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神经退行性疾病:有一个潜在的基因
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Petsko和其他人还研究了患者的家族性肌萎缩侧多发性硬化症(ALS)、Lou Gehrig病和他描述这项工作的一部分3. 他们知道许多基因突变的ALS编码RNA结合蛋白这些蛋白质聚集体形成的神经元ALS患者。这些蛋白质的表达,FUS TDP43,在酵母中,导致相同的表型。一个屏幕的酵母基因能抑制FUS / TDP43毒性鉴定五个基因和所有编码的RNA结合蛋白。令人兴奋的,一些这些基因的人类同源也显示块FUS /人类神经元与新生鼠TDP43毒性。这些令人鼓舞的结果产生了希望有针对性的基因治疗可能提供一个未来的治疗对于这种可怕的神经退行性疾病。
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  • 讲师:Gregory Petsko(博士)

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