Stem Cell Res:从患有非综合征性CLN3相关视网膜变性患者中成功建立诱导性多能干细胞系

2018-05-19 cuiguizhong MedSci原创

澳大利亚大学眼科与视觉科学中心的Zhang X近日在Stem Cell Res发表了一篇文章,他们建立了人源晚发性非综合征视网膜色素变性的iPSC细胞系LEIi004-A。

澳大利亚大学眼科与视觉科学中心的Zhang X近日在Stem Cell Res发表了一篇文章,他们建立了人源晚发性非综合征视网膜色素变性的iPSC细胞系LEIi004-A。

该患者是由CLN3基因中杂合突变引起的晚发性非综合征视网膜色素变性,研究者从患者获取细胞,进行诱导培养后获得人类iPSC细胞系,命名为LEIi004-A。他们使用含有OCT4、SOX2、KLF4、L-MYC、LIN28和针对p53的shRNA,以及mir302 / 367等microRNA的质粒转染细胞,对患者来源的原代真皮成纤维细胞进行重编程。他们还构建了对照的细胞系。具体操作是使用CRISPR / Cas9基因编辑的方法,在患者来源的iPSC细胞中纠正一个CLN3的突变,获得了iPSC细胞系,命名为LEIi004-A-1。

因此,这项工作建立了人源晚发性非综合征视网膜色素变性的iPSC细胞系,这将促进科研人员对晚发性非综合征视网膜色素变性疾病发生发展的研究,提供可行的临床治疗措施。

原文出处:

Zhang, X., et al., Generation of an induced pluripotent stem cell line from a patient with non-syndromic CLN3-associated retinal degeneration and a coisogenic control line. Stem Cell Res, 2018. 29: p. 245-249.

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createdName=zhaozhouchifen, createdTime=Sun Mar 17 10:41:00 CST 2019, time=2019-03-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2005573, encodeId=c16e20055e384, content=<a href='/topic/show?id=6e89485e82' target=_blank style='color:#2F92EE;'>#CLN3#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=4857, encryptionId=6e89485e82, topicName=CLN3)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/WQ1WyMBGic8d2fKdCe6Cm3vaRh4X8YnlumYndC46iagNCBl9Df8RtoCaQchnFtZldHUctGJjsgsyutUlu8vPq6yQ/132, createdBy=97862500016, createdName=ms3117646317107274, createdTime=Wed Nov 21 17:41:00 CST 2018, time=2018-11-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1960921, encodeId=bedc19609215a, content=<a href='/topic/show?id=dbe6445416' target=_blank style='color:#2F92EE;'>#CEL#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, 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=Sat Sep 08 20:41:00 CST 2018, time=2018-09-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1813253, encodeId=9fa918132538a, content=<a href='/topic/show?id=92b5918e845' target=_blank style='color:#2F92EE;'>#诱导性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=91878, encryptionId=92b5918e845, topicName=诱导性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4193478, createdName=wgx306, createdTime=Fri Apr 19 21:41:00 CST 2019, time=2019-04-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1722235, encodeId=e97a1e2223514, content=<a href='/topic/show?id=5e0d48e897f' target=_blank style='color:#2F92EE;'>#干细胞系#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=48789, encryptionId=5e0d48e897f, topicName=干细胞系)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=482a33230984, createdName=andy2098, createdTime=Thu Jul 12 18:41:00 CST 2018, time=2018-07-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1747099, encodeId=ede01e4709953, content=<a href='/topic/show?id=76a090e5544' target=_blank style='color:#2F92EE;'>#视网膜变性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=90755, encryptionId=76a090e5544, topicName=视网膜变性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=a6db35920976, createdName=circumcision, createdTime=Tue Jun 19 08:41:00 CST 2018, time=2018-06-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=317546, encodeId=bca231e5460f, content=学习了.长知识, beContent=null, objectType=article, channel=null, level=null, likeNumber=66, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220115/46bcf39c32de4aa6b45c5f9d66c8ee77/6cb4a20c55bb4b7691122f47747bfca2.jpg, createdBy=9dad1662329, createdName=1ddf0692m34(暂无匿称), createdTime=Tue May 22 13:00:11 CST 2018, time=2018-05-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1270060, encodeId=53a612e00608a, content=<a href='/topic/show?id=979690e33bd' target=_blank style='color:#2F92EE;'>#视网膜#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=90733, encryptionId=979690e33bd, topicName=视网膜)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=742793, createdName=zutt, createdTime=Mon May 21 12:41:00 CST 2018, time=2018-05-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1431216, encodeId=8e47143121607, content=<a href='/topic/show?id=f9b9e9124a8' target=_blank style='color:#2F92EE;'>#综合征#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=20, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79124, encryptionId=f9b9e9124a8, topicName=综合征)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Mon May 21 12:41:00 CST 2018, time=2018-05-21, status=1, ipAttribution=)]
    2018-09-08 维他命
  5. [GetPortalCommentsPageByObjectIdResponse(id=1794601, encodeId=94e91e94601e3, content=<a href='/topic/show?id=82b416e79f5' target=_blank style='color:#2F92EE;'>#stem cell#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, 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=Mon Jun 25 03:41:00 CST 2018, time=2018-06-25, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1870652, encodeId=e69018e0652dd, content=<a href='/topic/show?id=fd764459a8' target=_blank style='color:#2F92EE;'>#Cell#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=4459, encryptionId=fd764459a8, topicName=Cell)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=524d95, createdName=zhaozhouchifen, createdTime=Sun Mar 17 10:41:00 CST 2019, time=2019-03-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2005573, encodeId=c16e20055e384, content=<a href='/topic/show?id=6e89485e82' target=_blank style='color:#2F92EE;'>#CLN3#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=4857, encryptionId=6e89485e82, topicName=CLN3)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/WQ1WyMBGic8d2fKdCe6Cm3vaRh4X8YnlumYndC46iagNCBl9Df8RtoCaQchnFtZldHUctGJjsgsyutUlu8vPq6yQ/132, createdBy=97862500016, createdName=ms3117646317107274, createdTime=Wed Nov 21 17:41:00 CST 2018, time=2018-11-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1960921, encodeId=bedc19609215a, content=<a href='/topic/show?id=dbe6445416' target=_blank style='color:#2F92EE;'>#CEL#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, 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=Sat Sep 08 20:41:00 CST 2018, time=2018-09-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1813253, encodeId=9fa918132538a, content=<a href='/topic/show?id=92b5918e845' target=_blank style='color:#2F92EE;'>#诱导性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=91878, encryptionId=92b5918e845, topicName=诱导性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4193478, createdName=wgx306, createdTime=Fri Apr 19 21:41:00 CST 2019, time=2019-04-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1722235, encodeId=e97a1e2223514, content=<a href='/topic/show?id=5e0d48e897f' target=_blank style='color:#2F92EE;'>#干细胞系#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=48789, encryptionId=5e0d48e897f, topicName=干细胞系)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=482a33230984, createdName=andy2098, createdTime=Thu Jul 12 18:41:00 CST 2018, time=2018-07-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1747099, encodeId=ede01e4709953, content=<a href='/topic/show?id=76a090e5544' target=_blank style='color:#2F92EE;'>#视网膜变性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=90755, encryptionId=76a090e5544, topicName=视网膜变性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=a6db35920976, createdName=circumcision, createdTime=Tue Jun 19 08:41:00 CST 2018, time=2018-06-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=317546, encodeId=bca231e5460f, content=学习了.长知识, beContent=null, objectType=article, channel=null, level=null, likeNumber=66, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220115/46bcf39c32de4aa6b45c5f9d66c8ee77/6cb4a20c55bb4b7691122f47747bfca2.jpg, createdBy=9dad1662329, createdName=1ddf0692m34(暂无匿称), createdTime=Tue May 22 13:00:11 CST 2018, time=2018-05-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1270060, encodeId=53a612e00608a, content=<a href='/topic/show?id=979690e33bd' target=_blank style='color:#2F92EE;'>#视网膜#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=90733, encryptionId=979690e33bd, topicName=视网膜)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=742793, createdName=zutt, createdTime=Mon May 21 12:41:00 CST 2018, time=2018-05-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1431216, encodeId=8e47143121607, content=<a href='/topic/show?id=f9b9e9124a8' target=_blank style='color:#2F92EE;'>#综合征#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=20, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79124, encryptionId=f9b9e9124a8, topicName=综合征)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Mon May 21 12:41:00 CST 2018, time=2018-05-21, status=1, ipAttribution=)]
    2019-04-19 wgx306
  6. [GetPortalCommentsPageByObjectIdResponse(id=1794601, encodeId=94e91e94601e3, content=<a href='/topic/show?id=82b416e79f5' target=_blank style='color:#2F92EE;'>#stem cell#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, 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=Mon Jun 25 03:41:00 CST 2018, time=2018-06-25, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1870652, encodeId=e69018e0652dd, content=<a href='/topic/show?id=fd764459a8' target=_blank style='color:#2F92EE;'>#Cell#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=4459, encryptionId=fd764459a8, topicName=Cell)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=524d95, 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objectType=article, channel=null, level=null, likeNumber=23, 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=Sat Sep 08 20:41:00 CST 2018, time=2018-09-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1813253, encodeId=9fa918132538a, content=<a href='/topic/show?id=92b5918e845' target=_blank style='color:#2F92EE;'>#诱导性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=91878, encryptionId=92b5918e845, topicName=诱导性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4193478, createdName=wgx306, createdTime=Fri Apr 19 21:41:00 CST 2019, time=2019-04-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1722235, encodeId=e97a1e2223514, content=<a href='/topic/show?id=5e0d48e897f' target=_blank style='color:#2F92EE;'>#干细胞系#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=48789, encryptionId=5e0d48e897f, topicName=干细胞系)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=482a33230984, createdName=andy2098, createdTime=Thu Jul 12 18:41:00 CST 2018, time=2018-07-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1747099, encodeId=ede01e4709953, content=<a href='/topic/show?id=76a090e5544' target=_blank style='color:#2F92EE;'>#视网膜变性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=90755, encryptionId=76a090e5544, topicName=视网膜变性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=a6db35920976, createdName=circumcision, createdTime=Tue Jun 19 08:41:00 CST 2018, time=2018-06-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=317546, encodeId=bca231e5460f, content=学习了.长知识, beContent=null, objectType=article, channel=null, level=null, likeNumber=66, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220115/46bcf39c32de4aa6b45c5f9d66c8ee77/6cb4a20c55bb4b7691122f47747bfca2.jpg, createdBy=9dad1662329, createdName=1ddf0692m34(暂无匿称), createdTime=Tue May 22 13:00:11 CST 2018, time=2018-05-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1270060, encodeId=53a612e00608a, content=<a href='/topic/show?id=979690e33bd' target=_blank style='color:#2F92EE;'>#视网膜#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=90733, encryptionId=979690e33bd, topicName=视网膜)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=742793, createdName=zutt, createdTime=Mon May 21 12:41:00 CST 2018, time=2018-05-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1431216, encodeId=8e47143121607, content=<a href='/topic/show?id=f9b9e9124a8' target=_blank style='color:#2F92EE;'>#综合征#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=20, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79124, encryptionId=f9b9e9124a8, topicName=综合征)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Mon May 21 12:41:00 CST 2018, time=2018-05-21, status=1, ipAttribution=)]
  7. 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  8. 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createdBy=742793, createdName=zutt, createdTime=Mon May 21 12:41:00 CST 2018, time=2018-05-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1431216, encodeId=8e47143121607, content=<a href='/topic/show?id=f9b9e9124a8' target=_blank style='color:#2F92EE;'>#综合征#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=20, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79124, encryptionId=f9b9e9124a8, topicName=综合征)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Mon May 21 12:41:00 CST 2018, time=2018-05-21, status=1, ipAttribution=)]
    2018-05-22 1ddf0692m34(暂无匿称)

    学习了.长知识

    0

  9. 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time=2018-06-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=317546, encodeId=bca231e5460f, content=学习了.长知识, beContent=null, objectType=article, channel=null, level=null, likeNumber=66, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220115/46bcf39c32de4aa6b45c5f9d66c8ee77/6cb4a20c55bb4b7691122f47747bfca2.jpg, createdBy=9dad1662329, createdName=1ddf0692m34(暂无匿称), createdTime=Tue May 22 13:00:11 CST 2018, time=2018-05-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1270060, encodeId=53a612e00608a, content=<a href='/topic/show?id=979690e33bd' target=_blank style='color:#2F92EE;'>#视网膜#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=90733, encryptionId=979690e33bd, topicName=视网膜)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=742793, createdName=zutt, createdTime=Mon May 21 12:41:00 CST 2018, time=2018-05-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1431216, encodeId=8e47143121607, content=<a href='/topic/show?id=f9b9e9124a8' target=_blank style='color:#2F92EE;'>#综合征#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=20, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79124, encryptionId=f9b9e9124a8, topicName=综合征)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Mon May 21 12:41:00 CST 2018, time=2018-05-21, status=1, ipAttribution=)]
    2018-05-21 zutt
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