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科研进展

亚博买球在基因治疗遗传代谢性肝病领域取得重要进展

发表日期:2020-12-11来源:放大 缩小

  11YUE19RI,ZHONGGUOKEXUEYUANGUANGZHOUSHENGWUYIYAOYUJIANKANGYANJIUYUANLAILIANGXUEKETIZUZAIGUOJIXUESHUQIKANMolecular Therapy(《FENZIZHILIAO》)ZAIXIANFABIAOLETIWEI“CRISPR/Cas9-mediated gene correction in newborn rabbits with hereditary tyrosinemia type I”DEYANJIULUNWEN。GAIYANJIUYINGYONGCRISPR/Cas9XITONGCHENGGONGSHIXIANLEDUIⅠXINGGAOLAOANSUANXUEZHENGJIATUMOXINGDEZAITIJIYINZHILIAO,JIANGTUIJINCRISPR/Cas9XITONGJIEDAODEZAITIJIYINZHILIAOCELVEDELINCHUANGYINGYONG。

  ⅠXINGGAOLAOANSUANXUEZHENG(JIANCHENGHT-I)SHIYOUYANHUSUOXIANYIXIANYISUANSHUIJIEMEI(FAH)QUESHISUODAOZHIDEYIZHONGDIANXINGDEYOUJIYINTUBIANYINQIDECHANGRANSETIYINXINGYICHUANDAIXIEJIBING,HUANZHEZAIYINGYOUERQIJIHUICHUXIANBUKENIDEGANSHENSUNHAI。JIYUXIAOSHUMOXINGDEYANJIUBIAOMING,CRISPR/Cas9XITONGJIEDAODEZAITIJIYINZHILIAOCELVENENGGOUJIUZHENGJIYINTUBIANBINGHUIFUDANBAIBIAODACONGERZHILIAOHT-I。DANYOUYUXIAOSHUMOXINGBUNENGWANQUANMONIRENLEIHT-IJIBINGDEZHENGZHUANG,RUWUJINXINGXINGGANYINGHUAYIJIDUOZHONGSHENZANGBINGBIAN。WEILEMIBUZHEIXIEQUEXIAN,XUYAOYINGYONGBIAOXINGGENGHAODEDADONGWUJIBINGMOXINGDUIIXINGGAOLAOANSUANXUEZHENGDEZAITIJIYINZHILIAOFANGANJINXINGYANJIU。

  ZAIBENYANJIUZHONG,YANJIURENYUANJIANGHANYOUCas9,sgRNAJIXIUFUMOBANDEZHONGZUXIANXIANGGUANBINGDUJINGERJINGMAIZHURUSHIWURILINGDEXINSHENGZITUTINEI。5GEYUEHOU,ZHILIAOZUYANGBENGANZANGZHONG,TUBIANJIYINDEDAOXIUFUDEGANXIBAOBILIKEDA5.06%ZHI8.20%,ZHEIXIEBEIXIUFUDEGANXIBAODEFAHDANBAIDEYIBIAODA,BEIZHILIAOHOUDEHT-IJIATUGEXIANGSHENGLISHENGHUAZHIBIAOYIJIGANZANGHESHENZANGJIEGOUJUNWEICHUXIANMINGXIANYICHANGBIANHUA。ZHILIAOYANGBENDEGANZANGZHUANLUZUYEDEYIHUIFUZHIZHENGCHANGBIAODASHUIPING,SUOYOUJIESHOUZHILIAODEYANGBENJUNNENGGOUZHENGCHANGCHENGZHANGZHIXINGCHENGSHUBINGFANZHIHOUDAI。ZHENGMING,CRISPR/Cas9XITONGJIEDAODEZAITIJIYINLIAOFAKEYOUXIAODEZHILIAOHT-IJIBING。LINGWAI,JINGJIYINJIANCE,ZAIBEIZHILIAOHT-IJIATUZHONG,WEICHUXIANYOUCas9YINQIDETUOBAXIAOYING,XIANSHIGAIJIYINZHILIAOFANGFAKENENGSHIANQUANDE。

  BENKETISHOUCISHIXIANLEYINGYONGCRISPR/Cas9JIEDAODETINEIJIYINZHILIAOFANGANDUIYOUNIANQIHT-IJIATUMOXINGJINXINGZAITIJIYINZHILIAO。GAIYANJIUCHENGGUOMIBULELIYONGXIAOXINGNIECHILEIDONGWUJINXINGJIYINZHILIAOYANJIUDEQUEXIAN,WEIPINGGUJIYINZHILIAODEANQUANXINGJIYOUXIAOXINGTIGONGLEXINDEZHENGJU,YEWEIHOUXUJIANGZAITIJIYINZHILIAOYINGYONGYUZHILIAOQITAGANZANGXIANGGUANDEYICHUANDAIXIEJIBINGTIGONGLECANKAOFANGAN。

  GAILUNWENDEGONGTONGTONGXUNZUOZHEWEIWANGKEPINFUYANJIUYUAN、LAILIANGXUEYANJIUYUANJIWUHANZHULIYANJIUYUAN。GUANGZHOUSHENGWUYIYAOYUJIANKANGYANJIUYUANBOSHIHOULINAN、BOSHIYANJIUSHENGGOUSHIXUEJIWANGJIAOWEIWEIGONGTONGDIYIZUOZHE。YANJIUGONGZUODEDAOGUOJIAZHONGDIANYANFAJIHUA、GUOJIAZIRANKEXUEJIJIN、SHENGWUDAOSHIYANSHI、ZHONGGUOYIXUEKEXUEYUANCHUANGXINDANYUAN、GUANGDONGSHENGHEGUANGZHOUSHIKEJIJIHUAJIZHONGGUOKEXUEYUANQINGNIANCHUANGXINCUJINHUIDENGDUOGEXIANGMUDEZHICHI。

  

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附件:
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