Laboratory of Circadian and Sleep Medicine, Department of Psychiatry and Biobehavioral Sciences, University of California Los Angeles, Los Angeles, California, USA.
PLoS One. 2013 Jul 30;8(7):e69993. doi: 10.1371/journal.pone.0069993. Print 2013.
Sleep and circadian disruptions are commonly reported by patients with neurodegenerative diseases, suggesting these may be an endophenotype of the disorders. Several mouse models of Huntington's disease (HD) that recapitulate the disease progression and motor dysfunction of HD also exhibit sleep and circadian rhythm disruption. Of these, the strongest effects are observed in the transgenic models with multiple copies of mutant huntingtin gene. For developing treatments of the human disease, knock-in (KI) models offer advantages of genetic precision of the insertion and control of mutation copy number. Therefore, we assayed locomotor activity and immobility-defined sleep in a new model of HD with an expansion of the KI repeats (Q175). We found evidence for gene dose- and age-dependent circadian disruption in the behavior of the Q175 line. We did not see evidence for loss of cells or disruption of the molecular oscillator in the master pacemaker, the suprachiasmatic nucleus (SCN). The combination of the precise genetic targeting in the Q175 model and the observed sleep and circadian disruptions make it tractable to study the interaction of the underlying pathology of HD and the mechanisms by which the disruptions occur.
睡眠和昼夜节律紊乱在神经退行性疾病患者中经常被报道,这表明这些可能是这些疾病的内表型。几种亨廷顿病 (HD) 的小鼠模型重现了 HD 的疾病进展和运动功能障碍,也表现出睡眠和昼夜节律紊乱。在这些模型中,具有多个突变亨廷顿基因拷贝的转基因模型观察到的效果最强。对于开发人类疾病的治疗方法,基因敲入 (KI) 模型具有插入的遗传精确性和突变拷贝数控制的优势。因此,我们在具有 KI 重复扩展 (Q175) 的新型 HD 模型中检测了运动活性和由不动性定义的睡眠。我们发现 Q175 系的行为存在与基因剂量和年龄相关的昼夜节律紊乱的证据。我们没有看到在主起搏器即视交叉上核 (SCN) 中细胞缺失或分子振荡器紊乱的证据。Q175 模型中精确的基因靶向与观察到的睡眠和昼夜节律紊乱的结合,使得研究 HD 的潜在病理学和发生紊乱的机制之间的相互作用变得可行。