STM：基因改造过的诱导性多功能干细胞（iPSCs）可治疗隐性遗传营养不良型大疱性表皮松解症（RDEB）

2015-01-13 Ashley Wu 译 MedSci原创

隐性遗传营养不良型大疱性表皮松解症（RDEB）是一种遗传疾病。它是由于VII型胶原的编码基因变异导致的。RDEB患者的皮肤很脆弱并且很容易在接收到压力时起水疱。特别是对于RDEB这种在现阶段没有有效治疗的疾病，使用诱导性多功能干细胞（iPSCs）有从根本上改变疾病细胞治疗的潜力。为了证明诱导性多功能干细胞(iPSCs)可以治疗RDEB，研究者们从有VII型胶原基因(Co17a1)变异的小鼠中提取

隐性遗传营养不良型大疱性表皮松解症（RDEB）是一种遗传疾病。它是由于VII型胶原的编码基因变异导致的。RDEB患者的皮肤很脆弱并且很容易在接收到压力时起水疱。特别是对于RDEB这种在现阶段没有有效治疗的疾病，使用诱导性多功能干细胞（iPSCs）有从根本上改变疾病细胞治疗的潜力。

为了证明诱导性多功能干细胞(iPSCs)可以治疗RDEB，研究者们从有VII型胶原基因(Co17a1)变异的小鼠中提取了多功能干细胞(iPSCs)。这些变异小鼠都显现了人类RDEB的症状，包括易损的皮肤及皮肤上容易起水疱。从基因方面来修复干细胞（iPSCs）可以使这些干细胞重新分化成为功能性的成纤维细胞，而这些成纤维细胞可以重新分泌VII型胶原。而且，这些从修复过的多功能干细胞(iPSCs)中分化出来的成纤维细胞没有在小鼠体内形成肿瘤，并在皮内注射后的16个星期内都能检测出细胞在体内的痕迹。除此之外，对有Co17al突变的小鼠使用诱导性多功能干细胞（iPSCs）为基础的细胞治疗，可以有效并长期的恢复VII型胶原在小鼠的真皮及表皮交界处的沉淀。

因为对小鼠进行皮内注射从修复过的多功能干细胞(iPSCs)中分化出来的成纤维细胞可以使患有RDEB的小鼠皮肤恢复防止长水疱的能力。研究者们认为这可以证明以多功能干细胞（iPSCs）为基础的的细胞治疗可以安全并有效的治疗RDEB（隐性遗传营养不良型大疱性表皮松解症）。

原始出处：

Daniel Wenzel, Jonathan Bayerl, Alexander Nyström, Leena Bruckner-Tuderman, Arabella Meixner,and Josef M. Penninger;Genetically corrected iPSCs as cell therapy for recessive dystrophic epidermolysis bullosa.Sci Transl Med 26 November 2014 6:264ra165. [DOI:10.1126/scitranslmed.3010083]

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2015-01-26 gdsun

#STM#

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2015-08-31 wgx306

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2015-01-15 chengjn

#iPS#

20 0
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2015-01-15 gjsgj

#PSC#

23 0
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2015-01-15 nian.wang1983

#大疱性表皮松解症#

40 0
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2015-01-15 caoliehu

#iPSCs#

46 0
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createdBy=4dfa37, createdName=智者为医08, createdTime=Thu Jan 15 07:28:00 CST 2015, time=2015-01-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=14267, encodeId=73f71426e63, content=谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=115, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0620, createdName=MedSci客户端网友, createdTime=Tue Jan 13 21:03:00 CST 2015, time=2015-01-13, status=1, ipAttribution=)]
2015-01-15 neurosurgeon

#iPSC#

47 0
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2015-01-15 1696533704_45270459

#多功能干细胞#

40 0
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createdBy=4dfa37, createdName=智者为医08, createdTime=Thu Jan 15 07:28:00 CST 2015, time=2015-01-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=14267, encodeId=73f71426e63, content=谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=115, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0620, createdName=MedSci客户端网友, createdTime=Tue Jan 13 21:03:00 CST 2015, time=2015-01-13, status=1, ipAttribution=)]
2015-01-15 智者为医08

#基因改造#

27 0
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createdBy=4dfa37, createdName=智者为医08, createdTime=Thu Jan 15 07:28:00 CST 2015, time=2015-01-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=14267, encodeId=73f71426e63, content=谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=115, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0620, createdName=MedSci客户端网友, createdTime=Tue Jan 13 21:03:00 CST 2015, time=2015-01-13, status=1, ipAttribution=)]
2015-01-13 MedSci客户端网友

谢分享

115 0

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皮肤性

STM：基因改造过的诱导性多功能干细胞（iPSCs）可治疗隐性遗传营养不良型大疱性表皮松解症（RDEB）

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