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SB216763对Rett综合征的有效性

2018-05-20 MedSci MedSci原创

SB216763Rett综合征神经系统疾病
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近日，研究人员描述了一种新药（SB216763）能够在临床前模型中减少Rett综合征特症状并激活Rett综合征特有的休眠神经元

近日，研究人员描述了一种新药（SB216763）能够在临床前模型中减少Rett综合征特症状并激活Rett综合征特有的休眠神经元。

Rett综合征是一种神经系统疾病，它严重影响儿童精神运动发育，发病率为1/10000-1/15000。临床特征为女孩起病，呈进行性智力下降，孤独症行为，手的失用，刻板动作及共济失调。Rett综合征是女性智力残疾的第二大常见原因，Rett综合症的主要遗传因素是MECP2基因的突变。目前没有针对该疾病的特定治疗药物，所以目前的努力集中在对其临床症状的控制上。

巴塞罗那大学遗传学授马内尔·埃斯特勒博士称：“近年来，我们测试了糖原合酶激酶3B（GSK3B）的抑制剂（SB216763）是否可以逆转的部分症状。在临床前模型中，我们使用具有MECP2缺陷的人类Rett综合征小鼠对候选药物进行研究”。研究结果是令人惊喜的，SB216763能够显著延长动物的寿命，显著减少震颤、呼吸困难和活动性限制。但值得注意的是GSK3B的抑制作用也会引起睡眠神经元的“觉醒”：这些脑细胞开始重新获得它们之间的接触，并且神经元突触之间的交流增加。但是无论如何，人们必须意识到，MECP2基因的突变仍然存在，只有纠正它，才能从根本上治疗该疾病。


原始出处：

https://www.europeanpharmaceuticalreview.com/news/75741/sb216763-efficacy-rett-syndrome/

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    2018-05-22 hanhaisha2008

    #SB216763#

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    2018-10-22 ysjykql

    #RET#

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    2018-05-22 智者为医08

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