Nat Commun:基因编辑技术用于临床又有新进展

2018-05-13 佚名 生物360

想像一下在未来的某一天,我们可以将一个被引导的生物机器放入患者的体内,它可以寻找每个细胞中有缺陷的基因序列,然后进行精准编辑从而纠正偏差。

想像一下在未来的某一天,我们可以将一个被引导的生物机器放入患者的体内,它可以寻找每个细胞中有缺陷的基因序列,然后进行精准编辑从而纠正偏差。

这项技术被称为基因编辑,阿尔伯塔大学的研究人员刚刚发表了一项研究成果,有望使该技术更接近于临床应用。



“我们已经发现了一种方法,通过用合成的称为桥联核酸或 BNA 来取代天然引导分子,大大提高了基因编辑技术的准确性,”加拿大分子疗法研究主席 Basil Hubbard 说。这位研究负责人也是阿尔伯塔大学药理学系的助理教授。

他和他的团队已经就他们的发现提交了专利,并希望与制药行业合作将其纳入治疗领域。

自发现 CRISPR / Cas9 系统以来,人们对基因编辑技术的关注度一直在迅速增加。该系统天然存在于细菌中,用于抵御其天敌,称为噬菌体。

“它允许细菌存储关于先前感染的信息,然后通过它来找出并切割入侵体的 DNA 来摧毁入侵体。” Hubbard 解释说。

“研究人员已经意识到,这个系统还可以按特定的程式在人类细胞中切割特定的 DNA 序列,从而允许我们编辑我们的基因。然而,其中一个主要问题是此系统不是能够完全的精准操作,有时它会削减了一个类似的但并不是靶向的基因。”

他指出,使用天然的 RNA 引导分子,Cas9 系统已经比较准确,只有大约百分之一的时侯出错。

“然而,鉴于人体内有数万亿个细胞,哪怕只有一个百分比的出错率也是非常可怕的,因为基因编辑是永久性的,一个错误的切割可能会导致患者最终会患上像癌症这样的严重疾病。”

经由 Hubbard 和他的团队(其中包括研究的第一作者,博士生 Christopher Cromwell)的改进,新的 BNA 引导分子在寻求正确的 DNA 切割方面表现则更为稳定和严谨。

“我们的研究表明,在某些情况下,使用桥接核酸引导 Cas9 可以将其特异性提高 10,000 倍以上 – 这是一个显着的改进。”Hubbard 说。

虽然基因编辑技术仍然有几个需要克服的障碍,包括如何将其有效地输送到人体内等,但它还是很可能有一天会用于治疗各种遗传疾病,从肌营养不良到血友病和各种癌症等。

上述研究论文 刊登 在了近期的《自然 - 通讯》杂志上。

原始出处:Cromwel CR, Sung K, Park J, et al. Incorporation of bridged nucleic acids into CRISPR RNAs improves Cas9 endonuclease specificity. Nat Commun. 2018 Apr 13;9(1):1448. 

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    2018-06-07 liye789132251
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    2019-02-02 liuli5079
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encodeId=4b6c314915bf, content=学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmhead/Q3auHgzwzM6P57Bs8gOPaY1cQP9lIia0lmZOEXraGaoicqNToIslXDKw/0, createdBy=7a041951742, createdName=衣带渐宽, createdTime=Mon May 14 06:35:34 CST 2018, time=2018-05-14, status=1, ipAttribution=)]
    2018-05-14 明月清辉

    谢谢分享.学习了

    0

  5. 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encodeId=4b6c314915bf, content=学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmhead/Q3auHgzwzM6P57Bs8gOPaY1cQP9lIia0lmZOEXraGaoicqNToIslXDKw/0, createdBy=7a041951742, createdName=衣带渐宽, createdTime=Mon May 14 06:35:34 CST 2018, time=2018-05-14, status=1, ipAttribution=)]
    2018-05-14 龙胆草

    学习谢谢分享

    0

  6. 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encodeId=4b6c314915bf, content=学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmhead/Q3auHgzwzM6P57Bs8gOPaY1cQP9lIia0lmZOEXraGaoicqNToIslXDKw/0, createdBy=7a041951742, createdName=衣带渐宽, createdTime=Mon May 14 06:35:34 CST 2018, time=2018-05-14, status=1, ipAttribution=)]
    2018-05-14 1ddf0692m34(暂无匿称)

    学习了.长知识

    0

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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=Mon May 14 07:25:50 CST 2018, time=2018-05-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=314937, encodeId=80b331493e2b, content=好好好好好好好好, beContent=null, objectType=article, channel=null, level=null, likeNumber=57, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://cdnapi.center.medsci.cn/medsci/head/2017/08/29/bbc5cc0eed9b8d17623d2fd49dbdf19a.jpg, createdBy=79522135794, createdName=yjs木玉, createdTime=Mon May 14 07:02:29 CST 2018, time=2018-05-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=314923, encodeId=189731492378, content=了解了谢谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/Q0j4TwGTfTLbTp9nZtjwVrSZNxAXbAicnKQWnic2eBdJUQYhxO36XdkstSicr7FVF8p5ReH4omtmsbnLdvZLiaJJ1A/132, createdBy=8bff2327056, createdName=kafei, createdTime=Mon May 14 06:50:38 CST 2018, time=2018-05-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=314919, encodeId=748831491982, content=学习一下谢谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/jW482SpianMayicTRbRZ5RzaiabIFicf63hKvVMjL6aJlx4yX4UcXmzX9DyibCRcicq0QtCRWMaQKh8MYjE1D3EKUBopgiaGWYuqbeR/0, createdBy=0c2b1999806, createdName=三生有幸9135, createdTime=Mon May 14 06:49:47 CST 2018, time=2018-05-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=314915, encodeId=4b6c314915bf, content=学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmhead/Q3auHgzwzM6P57Bs8gOPaY1cQP9lIia0lmZOEXraGaoicqNToIslXDKw/0, createdBy=7a041951742, createdName=衣带渐宽, createdTime=Mon May 14 06:35:34 CST 2018, time=2018-05-14, status=1, ipAttribution=)]
    2018-05-14 yjs木玉

    好好好好好好好好

    0

  8. 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encodeId=4b6c314915bf, content=学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmhead/Q3auHgzwzM6P57Bs8gOPaY1cQP9lIia0lmZOEXraGaoicqNToIslXDKw/0, createdBy=7a041951742, createdName=衣带渐宽, createdTime=Mon May 14 06:35:34 CST 2018, time=2018-05-14, status=1, ipAttribution=)]
    2018-05-14 kafei

    了解了谢谢

    0

  9. 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encodeId=4b6c314915bf, content=学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmhead/Q3auHgzwzM6P57Bs8gOPaY1cQP9lIia0lmZOEXraGaoicqNToIslXDKw/0, createdBy=7a041951742, createdName=衣带渐宽, createdTime=Mon May 14 06:35:34 CST 2018, time=2018-05-14, status=1, ipAttribution=)]
    2018-05-14 三生有幸9135

    学习一下谢谢分享

    0

  10. 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