Department of Neurology, Icahn School of Medicine at Mount Sinai, New York, NY10029, USA.
School of Health and Rehabilitation Sciences, Speech Pathology, University of Queensland, Brisbane, Queensland, QLD, 4072, Australia.
Cereb Cortex. 2018 Jan 1;28(1):158-166. doi: 10.1093/cercor/bhw363.
Spasmodic dysphonia (SD), or laryngeal dystonia, is an isolated task-specific dystonia of unknown causes and pathophysiology that selectively affects speech production. Using next-generation whole-exome sequencing in SD patients, we computed polygenic risk score from 1804 genetic markers based on a genome-wide association study in another form of similar task-specific focal dystonia, musician's dystonia. We further examined the associations between the polygenic risk score, resting-state functional connectivity abnormalities within the sensorimotor network, and SD clinical characteristics. We found that the polygenic risk of dystonia was significantly associated with decreased functional connectivity in the left premotor/primary sensorimotor and inferior parietal cortices in SD patients. Reduced connectivity of the inferior parietal cortex was correlated with the age of SD onset. The polygenic risk score contained a significant number of genetic variants lying near genes related to synaptic transmission and neural development. Our study identified a polygenic contribution to the overall genetic risk of dystonia in the cohort of SD patients. Associations between the polygenic risk and reduced functional connectivity of the sensorimotor and inferior parietal cortices likely represent an endophenotypic imaging marker of SD, while genes involved in synaptic transmission and neuron development may be linked to the molecular pathophysiology of this disorder.
痉挛性发音障碍(SD),又称喉肌张力障碍,是一种病因和病理生理学不明的孤立性任务特异性肌张力障碍,选择性地影响言语产生。我们在 SD 患者中使用下一代全外显子测序,根据另一项音乐家肌张力障碍的类似任务特异性局灶性肌张力障碍的全基因组关联研究,计算了 1804 个遗传标记的多基因风险评分。我们进一步研究了多基因风险评分、感觉运动网络内静息状态功能连接异常与 SD 临床特征之间的关系。我们发现,肌张力障碍的多基因风险与 SD 患者左前运动/初级感觉运动和下顶叶皮层的功能连接减少显著相关。下顶叶皮层的连接减少与 SD 发病年龄相关。风险评分包含大量位于与突触传递和神经发育相关基因附近的遗传变异。我们的研究确定了 SD 患者队列中整体肌张力障碍遗传风险的多基因贡献。多基因风险与感觉运动和下顶叶皮层功能连接减少之间的关联可能代表 SD 的一种内表型影像学标志物,而涉及突触传递和神经元发育的基因可能与该疾病的分子病理生理学有关。