Skaggs School of Pharmacy and Pharmaceutical Sciences, University of California, 9500 Gilman Drive, MC0719, La Jolla, San Diego, CA, 92093-0719, USA.
Department of Neurosciences, University of California, 9500 Gilman Drive, MC0719, La Jolla, San Diego, CA, 92093-0719, USA.
J Neurol. 2019 Mar;266(3):551-564. doi: 10.1007/s00415-018-8940-6. Epub 2018 Jun 28.
Huntington's disease (HD) is a fatal neurodegenerative disease caused by mutant HTT gene expansions of CAG triplet repeat numbers that are inherited in an autosomal dominant manner. HD patients display multiple clinical features that are correlated with HTT CAG repeat numbers that include age of disease onset, motor dysfunction, cognitive deficits, compromised daily living capacity, and brain neurodegeneration. It is important to understand the significant relationships of the multiple HD clinical deficits correlated with the number of mutant HTT CAG expansions that are the genetic basis for HD disabilities. Therefore, this review highlights the significant correlations of the HD clinical features of age of onset, motor and cognitive disabilities, decline in living capabilities, weight loss, risk of death, and brain neurodegeneration with respect to their associations with CAG repeat lengths of the HTT gene. Quantitative HTT gene expression patterns analyzed in normal adult human brain regions demonstrated its distribution in areas known to undergo neurodegeneration in HD, as well as in other brain regions. Future investigation of the relationships of the spectrum of clinical HD features with mutant HTT molecular mechanisms will be important to gain understanding of how mutant CAG expansions of the HTT gene result in the devastating disabilities of HD patients.
亨廷顿病 (HD) 是一种致命的神经退行性疾病,由 HTT 基因突变引起,该基因中的 CAG 三核苷酸重复数呈常染色体显性遗传。HD 患者表现出多种与 HTT CAG 重复数相关的临床特征,包括发病年龄、运动功能障碍、认知缺陷、日常生活能力受损以及大脑神经退行性变。了解与突变 HTT CAG 扩展数相关的多个 HD 临床缺陷的显著相关性非常重要,这些相关性是 HD 残疾的遗传基础。因此,本综述重点介绍了 HD 临床特征与 HTT 基因突变 CAG 重复长度的相关性,这些特征包括发病年龄、运动和认知障碍、生活能力下降、体重减轻、死亡风险以及大脑神经退行性变。对正常成年人大脑区域中定量 HTT 基因表达模式的分析表明,它分布在已知在 HD 中发生神经退行性变的区域以及其他大脑区域。未来对 HD 临床特征与突变 HTT 分子机制之间关系的研究将有助于了解 HTT 基因突变 CAG 重复扩展如何导致 HD 患者的毁灭性残疾。