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亨廷顿舞蹈症的基因治疗:治愈的最终策略?

Gene Therapy for Huntington's Disease: The Final Strategy for a Cure?

作者信息

Byun Seulgi, Lee Mijung, Kim Manho

机构信息

Department of Neurology, Seoul National University Hospital, Seoul, Korea.

Neurobiology Section, Division of Biological Science, University of California San Diego, La Jolla, CA, USA.

出版信息

J Mov Disord. 2022 Jan;15(1):15-20. doi: 10.14802/jmd.21006. Epub 2021 Nov 17.


DOI:10.14802/jmd.21006
PMID:34781633
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8820879/
Abstract

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.

摘要

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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本文引用的文献

[1]
Altered microRNA expression in animal models of Huntington's disease and potential therapeutic strategies.

Neural Regen Res. 2021-11

[2]
Gene therapy for neurodegenerative disorders: advances, insights and prospects.

Acta Pharm Sin B. 2020-8

[3]
Huntington's Disease Clinical Trials Corner: April 2020.

J Huntingtons Dis. 2020

[4]
Huntingtin Lowering Strategies.

Int J Mol Sci. 2020-3-20

[5]
Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives.

Ther Clin Risk Manag. 2019-10-2

[6]
Challenges of gene delivery to the central nervous system and the growing use of biomaterial vectors.

Brain Res Bull. 2019-6-5

[7]
Targeting Huntingtin Expression in Patients with Huntington's Disease.

N Engl J Med. 2019-5-6

[8]
Lipid-Based DNA Therapeutics: Hallmarks of Non-Viral Gene Delivery.

ACS Nano. 2019-4-2

[9]
In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease.

Nat Neurosci. 2019-3-11

[10]
The current state and future directions of RNAi-based therapeutics.

Nat Rev Drug Discov. 2019-6

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