1 Department of Neurology, Yale University School of Medicine, New Haven, CT 06510, and Centre for Neuroscience and Regeneration Research, Veterans Affairs Medical Centre, West Haven, CT 06516, USA.
2 Department of Neurology, University Medical Centre Maastricht, Maastricht, The Netherlands.
Brain. 2014 Jun;137(Pt 6):1627-42. doi: 10.1093/brain/awu079. Epub 2014 Apr 27.
Sodium channel Nav1.9 is expressed in peripheral nociceptive neurons, as well as visceral afferents, and has been shown to act as a threshold channel. Painful peripheral neuropathy represents a significant public health challenge and may involve gain-of-function variants in sodium channels that are preferentially expressed in peripheral sensory neurons. Although gain-of-function variants of peripheral sodium channels Nav1.7 and Nav1.8 have recently been found in painful small fibre neuropathy, the aetiology of peripheral neuropathy in many cases remains unknown. We evaluated 459 patients who were referred for possible painful peripheral neuropathy, and confirmed the diagnosis of small fibre neuropathy in a cohort of 393 patients (369 patients with pure small fibre neuropathy, and small fibre neuropathy together with large fibre involvement in an additional 24 patients). From this cohort of 393 patients with peripheral neuropathy, we sequenced SCN11A in 345 patients without mutations in SCN9A and SCN10A, and found eight variants in 12 patients. Functional profiling by electrophysiological recordings showed that these Nav1.9 mutations confer gain-of-function attributes to the channel, depolarize resting membrane potential of dorsal root ganglion neurons, enhance spontaneous firing, and increase evoked firing of these neurons. Our data show, for the first time, missense mutations of Nav1.9 in individuals with painful peripheral neuropathy. These genetic and functional observations identify missense mutations of Nav1.9 as a cause of painful peripheral neuropathy.
钠离子通道 Nav1.9 在外周伤害性神经元和内脏传入神经中表达,并被证明具有阈通道作用。疼痛性周围神经病变是一个重大的公共健康挑战,可能涉及优先在外周感觉神经元中表达的钠离子通道的功能获得性变异。尽管最近在疼痛性小纤维神经病中发现了外周钠离子通道 Nav1.7 和 Nav1.8 的功能获得性变异,但许多情况下周围神经病的病因仍然未知。我们评估了 459 名可能患有疼痛性周围神经病的患者,并在 393 名患者的队列中确认了小纤维神经病的诊断(369 名患者为单纯小纤维神经病,另外 24 名患者为小纤维神经病伴大纤维受累)。在这 393 名周围神经病患者中,我们对 345 名 SCN9A 和 SCN10A 无突变的患者进行了 SCN11A 测序,在 12 名患者中发现了 8 个变异。通过电生理记录进行的功能特征分析表明,这些 Nav1.9 突变赋予通道功能获得性属性,使背根神经节神经元的静息膜电位去极化,增强自发放电,并增加这些神经元的诱发放电。我们的数据首次显示了疼痛性周围神经病患者 Nav1.9 的错义突变。这些遗传和功能观察结果确定了 Nav1.9 的错义突变是疼痛性周围神经病的一个原因。
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