Rodrigues Filipe B, Byrne Lauren M, Wild Edward J
Huntington's Disease Centre, Department of Neurodegenerative Disease, Institute of Neurology, University College London, London, UK.
Methods Mol Biol. 2018;1780:329-396. doi: 10.1007/978-1-4939-7825-0_17.
Huntington's disease (HD) is a chronic progressive neurodegenerative condition where new markers of disease progression are needed. So far no disease-modifying interventions have been found, and few interventions have been proven to alleviate symptoms. This may be partially explained by the lack of reliable indicators of disease severity, progression, and phenotype.Biofluid biomarkers may bring advantages in addition to clinical measures, such as reliability, reproducibility, price, accuracy, and direct quantification of pathobiological processes at the molecular level; and in addition to empowering clinical trials, they have the potential to generate useful hypotheses for new drug development.In this chapter we review biofluid biomarker reports in HD, emphasizing those we feel are likely to be closest to clinical applicability.
亨廷顿舞蹈症(HD)是一种慢性进行性神经退行性疾病,需要新的疾病进展标志物。到目前为止,尚未发现改变疾病进程的干预措施,且很少有干预措施被证明能缓解症状。这可能部分归因于缺乏疾病严重程度、进展和表型的可靠指标。生物流体生物标志物除了具有临床测量的优点外,还可能带来其他优势,如可靠性、可重复性、价格、准确性以及在分子水平直接定量病理生物学过程;除了助力临床试验外,它们还有潜力为新药研发产生有用的假设。在本章中,我们回顾了亨廷顿舞蹈症生物流体生物标志物的报告,重点关注那些我们认为可能最接近临床应用的报告。