Cell Stem Cell:中科院用CRISPR技术治愈小鼠白内障

2013-12-11 佚名 中国科学报

中科院上海生物化学与细胞生物学研究所李劲松研究组在一项最新研究中,利用CRISPR-Cas9技术,治愈了小鼠的白内障遗传疾病。专家认为,这项实验为人类基因治疗提供了一条新思路。12月5日,相关成果在线发表于《细胞—干细胞》杂志。中科院李劲松博士同时,该成果被国际杂志《科学家》选择进行同期新闻发布。CRISPR-Cas9技术是利用一段与靶序列相同的单导向RNA(sgRNA)来引导Cas9核酸酶对特异

中科院上海生物化学与细胞生物学研究所李劲松研究组在一项最新研究中,利用CRISPR-Cas9技术,治愈了小鼠的白内障遗传疾病。专家认为,这项实验为人类基因治疗提供了一条新思路。12月5日,相关成果在线发表于《细胞—干细胞》杂志。


中科院李劲松博士

同时,该成果被国际杂志《科学家》选择进行同期新闻发布。CRISPR-Cas9技术是利用一段与靶序列相同的单导向RNA(sgRNA)来引导Cas9核酸酶对特异靶向DNA进行识别和切割,造成DNA的双链或单链断裂,然后,细胞会利用自身具备的两种修复机制对断裂的DNA进行修复,即非同源性末端接合(NHEJ)或同源介导修复(HDR)。【原文下载】

为验证这项技术能否用于高效治疗遗传病,研究者选择小鼠白内障遗传疾病模型进行研究。这批模型小鼠携带显性突变的Crygc基因,眼睛会产生变性的晶状体蛋白而发生混浊。研究人员设计出针对突变基因的单导向RNA,将它与核酸酶直接注入受精卵,发现1/3新生小鼠的白内障症状得到治愈。白内障小鼠治愈后,也能通过生殖细胞将修复的基因传递给下一代,证明白内障遗传疾病可被根治。

研究人员根据sgRNA同源程度,预测存在10个潜在脱靶位点,并对这些位点进行测序发现,12只治愈的小鼠中只有两只在一个位点上存在脱靶现象。

李劲松表示:“今后的研究还需要进一步提高治愈的效率,避免出现脱靶。”

原文出处:

Wu Y, Liang D, Wang Y, Bai M, Tang W, Bao S, Yan Z, Li D, Li J.Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9.Cell Stem Cell. 2013 Dec 5;13(6):659-62. doi: 10.1016/j.stem.2013.10.016.【原文下载】

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    2014-02-19 维他命
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    2014-05-19 114.84.226.239

    没干过任何事情的人也敢出现在作者中

    0

  5. [GetPortalCommentsPageByObjectIdResponse(id=1794347, encodeId=671f1e9434734, content=<a href='/topic/show?id=82b416e79f5' target=_blank style='color:#2F92EE;'>#stem cell#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16779, encryptionId=82b416e79f5, topicName=stem cell)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4c37265, createdName=hb2008ye, createdTime=Tue Jun 24 11:23:00 CST 2014, time=2014-06-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1958967, encodeId=4426195896e8a, content=<a href='/topic/show?id=dbe6445416' target=_blank style='color:#2F92EE;'>#CEL#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=4454, encryptionId=dbe6445416, topicName=CEL)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c3ff68, createdName=维他命, createdTime=Wed Feb 19 01:23:00 CST 2014, time=2014-02-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1868727, encodeId=f6eb1868e2733, content=<a href='/topic/show?id=fd764459a8' target=_blank style='color:#2F92EE;'>#Cell#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=4459, encryptionId=fd764459a8, topicName=Cell)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=524d95, createdName=zhaozhouchifen, createdTime=Tue Aug 26 08:23:00 CST 2014, time=2014-08-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=9530, encodeId=e40b953085, content=没干过任何事情的人也敢出现在作者中, beContent=null, objectType=article, channel=null, level=null, likeNumber=167, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0620, createdName=114.84.226.239, createdTime=Mon May 19 22:13:00 CST 2014, time=2014-05-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1862487, encodeId=c6c8186248ecd, content=<a href='/topic/show?id=a121223385b' target=_blank style='color:#2F92EE;'>#中科院#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=22338, encryptionId=a121223385b, topicName=中科院)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=7a9987, createdName=gous, createdTime=Thu May 15 10:23:00 CST 2014, time=2014-05-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1275796, encodeId=76a712e5796e5, content=<a href='/topic/show?id=755d5211a0' target=_blank style='color:#2F92EE;'>#CRISPR#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5211, encryptionId=755d5211a0, topicName=CRISPR)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=583c133, createdName=yuandd, createdTime=Fri Dec 13 04:23:00 CST 2013, time=2013-12-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1481810, encodeId=8b2d148181055, content=<a href='/topic/show?id=8cc416e78f3' target=_blank style='color:#2F92EE;'>#STEM#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=18, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16778, encryptionId=8cc416e78f3, topicName=STEM)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d1067833268, createdName=12498bb3m92(暂无昵称), createdTime=Fri Dec 13 04:23:00 CST 2013, time=2013-12-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1618685, encodeId=85401618685f7, content=<a href='/topic/show?id=e798524076' target=_blank style='color:#2F92EE;'>#Crispr技术#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5240, encryptionId=e798524076, topicName=Crispr技术)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=a49419938232, createdName=ms1779634646866130, createdTime=Fri Dec 13 04:23:00 CST 2013, time=2013-12-13, status=1, ipAttribution=)]
    2014-05-15 gous
  6. [GetPortalCommentsPageByObjectIdResponse(id=1794347, encodeId=671f1e9434734, content=<a href='/topic/show?id=82b416e79f5' target=_blank style='color:#2F92EE;'>#stem cell#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16779, encryptionId=82b416e79f5, topicName=stem cell)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4c37265, createdName=hb2008ye, createdTime=Tue Jun 24 11:23:00 CST 2014, time=2014-06-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1958967, encodeId=4426195896e8a, content=<a href='/topic/show?id=dbe6445416' target=_blank style='color:#2F92EE;'>#CEL#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=4454, encryptionId=dbe6445416, topicName=CEL)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c3ff68, createdName=维他命, createdTime=Wed Feb 19 01:23:00 CST 2014, time=2014-02-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1868727, encodeId=f6eb1868e2733, content=<a href='/topic/show?id=fd764459a8' target=_blank style='color:#2F92EE;'>#Cell#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=4459, encryptionId=fd764459a8, topicName=Cell)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=524d95, createdName=zhaozhouchifen, createdTime=Tue Aug 26 08:23:00 CST 2014, time=2014-08-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=9530, encodeId=e40b953085, content=没干过任何事情的人也敢出现在作者中, beContent=null, objectType=article, channel=null, level=null, likeNumber=167, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0620, createdName=114.84.226.239, createdTime=Mon May 19 22:13:00 CST 2014, time=2014-05-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1862487, encodeId=c6c8186248ecd, content=<a href='/topic/show?id=a121223385b' target=_blank style='color:#2F92EE;'>#中科院#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=22338, encryptionId=a121223385b, topicName=中科院)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=7a9987, createdName=gous, createdTime=Thu May 15 10:23:00 CST 2014, time=2014-05-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1275796, encodeId=76a712e5796e5, content=<a href='/topic/show?id=755d5211a0' target=_blank style='color:#2F92EE;'>#CRISPR#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5211, encryptionId=755d5211a0, topicName=CRISPR)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=583c133, createdName=yuandd, createdTime=Fri Dec 13 04:23:00 CST 2013, time=2013-12-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1481810, encodeId=8b2d148181055, content=<a href='/topic/show?id=8cc416e78f3' target=_blank style='color:#2F92EE;'>#STEM#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=18, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16778, encryptionId=8cc416e78f3, topicName=STEM)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d1067833268, createdName=12498bb3m92(暂无昵称), createdTime=Fri Dec 13 04:23:00 CST 2013, time=2013-12-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1618685, encodeId=85401618685f7, content=<a href='/topic/show?id=e798524076' target=_blank style='color:#2F92EE;'>#Crispr技术#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5240, encryptionId=e798524076, topicName=Crispr技术)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=a49419938232, createdName=ms1779634646866130, createdTime=Fri Dec 13 04:23:00 CST 2013, time=2013-12-13, status=1, ipAttribution=)]
    2013-12-13 yuandd
  7. [GetPortalCommentsPageByObjectIdResponse(id=1794347, encodeId=671f1e9434734, content=<a href='/topic/show?id=82b416e79f5' target=_blank style='color:#2F92EE;'>#stem cell#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16779, encryptionId=82b416e79f5, topicName=stem cell)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4c37265, createdName=hb2008ye, createdTime=Tue Jun 24 11:23:00 CST 2014, time=2014-06-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1958967, encodeId=4426195896e8a, content=<a href='/topic/show?id=dbe6445416' target=_blank style='color:#2F92EE;'>#CEL#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=4454, encryptionId=dbe6445416, topicName=CEL)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c3ff68, createdName=维他命, createdTime=Wed Feb 19 01:23:00 CST 2014, time=2014-02-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1868727, encodeId=f6eb1868e2733, content=<a href='/topic/show?id=fd764459a8' target=_blank style='color:#2F92EE;'>#Cell#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=4459, encryptionId=fd764459a8, topicName=Cell)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=524d95, createdName=zhaozhouchifen, createdTime=Tue Aug 26 08:23:00 CST 2014, time=2014-08-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=9530, encodeId=e40b953085, content=没干过任何事情的人也敢出现在作者中, beContent=null, objectType=article, channel=null, level=null, likeNumber=167, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0620, createdName=114.84.226.239, createdTime=Mon May 19 22:13:00 CST 2014, time=2014-05-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1862487, encodeId=c6c8186248ecd, content=<a href='/topic/show?id=a121223385b' target=_blank style='color:#2F92EE;'>#中科院#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=22338, encryptionId=a121223385b, topicName=中科院)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=7a9987, createdName=gous, createdTime=Thu May 15 10:23:00 CST 2014, time=2014-05-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1275796, encodeId=76a712e5796e5, content=<a href='/topic/show?id=755d5211a0' target=_blank style='color:#2F92EE;'>#CRISPR#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5211, encryptionId=755d5211a0, topicName=CRISPR)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=583c133, createdName=yuandd, createdTime=Fri Dec 13 04:23:00 CST 2013, time=2013-12-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1481810, encodeId=8b2d148181055, content=<a href='/topic/show?id=8cc416e78f3' target=_blank style='color:#2F92EE;'>#STEM#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=18, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16778, encryptionId=8cc416e78f3, topicName=STEM)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d1067833268, createdName=12498bb3m92(暂无昵称), createdTime=Fri Dec 13 04:23:00 CST 2013, time=2013-12-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1618685, encodeId=85401618685f7, content=<a href='/topic/show?id=e798524076' target=_blank style='color:#2F92EE;'>#Crispr技术#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5240, encryptionId=e798524076, topicName=Crispr技术)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=a49419938232, createdName=ms1779634646866130, createdTime=Fri Dec 13 04:23:00 CST 2013, time=2013-12-13, status=1, ipAttribution=)]
  8. [GetPortalCommentsPageByObjectIdResponse(id=1794347, encodeId=671f1e9434734, content=<a href='/topic/show?id=82b416e79f5' target=_blank style='color:#2F92EE;'>#stem cell#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16779, encryptionId=82b416e79f5, topicName=stem cell)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4c37265, createdName=hb2008ye, createdTime=Tue Jun 24 11:23:00 CST 2014, time=2014-06-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1958967, encodeId=4426195896e8a, content=<a href='/topic/show?id=dbe6445416' target=_blank style='color:#2F92EE;'>#CEL#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=4454, encryptionId=dbe6445416, topicName=CEL)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c3ff68, createdName=维他命, createdTime=Wed Feb 19 01:23:00 CST 2014, time=2014-02-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1868727, encodeId=f6eb1868e2733, content=<a href='/topic/show?id=fd764459a8' target=_blank style='color:#2F92EE;'>#Cell#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=4459, encryptionId=fd764459a8, topicName=Cell)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=524d95, createdName=zhaozhouchifen, createdTime=Tue Aug 26 08:23:00 CST 2014, time=2014-08-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=9530, encodeId=e40b953085, content=没干过任何事情的人也敢出现在作者中, beContent=null, objectType=article, channel=null, level=null, likeNumber=167, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0620, createdName=114.84.226.239, createdTime=Mon May 19 22:13:00 CST 2014, time=2014-05-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1862487, encodeId=c6c8186248ecd, content=<a href='/topic/show?id=a121223385b' target=_blank style='color:#2F92EE;'>#中科院#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=22338, encryptionId=a121223385b, topicName=中科院)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=7a9987, createdName=gous, createdTime=Thu May 15 10:23:00 CST 2014, time=2014-05-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1275796, encodeId=76a712e5796e5, content=<a href='/topic/show?id=755d5211a0' target=_blank style='color:#2F92EE;'>#CRISPR#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5211, encryptionId=755d5211a0, topicName=CRISPR)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=583c133, createdName=yuandd, createdTime=Fri Dec 13 04:23:00 CST 2013, time=2013-12-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1481810, encodeId=8b2d148181055, content=<a href='/topic/show?id=8cc416e78f3' target=_blank style='color:#2F92EE;'>#STEM#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=18, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16778, encryptionId=8cc416e78f3, topicName=STEM)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d1067833268, createdName=12498bb3m92(暂无昵称), createdTime=Fri Dec 13 04:23:00 CST 2013, time=2013-12-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1618685, encodeId=85401618685f7, content=<a href='/topic/show?id=e798524076' target=_blank style='color:#2F92EE;'>#Crispr技术#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5240, encryptionId=e798524076, topicName=Crispr技术)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=a49419938232, createdName=ms1779634646866130, createdTime=Fri Dec 13 04:23:00 CST 2013, time=2013-12-13, status=1, ipAttribution=)]