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首页> 外文期刊>Expert opinion on therapeutic targets >Novel therapeutic targets for Huntington's disease.
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Novel therapeutic targets for Huntington's disease.

机译：亨廷顿舞蹈病的新型治疗靶标。

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摘要

Huntington's disease (HD) is a fatal autosomal-dominant disorder involving progressive motor, cognitive and psychiatric symptoms. HD is one of a large family of neurodegenerative diseases caused by a trinucleotide (CAG) repeat mutation, encoding an expanded tract of glutamines in the disease protein. HD was one of the first neurological disorders for which accurate transgenic models were created, allowing mechanisms of pathogenesis to be explored at molecular, cellular and behavioural levels. In the last decade, the understanding of molecular and cellular changes which occur in HD prior to onset of symptoms, and at early and late stages of disease progression, has been greatly expanded. A wide range of potential molecular targets for therapeutic intervention have been identified, associated with a variety of cellular processes including gene transcription, protein trafficking, protein degradation, protein-protein interactions, glutamatergic synaptic transmission, presynaptic signalling, postsynaptic signalling, synaptic plasticity, dopaminergic and neurotrophic modulation of synaptic function, experience-dependent neurogenesis, mitochondrial function and oxidative metabolism. Presymptomatic testing for the HD gene mutation necessitates future development of novel therapeutics aimed at delaying onset of symptoms, as well as slowing or reversing disease progression.
机译：亨廷顿舞蹈病（HD）是一种致命的常染色体显性疾病，涉及进行性运动，认知和精神病学症状。 HD是由三核苷酸（CAG）重复突变引起的神经退行性疾病的一大家族，该突变编码疾病蛋白中谷氨酰胺的扩展片段。 HD是为之建立准确转基因模型的首批神经系统疾病之一，可在分子，细胞和行为水平上探讨发病机理。在过去的十年中，人们对在症状发作之前以及疾病发展的早期和晚期阶段发生于HD的分子和细胞变化的了解已大大扩展。已经确定了用于治疗干预的广泛潜在分子靶标，与各种细胞过程相关，包括基因转录，蛋白质运输，蛋白质降解，蛋白质-蛋白质相互作用，谷氨酸能突触传递，突触前信号传导，突触后信号传导，突触可塑性，多巴胺能和神经营养调节突触功能，经验依赖的神经发生，线粒体功能和氧化代谢。对HD基因突变的症状前测试需要将来开发新的治疗方法，以延缓症状的发作以及减慢或逆转疾病的进展。

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