Abedrabbo Muna, Kazemian Pardis, Connolly Colúm, Leavitt Blair R
Department of Medical Genetics, Centre for Molecular Medicine and Therapeutics, BC Children's Hospital, University of British Columbia, Vancouver, BC, Canada.
Bloom Funding, New Brunswick, Canada.
J Huntingtons Dis. 2025 Aug;14(3):241-257. doi: 10.1177/18796397251330144. Epub 2025 Mar 28.
Microglia, the resident immune cells of the central nervous system, play a pivotal role in the response to Huntington's disease (HD) pathology. Through both cell-autonomous mechanisms and exposure to external pathogenic stimuli, microglia transition from a resting to an activated state, producing pro-inflammatory cytokines and chemokines that mediate inflammation. While this inflammatory response attempts to have a neuroprotective compensatory effect, chronic microglial activation exacerbates neuroinflammation, neurodegeneration and contributes to disease progression. Evidence from postmortem analyses and neuroimaging studies indicates that activated microglia are present in various stages of HD, correlating with neuronal degeneration and clinical symptoms. Enhanced microglial activation has been identified as an early predictor of disease onset, particularly in premanifest HD, highlighting the potential of targeting microglial pathways for therapeutic interventions. This review explores microglia's dual role in HD pathophysiology, exploring their contributions to both neuroinflammation and neuroprotection. It also examines recent advances in clinical trials aimed at modulating microglial activity, paving the way for novel therapeutic strategies to alter disease progression and improve patient outcomes.
小胶质细胞是中枢神经系统的常驻免疫细胞,在对亨廷顿舞蹈病(HD)病理的反应中起关键作用。通过细胞自主机制和暴露于外部致病刺激,小胶质细胞从静止状态转变为激活状态,产生介导炎症的促炎细胞因子和趋化因子。虽然这种炎症反应试图产生神经保护补偿作用,但慢性小胶质细胞激活会加剧神经炎症、神经退行性变并促进疾病进展。尸检分析和神经影像学研究的证据表明,激活的小胶质细胞存在于HD的各个阶段,与神经元变性和临床症状相关。增强的小胶质细胞激活已被确定为疾病发作的早期预测指标,特别是在症状前HD中,这突出了针对小胶质细胞途径进行治疗干预的潜力。本综述探讨了小胶质细胞在HD病理生理学中的双重作用,探讨了它们对神经炎症和神经保护的贡献。它还研究了旨在调节小胶质细胞活性的临床试验的最新进展,为改变疾病进展和改善患者预后的新型治疗策略铺平了道路。