Byun Seulgi, Lee Mijung, Kim Manho
Department of Neurology, Seoul National University Hospital, Seoul, Korea.
Neurobiology Section, Division of Biological Science, University of California San Diego, La Jolla, CA, USA.
J Mov Disord. 2022 Jan;15(1):15-20. doi: 10.14802/jmd.21006. Epub 2021 Nov 17.
Huntington's disease (HD) has become a target of the first clinical trials for gene therapy among movement disorders with a genetic origin. More than 100 clinical trials regarding HD have been tried, but all failed, although there were some improvements limited to symptomatic support. Compared to other neurogenetic disorders, HD is known to have a single genetic target. Thus, this is an advantage and its cure is more feasible than any other movement disorder with heterogeneous genetic causes. In this review paper, the authors attempt to cover the characteristics of HD itself while providing an overview of the gene transfer methods currently being researched, and will introduce an experimental trial with a preclinical model of HD followed by an update on the ongoing clinical trials for patients with HD.
亨廷顿舞蹈症(HD)已成为基因治疗在遗传性运动障碍领域的首个临床试验对象。尽管有一些改善仅限于症状支持,但针对HD的100多项临床试验均已失败。与其他神经遗传性疾病相比,HD已知有单一的遗传靶点。因此,这是一个优势,其治愈比任何其他具有异质性遗传病因的运动障碍更可行。在这篇综述文章中,作者试图在介绍目前正在研究的基因转移方法的同时,涵盖HD本身的特征,并将介绍HD临床前模型的实验性试验,随后更新HD患者正在进行的临床试验情况。
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