c2021\uff09\u958b\u767c\u7684\u91dd\u5c0d\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7684\u9996\u6b3e\u9ad4\u5167\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269\u3002tBE\u7531\u8102\u8cea\u7d0d\u7c73\u7c92\uff08Lipid Nanoparticle\uff0cLNP\uff09\u5305\u88dd\uff0c\u901a\u904e\u975c\u8108\u6ce8\u5c04\u7d66\u85e5\u905e\u9001\u81f3\u60a3\u8005\u809d\u81df\uff0c\u5c0d\u9776\u6a19\u57fa\u56e0APOC3\u9032\u884c\u7cbe\u51c6\u9e7c\u57fa\u7de8\u8f2f\uff0c\u6a21\u64ec\u5929\u7136APOC3\u529f\u80fd\u7f3a\u5931\u4eba\u7fa4\u4e2d\u7684\u6709\u76ca\u7a81\u8b8a\uff0c\u4e0b\u8abfAPOC3\u7684\u57fa\u56e0\u8868\u9054\uff0c\u9032\u800c\u9054\u5230\u964d\u4f4e\u7518\u6cb9\u4e09\u916f\u6c34\u6e96\u7684\u76ee\u7684\u3002\u901a\u904e\u57fa\u56e0\u6c34\u6e96\u7684\u7de8\u8f2f\uff0c\u8a72\u7642\u6cd5\u6709\u671b\u5be6\u73fe\"\u4e00\u6b21\u7d66\u85e5\uff0c\u7d42\u8eab\u6709\u6548\"\u7684\u964d\u7518\u6cb9\u4e09\u8102\u7684\u76ee\u6a19\u3002tBE\u5c6c\u65bc\u4e0b\u4e00\u4ee3\u57fa\u56e0\u7de8\u8f2f\u6280\u8853\uff0c\u4e0d\u9700\u8981\u7834\u58deDNA\u96d9\u93c8\u5373\u53ef\u5be6\u73fe\u5c0d\u55ae\u9e7c\u57fa\u7a81\u8b8a\u9032\u884c\u7cbe\u51c6\u6821\u6b63\uff0c\u4e0d\u6703\u4fc3\u767cp53\u901a\u8def\u555f\u52d5\u548c\u67d3\u8272\u9ad4\u7570\u5e38\u7b49\u5b89\u5168\u98a8\u96aa\u3002\u76f8\u6bd4\u8f03\u5176\u4ed6\u57fa\u65bcCRISPR\u6280\u8853\u7684\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5\uff0cCS-121\u5b89\u5168\u6027\u66f4\u9ad8\uff0c\u4e0d\u6703\u5f15\u767c\u60a3\u8005\u57fa\u56e0\u7d44DNA\u5927\u7247\u6bb5\u7f3a\u5931\u3001\u67d3\u8272\u9ad4\u7a81\u8b8a\u3001\u812b\u9776\u7a81\u8b8a\u7b49\u98a8\u96aa\uff0c\u66f4\u4e0d\u6703\u5e36\u4f86\u7531CRISPR\/Cas9\u5207\u65b7DNA\u96d9\u93c8\u800c\u555f\u52d5p53\u901a\u8def\u5c0e\u81f4\u7684\u809d\u6bd2\u6027\u7b49\u526f\u53cd\u61c9\u3002\u5728\u524d\u671f\u7684\u52d5\u7269\u8a66\u9a57\u4e2d\uff0cCS-121\u7684\u5b89\u5168\u6027\u548c\u9577\u671f\u6709\u6548\u6027\u5df2\u7d93\u7372\u5f97\u9a57\u8b49\uff0c\u5728\u809d\u81df\u3001\u80ba\u3001\u808c\u8089\u3001\u813e\u81df\u3001\u5375\u5de2\u3001\u5fc3\u81df\u3001\u814e\u81df\u7b49\u5404\u7a2e\u7d44\u7e54\u4e2d\u5747\u6c92\u6709\u6aa2\u6e2c\u5230\u812b\u9776\u7de8\u8f2f\u3002 \u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2\u6cbb\u7642\u7684\u9996\u4f4d\u60a3\u8005\u662f\u4e00\u540d\u60a3\u6709\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\u768463\u6b72\u7537\u6027\u3002\u60a3\u8005\u57282025\u5e7410\u670818\u65e5\u63a5\u53d7CS-121\u6700\u4f4e\u5291\u91cf\u55ae\u6b21\u7d66\u85e5\uff0c\u7d66\u85e5\u5f8c\u4e09\u5929\u5167\uff0c\u7a7a\u8179TG\u6c34\u6e96\u986f\u8457\u4e0b\u964d\uff0c\u4e26\u65bc\u7d66\u85e5\u5f8c\u7b2c3\u5929\u9806\u5229\u51fa\u9662\u3002\u622a\u81f3\u5831\u5c0e\u65e5\u671f\uff0c\u672a\u51fa\u73fe\u7522\u54c1\u76f8\u95dc\u7684\u4e0d\u826f\u4e8b\u4ef6\u3002 \u6b63\u5e8f\u751f\u7269CS-121\u6ce8\u5c04\u6db2IIT\u5c08\u6848\u7684\u4e3b\u8981\u7814\u7a76\u8005\u70ba\u5b89\u5fbd\u91ab\u79d1\u5927\u5b78\u7b2c\u4e00\u9644\u5c6c\u91ab\u9662\u5468\u7165\u6559\u6388\uff0c\u5433\u5fd7\u9e97\u4e3b\u4efb\u3002 \u6b63\u5e8f\u751f\u7269\u662f\u4e00\u5bb6\u5df2\u7d93\u9032\u5165IND\u81e8\u5e8a\u968e\u6bb5\u7684\u5275\u65b0\u751f\u7269\u91ab\u85e5\u79d1\u6280\u516c\u53f8\uff0c\u5176\u958b\u767c\u7684\u9996\u6b3e\u9ad4\u5916\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269CS-101\u6ce8\u5c04\u6db2\u5df2\u7d93\u6210\u529f\u6cbb\u7652\u6578\u5341\u4f8b\u03b2-\u5730\u4e2d\u6d77\u8ca7\u8840\u548c\u942e\u5200\u578b\u7d30\u80de\u8ca7\u8840\u75c5\u60a3\u8005\u3002\u6b63\u5e8f\u751f\u7269\u6b63\u5728\u52a0\u901f\u63a8\u9032\u9996\u6b3e\u9ad4\u5167\u57fa\u56e0\u7de8\u8f2f\u85e5\u7269CS-121\u6ce8\u5c04\u6db2\u9032\u5165IND\u81e8\u5e8a\u53ca\u4e0a\u5e02\uff0c\u6709\u671b\u70ba\u9ad8\u4e73\u7cdc\u5fae\u7c92\u8840\u75c7\/\u9ad8\u7518\u6cb9\u4e09\u916f\u8840\u75c7\u7b49\u4ee3\u8b1d\u985e\u75be\u75c5\u60a3\u8005\u5e36\u4f86\"\u4e00\u6b21\u6cbb\u7642\uff0c\u7d42\u8eab\u6709\u6548\"\u7684\u985b\u8986\u6027\u6cbb\u7642\u65b9\u6848\u3002 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