Neuron：基因疗法有望逆转先天性耳聋

2012-08-13 ZinFingerNase 生物谷

根据一项于2012年7月26日发表在Neuron期刊上的临床前研究，在患有先天性耳聋的小鼠模式动物中，一种新的基因疗法能够逆转由于遗传缺陷而导致的耳聋。论文通讯作者、美国加州大学旧金山分校研究员Lawrence Lustig说，他们是第一次在实验室小鼠中成功地治疗一种遗传性的耳聋。由于一种编码蛋白囊泡谷氨酸转运体-3(vesicular glutamate transporter-3, VGL

根据一项于2012年7月26日发表在Neuron期刊上的临床前研究，在患有先天性耳聋的小鼠模式动物中，一种新的基因疗法能够逆转由于遗传缺陷而导致的耳聋。论文通讯作者、美国加州大学旧金山分校研究员Lawrence Lustig说，他们是第一次在实验室小鼠中成功地治疗一种遗传性的耳聋。

由于一种编码蛋白囊泡谷氨酸转运体-3(vesicular glutamate transporter-3, VGLUT3)的基因发表突变而导致小鼠患上遗传性耳聋。在这项研究中，Lustig和他的研究小组利用这种患上遗传性耳聋的小鼠开展研究。蛋白VGLUT3在内毛细胞(inner hair cell)发送产生听力的信号中发挥着至关重要的作用。研究人员通过注射将基因VGLUT3导入小鼠内耳中，两周之后，所有小鼠都恢复听力。当利用这种方法治疗成年小鼠时，这种改善能够持续7周到一年半的时间，而当治疗新生小鼠时，则能够持续至少9周的时间。

这种疗法并不伤害它们的内耳，而且它甚至还能校正内耳细胞中的一些结构缺陷。因为这种特异性的基因导入方法在动物体内是安全的和有效的，所以这些发现有望在未来用于人类研究之中。

原始出处

Akil O1, Seal RP, Burke K, Wang C, Alemi A, During M, Edwards RH, Lustig LR.Restoration of hearing in the VGLUT3 knockout mouse using virally mediated gene therapy.Neuron. 2012 Jul 26;

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2019-03-01 12555d9bm33暂无昵称

这需要不菲的价钱吧？？

105 0
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2016-01-03 三三五五

棒

176 0
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2014-03-19 113.142.17.132

期待快点临床应用

169 0
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2013-03-26 stfoxst

#先天性耳聋#

73 0
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2013-04-22 柳叶一刀

#Neuron#

60 0
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2012-08-15 huirong

#先天性#

84 0

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