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Nat Commun：减少肌脂蛋白的表达或可用于杜氏肌营养不良的治疗

2017-11-05 MedSci MedSci原创

肌脂蛋白杜氏肌营养不良
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肌脂蛋白（SLN）是肌肉/内质网（SR）Ca 2+ ATP酶（SERCA）的抑制剂，在杜氏肌营养不良（DMD）患者和动物模型中肌肉里表达异常升高。本研究中，研究人员发现减少SLN的水平可改善重症肌营养不良蛋白/肌萎缩蛋白双重突变体（mdx：utr - / -）DMD模型小鼠的营养不良。SLN基因的一个等位基因的种系失活可使SLN的表达正常化，恢复SERCA的功能，减轻骨骼肌和心脏病理，改善肌肉再生

肌脂蛋白（SLN）是肌肉/内质网（SR）Ca 2+ ATP酶（SERCA）的抑制剂，在杜氏肌营养不良（DMD）患者和动物模型中肌肉里表达异常升高。

本研究中，研究人员发现减少SLN的水平可改善重症肌营养不良蛋白/肌萎缩蛋白双重突变体（mdx：utr - / -）DMD模型小鼠的营养不良。SLN基因的一个等位基因的种系失活可使SLN的表达正常化，恢复SERCA的功能，减轻骨骼肌和心脏病理，改善肌肉再生，延长寿命。为了将此研究发现转化为治疗策略，研究人员通过腺相关病毒（AAV）9介导的RNA干扰敲低1月龄mdx：utr - / - 小鼠中SLN的表达。结果显示AAV治疗可明显降低SLN的表达，减轻肌肉病理并改善膈肌、骨骼肌和心脏功能。

综上所述，我们的研究结果表明减少SLN是治疗DMD的一种有希望的方法。

原始出处：

Antanina Voit, Vishwendra Patel, et al., Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice. Nat Commun. 2017; 8: 1068. doi:  10.1038/s41467-017-01146-7

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    2018-06-01 一叶知秋

    #Nat#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1882604, encodeId=bea618826049e, content=<a href='/topic/show?id=3b2112532d8' target=_blank style='color:#2F92EE;'>#Nat#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=12532, encryptionId=3b2112532d8, topicName=Nat)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=30bd35, createdName=一叶知秋, createdTime=Fri Jun 01 15:29:00 CST 2018, time=2018-06-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2087189, encodeId=4e7c208e1890b, content=<a href='/topic/show?id=490750196b' target=_blank style='color:#2F92EE;'>#COMMUN#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5019, encryptionId=490750196b, topicName=COMMUN)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=7931272, createdName=liuli5079, createdTime=Tue Nov 07 13:29:00 CST 2017, time=2017-11-07, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1737047, encodeId=3be71e370470b, content=<a href='/topic/show?id=6ee1810e0ad' target=_blank style='color:#2F92EE;'>#肌营养不良#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=33, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81070, encryptionId=6ee1810e0ad, topicName=肌营养不良)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=cd6034244098, createdName=751943081_32627960, createdTime=Sat Sep 15 04:29:00 CST 2018, time=2018-09-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1424684, encodeId=dc17142468488, content=<a href='/topic/show?id=5a3b8441438' target=_blank style='color:#2F92EE;'>#脂蛋白#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=84414, encryptionId=5a3b8441438, topicName=脂蛋白)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=23ea3924919, createdName=juliusluan79, createdTime=Tue Nov 07 12:29:00 CST 2017, time=2017-11-07, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=258967, encodeId=774525896e74, content=非常好的文章.学习了, beContent=null, objectType=article, channel=null, level=null, likeNumber=54, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/NUyjXTCJjo7LIiaRZTzJ3SEfiauCzTMPW7YvPPYLNJBXG9oh6Al1icq2VQQkIHWxqXehcicTS62YKJVBhxeth3wggw/0, createdBy=7ce01621306, createdName=天涯183, createdTime=Sun Nov 05 22:03:43 CST 2017, time=2017-11-05, status=1, ipAttribution=)]
    2017-11-07 liuli5079

    #COMMUN#

    0

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    2018-09-15 751943081_32627960

    #肌营养不良#

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    2017-11-07 juliusluan79

    #脂蛋白#

    0

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    2017-11-05 天涯183

    非常好的文章.学习了

    0