Department of Neuroscience & Center for Translational Research in Neurodegenerative Disease, University of Florida, Gainesville, FL 32669, USA.
Sanders Brown Center on Aging & Department of Physiology, University of Kentucky, Lexington, KY 40504, USA.
Int J Mol Sci. 2021 Jan 26;22(3):1186. doi: 10.3390/ijms22031186.
Tauopathies are a group of more than twenty known disorders that involve progressive neurodegeneration, cognitive decline and pathological tau accumulation. Current therapeutic strategies provide only limited, late-stage symptomatic treatment. This is partly due to lack of understanding of the molecular mechanisms linking tau and cellular dysfunction, especially during the early stages of disease progression. In this study, we treated early stage tau transgenic mice with a multi-target kinase inhibitor to identify novel substrates that contribute to cognitive impairment and exhibit therapeutic potential. Drug treatment significantly ameliorated brain atrophy and cognitive function as determined by behavioral testing and a sensitive imaging technique called manganese-enhanced magnetic resonance imaging (MEMRI) with quantitative R1 mapping. Surprisingly, these benefits occurred despite unchanged hyperphosphorylated tau levels. To elucidate the mechanism behind these improved cognitive outcomes, we performed quantitative proteomics to determine the altered protein network during this early stage in tauopathy and compare this model with the human Alzheimer's disease (AD) proteome. We identified a cluster of preserved pathways shared with human tauopathy with striking potential for broad multi-target kinase intervention. We further report high confidence candidate proteins as novel therapeutically relevant targets for the treatment of tauopathy. Proteomics data are available via ProteomeXchange with identifier PXD023562.
tau 病是一组超过二十种已知的疾病,涉及进行性神经退行性变、认知能力下降和病理性 tau 积累。目前的治疗策略仅提供有限的、晚期的症状治疗。这在一定程度上是由于缺乏对 tau 与细胞功能障碍之间的分子机制的理解,特别是在疾病进展的早期阶段。在这项研究中,我们用一种多靶点激酶抑制剂治疗早期 tau 转基因小鼠,以确定新的底物,这些底物有助于认知障碍,并具有治疗潜力。药物治疗显著改善了大脑萎缩和认知功能,这是通过行为测试和一种称为锰增强磁共振成像(MEMRI)的敏感成像技术与定量 R1 映射来确定的。令人惊讶的是,尽管 tau 的过度磷酸化水平没有改变,但这些益处仍然存在。为了阐明这些改善认知结果背后的机制,我们进行了定量蛋白质组学研究,以确定 tau 病早期改变的蛋白质网络,并将该模型与人类阿尔茨海默病(AD)蛋白质组进行比较。我们发现了一组与人类 tau 病共享的被保留的通路,这些通路具有广泛的多靶点激酶干预的显著潜力。我们进一步报告了高可信度的候选蛋白作为治疗 tau 病的新的治疗相关靶标。蛋白质组学数据可通过 ProteomeXchange 以标识符 PXD023562 获得。