Huntington's disease (HD) is an inherited neurodegenerative disorder caused by the abnormal expansion of CAG trinucleotide repeats in the Huntingtin gene (HTT) located on chromosome 4. It is transmitted in an autosomal dominant manner and is characterized by motor dysfunction, cognitive decline, and emotional disturbances. To date, there are no curative treatments for HD have been developed; current therapeutic approaches focus on symptom relief and comprehensive care through coordinated pharmacological and non-pharmacological methods to manage the diverse phenotypes of the disease. International clinical guidelines for the treatment of HD are continually being revised in an effort to enhance care within a multidisciplinary framework. Additionally, innovative gene and cell therapy strategies are being actively researched and developed to address the complexities of the disorder and improve treatment outcomes. This review endeavours to elucidate the current and emerging gene and cell therapy strategies for HD, offering a detailed insight into the complexities of the disorder and looking forward to future treatment paradigms. Considering the complexity of the underlying mechanisms driving HD, a synergistic treatment strategy that integrates various factors-such as distinct cell types, epigenetic patterns, genetic components, and methods to improve the cerebral microenvironment-may significantly enhance therapeutic outcomes. In the future, we eagerly anticipate ongoing innovations in interdisciplinary research that will bring profound advancements and refinements in the treatment of HD.

中文翻译：

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亨廷顿舞蹈病基因和细胞治疗方法的进展。


亨廷顿病 (HD) 是一种遗传性神经退行性疾病，由位于 4 号染色体的亨廷顿基因 (HTT) 中的 CAG 三核苷酸重复异常扩增引起。它以常染色体显性方式传播，特征为运动功能障碍、认​​知能力下降和情绪困扰。迄今为止，还没有开发出针对 HD 的治疗方法；目前的治疗方法侧重于通过协调的药理学和非药理学方法来缓解症状和综合护理，以管理疾病的不同表型。 HD 治疗的国际临床指南正在不断修订，以加强多学科框架内的护理。此外，正在积极研究和开发创新的基因和细胞治疗策略，以解决该疾病的复杂性并改善治疗结果。这篇综述致力于阐明当前和新兴的 HD 基因和细胞治疗策略，详细了解该疾病的复杂性，并展望未来的治疗范例。考虑到驱动 HD 的潜在机制的复杂性，整合各种因素（例如不同的细胞类型、表观遗传模式、遗传成分和改善大脑微环境的方法）的协同治疗策略可能会显着提高治疗效果。未来，我们热切期待跨学科研究的持续创新，这将为 HD 治疗带来深刻的进步和完善。