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亨廷顿舞蹈症研究现状 被引量:5

Current Research Status of Huntington's Disease
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摘要 亨廷顿舞蹈症是神经退行性疾病中最重要的单基因遗传病之一,其清晰的遗传学图景为该疾病的研究提供了独特优势。近年来,亨廷顿舞蹈症的机制及潜在治疗方法的研究取得了突破性进展,并且为类似疾病(例如阿尔兹海默氏症、帕金森氏症及共济失调等)的研究提供了思路。该文主要对上述进展作一简单综述。 Huntington's disease is an important neurodegenerative disorder, and its monogenetic nature provides unique advantages for its research. In recent years, major advances have been made in field revealing its mechanisms and promising therapeutic strategies, which provide novel insights into similar diseases such as Alzheimer's disease, Parkinson's disease and ataxias. Here, we briefly review the key recent advances in Huntington's disease research.
作者 安平 鲁伯埙 An Ping;Lu Boxun(State Key Laboratory of Medical Neurobiology,Huashan Hospital,School of Life Sciences,Fudan University,Shanghai 200438,China)
出处 《中国细胞生物学学报》 CAS CSCD 2018年第10期1621-1632,共12页 Chinese Journal of Cell Biology
基金 国家自然科学基金(批准号:81870990 91649105)资助的课题~~
关键词 亨廷顿舞蹈症 神经退行性疾病 HTT polyQ 蛋白质降解 遗传学筛选 基因治疗 Huntington's disease neurodegenerative disorders HTT polyQ protein degradation genetic screening gene therapy
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