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Genes & Dev：脊髓性肌肉萎缩症研究取得重大突破

2015-02-04 佚名 中国科学网

脊髓性肌肉萎缩症运动神经
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日前，苏州大学神经科学研究所教授华益民与美国冷泉港实验室合作，通过对大量小鼠疾病模型的研究，发现提高外周组织运动神经元存活蛋白的表达能有效治疗脊髓性肌肉萎缩症。相关研究成果已发表于国际著名生物医学期刊基因与发育，华益民为第一作者兼共同通讯作者。 脊髓性肌肉萎缩症为隐性常染色体遗传病，是婴幼儿死亡的第一杀手。在最新研究中，华益民和美国合作者设计了一个称作“诱饵”的正义寡核苷酸，



日前，苏州大学神经科学研究所教授华益民与美国冷泉港实验室合作，通过对大量小鼠疾病模型的研究，发现提高外周组织运动神经元存活蛋白的表达能有效治疗脊髓性肌肉萎缩症。相关研究成果已发表于国际著名生物医学期刊基因与发育，华益民为第一作者兼共同通讯作者。

脊髓性肌肉萎缩症为隐性常染色体遗传病，是婴幼儿死亡的第一杀手。在最新研究中，华益民和美国合作者设计了一个称作“诱饵”的正义寡核苷酸，并将其注射到小鼠脑室中，通过扰乱药物在中枢神经系统的作用，确保运动神经元存活蛋白表达的增加仅局限于外周组织。令人惊奇的是，这种治疗途径能延长小鼠寿命达20余倍。该发现突破该领域现有观点，即认为提高脊髓中运动神经元存活蛋白的表达是治疗脊髓性肌肉萎缩症的必要条件。

原始出处：

Hua Y1, Liu YH2, Sahashi K2, Rigo F3, Bennett CF3, Krainer AR4.Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models.Genes Dev. 2015 Feb 1;29(3):288-97. doi: 10.1101/gad.256644.114. Epub 2015 Jan 12.

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    2016-01-07 fzwish20000

    #Dev#

    0

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    2015-07-26 cy0324

    #Gene#

    0

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    2015-07-09 cmn

    中国学者

    0

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    2015-11-23 chendoc252

    #萎缩#

    0

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    2015-02-16 windmilL1989

    已阅

    0

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    2015-02-06 zhouqu_8

    #髓性#

    0

  7. [GetPortalCommentsPageByObjectIdResponse(id=1982213, encodeId=b5671982213c7, content=<a href='/topic/show?id=13b658e849' target=_blank style='color:#2F92EE;'>#Dev#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=67, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5878, encryptionId=13b658e849, topicName=Dev)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=bcaa382, createdName=fzwish20000, createdTime=Thu Jan 07 03:15:00 CST 2016, time=2016-01-07, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2048643, encodeId=a28a204864338, content=<a href='/topic/show?id=4363e929ad' target=_blank style='color:#2F92EE;'>#Gene#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=49, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7929, encryptionId=4363e929ad, topicName=Gene)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=63a4277, createdName=cy0324, createdTime=Sun Jul 26 16:15:00 CST 2015, time=2015-07-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=32168, encodeId=7e9832168e7, content=中国学者, beContent=null, objectType=article, channel=null, level=null, likeNumber=109, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyhRUzmFEmHvdIaOns4DmF3BicVffXjkOjBTiaMUIA4o7feGvE5IJ0SfSco4ib955Rx7bvWlib7j9V15xp/0, createdBy=814d1631326, createdName=cmn, createdTime=Thu Jul 09 21:03:00 CST 2015, time=2015-07-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1695753, encodeId=9f411695e5312, content=<a href='/topic/show?id=03b98809697' target=_blank style='color:#2F92EE;'>#萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=50, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=88096, encryptionId=03b98809697, topicName=萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=53fc29868494, createdName=chendoc252, createdTime=Mon Nov 23 03:15:00 CST 2015, time=2015-11-23, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=15939, encodeId=01c7159393a, content=已阅, beContent=null, objectType=article, channel=null, level=null, likeNumber=88, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c68e1614119, createdName=windmilL1989, createdTime=Mon Feb 16 14:51:00 CST 2015, time=2015-02-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1514389, encodeId=32fd151438934, content=<a href='/topic/show?id=1ae010211143' target=_blank style='color:#2F92EE;'>#髓性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=102111, encryptionId=1ae010211143, topicName=髓性)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Fri Feb 06 05:15:00 CST 2015, time=2015-02-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1584175, encodeId=fd8a15841e533, content=<a href='/topic/show?id=f3308101eee' target=_blank style='color:#2F92EE;'>#肌肉萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=74, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81017, encryptionId=f3308101eee, topicName=肌肉萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=177b17012429, createdName=12498ebem24暂无昵称, createdTime=Fri Feb 06 05:15:00 CST 2015, time=2015-02-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1614082, encodeId=5af316140829d, content=<a href='/topic/show?id=b05281019f8' target=_blank style='color:#2F92EE;'>#肌肉萎缩症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=58, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81019, encryptionId=b05281019f8, topicName=肌肉萎缩症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c28719479710, createdName=12498768m19(暂无昵称), createdTime=Fri Feb 06 05:15:00 CST 2015, time=2015-02-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=15070, encodeId=94ab150e0e6, content=还是基础研究而已, beContent=null, objectType=article, channel=null, level=null, likeNumber=105, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c4a5105539, createdName=lovetcm, createdTime=Wed Feb 04 23:49:00 CST 2015, time=2015-02-04, status=1, ipAttribution=)]
    2015-02-06 12498ebem24暂无昵称

    #肌肉萎缩#

    0

  8. [GetPortalCommentsPageByObjectIdResponse(id=1982213, encodeId=b5671982213c7, content=<a href='/topic/show?id=13b658e849' target=_blank style='color:#2F92EE;'>#Dev#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=67, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5878, encryptionId=13b658e849, topicName=Dev)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=bcaa382, createdName=fzwish20000, createdTime=Thu Jan 07 03:15:00 CST 2016, time=2016-01-07, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2048643, encodeId=a28a204864338, content=<a href='/topic/show?id=4363e929ad' target=_blank style='color:#2F92EE;'>#Gene#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=49, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7929, encryptionId=4363e929ad, topicName=Gene)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=63a4277, createdName=cy0324, createdTime=Sun Jul 26 16:15:00 CST 2015, time=2015-07-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=32168, encodeId=7e9832168e7, content=中国学者, beContent=null, objectType=article, channel=null, level=null, likeNumber=109, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyhRUzmFEmHvdIaOns4DmF3BicVffXjkOjBTiaMUIA4o7feGvE5IJ0SfSco4ib955Rx7bvWlib7j9V15xp/0, createdBy=814d1631326, createdName=cmn, createdTime=Thu Jul 09 21:03:00 CST 2015, time=2015-07-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1695753, encodeId=9f411695e5312, content=<a href='/topic/show?id=03b98809697' target=_blank style='color:#2F92EE;'>#萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=50, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=88096, encryptionId=03b98809697, topicName=萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=53fc29868494, createdName=chendoc252, createdTime=Mon Nov 23 03:15:00 CST 2015, time=2015-11-23, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=15939, encodeId=01c7159393a, content=已阅, beContent=null, objectType=article, channel=null, level=null, likeNumber=88, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c68e1614119, createdName=windmilL1989, createdTime=Mon Feb 16 14:51:00 CST 2015, time=2015-02-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1514389, encodeId=32fd151438934, content=<a href='/topic/show?id=1ae010211143' target=_blank style='color:#2F92EE;'>#髓性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=102111, encryptionId=1ae010211143, topicName=髓性)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Fri Feb 06 05:15:00 CST 2015, time=2015-02-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1584175, encodeId=fd8a15841e533, content=<a href='/topic/show?id=f3308101eee' target=_blank style='color:#2F92EE;'>#肌肉萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=74, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81017, encryptionId=f3308101eee, topicName=肌肉萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=177b17012429, createdName=12498ebem24暂无昵称, createdTime=Fri Feb 06 05:15:00 CST 2015, time=2015-02-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1614082, encodeId=5af316140829d, content=<a href='/topic/show?id=b05281019f8' target=_blank style='color:#2F92EE;'>#肌肉萎缩症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=58, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81019, encryptionId=b05281019f8, topicName=肌肉萎缩症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c28719479710, createdName=12498768m19(暂无昵称), createdTime=Fri Feb 06 05:15:00 CST 2015, time=2015-02-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=15070, encodeId=94ab150e0e6, content=还是基础研究而已, beContent=null, objectType=article, channel=null, level=null, likeNumber=105, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c4a5105539, createdName=lovetcm, createdTime=Wed Feb 04 23:49:00 CST 2015, time=2015-02-04, status=1, ipAttribution=)]
    2015-02-06 12498768m19(暂无昵称)

    #肌肉萎缩症#

    0

  9. [GetPortalCommentsPageByObjectIdResponse(id=1982213, encodeId=b5671982213c7, content=<a href='/topic/show?id=13b658e849' target=_blank style='color:#2F92EE;'>#Dev#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=67, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5878, encryptionId=13b658e849, topicName=Dev)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=bcaa382, createdName=fzwish20000, createdTime=Thu Jan 07 03:15:00 CST 2016, time=2016-01-07, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2048643, encodeId=a28a204864338, content=<a href='/topic/show?id=4363e929ad' target=_blank style='color:#2F92EE;'>#Gene#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=49, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7929, encryptionId=4363e929ad, topicName=Gene)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=63a4277, createdName=cy0324, createdTime=Sun Jul 26 16:15:00 CST 2015, time=2015-07-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=32168, encodeId=7e9832168e7, content=中国学者, beContent=null, objectType=article, channel=null, level=null, likeNumber=109, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyhRUzmFEmHvdIaOns4DmF3BicVffXjkOjBTiaMUIA4o7feGvE5IJ0SfSco4ib955Rx7bvWlib7j9V15xp/0, createdBy=814d1631326, createdName=cmn, createdTime=Thu Jul 09 21:03:00 CST 2015, time=2015-07-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1695753, encodeId=9f411695e5312, content=<a href='/topic/show?id=03b98809697' target=_blank style='color:#2F92EE;'>#萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=50, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=88096, encryptionId=03b98809697, topicName=萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=53fc29868494, createdName=chendoc252, createdTime=Mon Nov 23 03:15:00 CST 2015, time=2015-11-23, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=15939, encodeId=01c7159393a, content=已阅, beContent=null, objectType=article, channel=null, level=null, likeNumber=88, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c68e1614119, createdName=windmilL1989, createdTime=Mon Feb 16 14:51:00 CST 2015, time=2015-02-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1514389, encodeId=32fd151438934, content=<a href='/topic/show?id=1ae010211143' target=_blank style='color:#2F92EE;'>#髓性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=102111, encryptionId=1ae010211143, topicName=髓性)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Fri Feb 06 05:15:00 CST 2015, time=2015-02-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1584175, encodeId=fd8a15841e533, content=<a href='/topic/show?id=f3308101eee' target=_blank style='color:#2F92EE;'>#肌肉萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=74, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81017, encryptionId=f3308101eee, topicName=肌肉萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=177b17012429, createdName=12498ebem24暂无昵称, createdTime=Fri Feb 06 05:15:00 CST 2015, time=2015-02-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1614082, encodeId=5af316140829d, content=<a href='/topic/show?id=b05281019f8' target=_blank style='color:#2F92EE;'>#肌肉萎缩症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=58, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81019, encryptionId=b05281019f8, topicName=肌肉萎缩症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c28719479710, createdName=12498768m19(暂无昵称), createdTime=Fri Feb 06 05:15:00 CST 2015, time=2015-02-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=15070, encodeId=94ab150e0e6, content=还是基础研究而已, beContent=null, objectType=article, channel=null, level=null, likeNumber=105, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c4a5105539, createdName=lovetcm, createdTime=Wed Feb 04 23:49:00 CST 2015, time=2015-02-04, status=1, ipAttribution=)]
    2015-02-04 lovetcm

    还是基础研究而已

    0

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