Purpose of Review

This article describes and discusses new potential disease-modifying therapies for Huntington’s disease that are currently in human clinical trials as well as promising new therapies in preclinical development.

Recent Findings

Multiple potential disease-modifying therapeutics for HD are in active development, including direct DNA/gene therapies, RNA modulation, and therapies targeted at aberrant downstream pathways.

Summary

The etiology of Huntington’s disease (HD) is well-known as an abnormally expanded trinucleotide repeat within the huntingtin gene. However, the pathogenesis downstream of the mutant huntingtin gene is complex, involving multiple toxic pathways, including abnormal protein fragmentation and neuroinflammation. The current treatment of HD focuses largely on symptomatic management. This article discusses new, potential disease-modifying therapies that are currently in human clinical trials and preclinical development.

中文翻译：

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亨廷顿舞蹈症：治疗学的新领域

审查目的

本文介绍并讨论了目前正在人类临床试验中使用的，针对亨廷顿氏病的新的潜在疾病改良疗法以及在临床前开发中有望实现的新疗法。

最近的发现

HD的多种潜在疾病缓解疗法正在积极开发中，包括直接DNA /基因疗法，RNA调节以及针对异常下游途径的疗法。

概括

亨廷顿舞蹈病（HD）的病因学众所周知是亨廷顿基因内三核苷酸重复序列异常扩增。但是，亨廷顿蛋白突变基因下游的发病机制很复杂，涉及多种毒性途径，包括异常的蛋白质片段化和神经炎症。目前对HD的治疗主要集中在对症管理上。本文讨论了目前正在人类临床试验和临床前开发中的新的，潜在的疾病缓解疗法。