Skerrett Rebecca, Malm Tarja, Landreth Gary
Department of Neurosciences, Case Western Reserve University, School of Medicine, Cleveland, OH 44106, USA.
Department of Neurosciences, Case Western Reserve University, School of Medicine, Cleveland, OH 44106, USA; A.I. Virtanen Institute for Molecular Sciences, Department of Neurobiology, University of Eastern Finland, Neulaniementie 2, 70211 Kuopio, Finland.
Neurobiol Dis. 2014 Dec;72 Pt A:104-16. doi: 10.1016/j.nbd.2014.05.019. Epub 2014 May 27.
Nuclear receptors have generated substantial interest in the past decade as potential therapeutic targets for the treatment of neurodegenerative disorders. Despite years of effort, effective treatments for progressive neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, Huntington's disease and ALS remain elusive, making non-classical drug targets such as nuclear receptors an attractive alternative. A substantial literature in mouse models of disease and several clinical trials have investigated the role of nuclear receptors in various neurodegenerative disorders, most prominently AD. These studies have met with mixed results, yet the majority of studies in mouse models report positive outcomes. The mechanisms by which nuclear receptor agonists affect disease pathology remain unclear. Deciphering the complex signaling underlying nuclear receptor action in neurodegenerative diseases is essential for understanding this variability in preclinical studies, and for the successful translation of nuclear receptor agonists into clinical therapies.
在过去十年中,核受体作为治疗神经退行性疾病的潜在治疗靶点引起了广泛关注。尽管经过多年努力,针对阿尔茨海默病、帕金森病、亨廷顿舞蹈病和肌萎缩侧索硬化症等进行性神经退行性疾病的有效治疗方法仍然难以捉摸,这使得核受体等非经典药物靶点成为有吸引力的替代选择。大量关于疾病小鼠模型的文献以及多项临床试验研究了核受体在各种神经退行性疾病中的作用,其中最突出的是阿尔茨海默病。这些研究结果不一,但大多数小鼠模型研究报告了积极结果。核受体激动剂影响疾病病理的机制仍不清楚。破解神经退行性疾病中核受体作用背后的复杂信号对于理解临床前研究中的这种变异性以及将核受体激动剂成功转化为临床治疗至关重要。