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生科院华益民教授课题组在《Nucleic Acids Research》上发表研究论文

yabo登录JINRI,SHENGKEYUANHUAYIMINJIAOSHOUKETIZUZAIFENZISHENGWUXUEQIKAN《Nucleic Acids Research》(IF=11.5)SHANGFABIAOTIWEI“Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model”DEYANJIULUNWEN,SHENRUTANJIULEBUTONGHUAXUEXIUSHIDUIYUASOYAOWUZHILIAOJISUIXINGJIWEISUOZHENGDEYAOXIAOJIZUOYONGJILI。NANJINGSHIFANDAXUESHENGMINGKEXUEXUEYUANWEIDIYITONGXUNDANWEI,SHENGKEYUANBOSHIHOUSHENGLEIWEIDIYIZUOZHE,HUAYIMINJIAOSHOUHEMEIGUOLENGQUANGANGSHIYANSHIAdrian R. KrainerJIAOSHOUWEIGAILUNWENDETONGXUNZUOZHE。LUNWENHEZUOZUOZHEBAOKUOMEIGUOIonis PharmaceuticalsGONGSIDEFrank RigoHEC. Frank BennettBOSHI。GAIYANJIUGONGZUODEDAOLEGUOJIAZIRANJIJINZHONGDIANXIANGMU,GUOJIAZIRANJIJINMIANSHANGXIANGMU,MEIGUONIHJIJINHEMEIGUOCure SMAZHIJIAJIJINDEZHICHI。

yabo登录JISUIXINGJIWEISUOZHENG(SMA)SHIYIZHONGYURNAJIANJIEQUEXIANXIANGGUANDEYINXINGKEYICHUANJIBING。SMAHUANZHEDUOYINGYOUERSHIQIFABING,BANSUIZHEBINGCHENGJINZHAN,HUANERSHIQUZIZHUXINGDONGNENGLISHENZHISIWANG,JIBINGHUANJIATINGDAILAICHENZHONGJINGJIHEJINGSHENFUDAN。YOUYUGAIJIBINGZAIZHENGCHANGRENQUNZHONGYOUYUE1/50DEZHIBINGJIYINXIEDAIBILI,ERZAIGUOJISHANGWEIJIANGGAIJIBINGLIERUCHANQIANSHAICHAMULU,YINCIHUANBINGXINSHENGERNANYITONGGUOYOUSHENGSHOUDUANBIMIAN。

HUAJIAOSHOUZAIMEIGUOLENGQUANGANGSHIYANSHIGONGZUOQIJIAN,ZUOWEIGONGTONGFAMINGRENYANFALEKANGSMADEFANYIGUAHEGANSUAN(ASO)YAOWU---SPINRAZA。ZI2016NIAN,GAIYAOXIANHOUZAIMEIGUO、OUZHOUDUOGUOSHANGSHI,WOGUOYU2019NIAN2YUEPIZHUNGAIYAOLINCHUANGSHIYONG。DANSHIQIMEIGELIAOCHENGGAODA210WANMEIYUAN(YUE1500WANRENMINBI)DEZHILIAOFEIYONG,NANYIWEIWOGUODABUFENHUANZHEJIATINGHEYIBAOXITONGCHENGSHOU。YINCIYANFAWOGUOZIZHUZHISHICHANQUANDEXIAYIDAIZHILIAOSMADEASOXINYAO,SHIWOGUOZHONGDAGONGGONGWEISHENGJIANKANGXUQIU,YESHIHUAYIMINJIAOSHOUKETIZUJINNIANLAIZHUYAOKEYANMUBIAO。

yabo登录ASOSHIYIDUANRENGONGHECHENGDEHEGANSUANHUOHEGANSUANLEISIWU,JUYOUJIYINCHENMOHUOGAIBIANRNAJIANJIEDEZUOYONG。YOUYUTIANRANHESUANZAITINEIYIBEIJIANGJIE,YINCITONGGUOHUAXUEXIUSHILAIYANZHANGQIBANSHUAIQI、ZENGQIANGNAISHOUXINGBINGTIGAOQIYUMUDERNAXULIEJIEHEDEQINHEXINGSHIYANFAASOYAOWUFEICHANGGUANJIANDEHUANJIE。BENYANJIUZHIZAIZHAODAOGENGKEXUEDEHUAXUEXIUSHIFANGSHI,YILAITIGAOZHILIAOSMADEASOYAOWULIAOXIAO。ZUOZHEYIYANZHONGXINGSMAXIAOSHUWEIMOXING,XITONGDIBIJIAOMOE (2’-O-methoxyethyl)HEPMO (Phosphorodiamidate Morpholino Oligomer)LIANGZHONGHUAXUEXIUSHIHOUASO10-29DELIAOXIAO。FAXIANZAIXIANGTONGMOERNONGDUXIA,SHIYONGMOEXIUSHIDEASO10-29(SMAZHILIAODETEXIAOFANYIGUAHEGANSUAN)JINXINGZHILIAO,XIAOSHUSHENGLIBIAOXIANDEDAOXIANZHUGAISHAN,BAOKUOTIGAOTIZENGZHONG,YANHUANYUANDUANZUZHIHUAISI。TEBIEZHIDEGUANZHUDESHIMOEXIUSHIKEGENGHAOWEICHIYUNDONGSHENJINGYUANSHULIANGHEYANZHANGXIAOSHUSHOUMING。ZUOZHEJINYIBUFENXIFAXIANXIANGJIAOYUPMOXIUSHI,MOEXIUSHIKEGENGYOUXIAOYANHUANASO10-29BANSHUAIQI,JINERCHIXUFAHUIYAOXIAO。

yabo登录JINNIANLAI,SHENGKEYUANJINJINWEIRAOGUOJIAZHONGDAXUQIU,JIJIJIANXINGyaboDENGLU“DINGTIANLIDI”DEKEYANZHIDAOFANGZHEN,BUDANZAILILUNYANJIUSHANGLVHUOSHUOGUO,LIANXUFABIAODUOPIANBAOKUOPNASZAINEIDEGAOSHUIPINGLUNWEN;TONGSHIDALITUIJINCHANXUEYANHEZUO,CHENGGONGWANCHENGDUOGECHENGGUODECHANYEZHUANHUA,QUDEJIAOHAODESHEHUIHEJINGJIXIAOYI。HUAYIMINJIAOSHOUDEFANYIGUAHEGANSUANYAOWUYANJIUZAIGUONEIWAIJUYULINGXIANDIWEI,SHISHENGKEYUANJIANXINGyaboDENGLU“DINGTIANLIDI”KEYANZHANLVEJIECHUDEYOUYISHUOGUO。

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    2020NIAN03YUE02RI 15:46

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