Department of Medical and Molecular Genetics, Kings College, London, SE1 9RT, UK.
Am J Hum Genet. 2010 Mar 12;86(3):485-9. doi: 10.1016/j.ajhg.2010.02.006. Epub 2010 Mar 4.
Brown-Vialetto-Van Laere syndrome is a rare neurological disorder with a variable age at onset and clinical course. The key features are progressive ponto-bulbar palsy and bilateral sensorineural deafness. A complex neurological phenotype with a mixed picture of upper and lower motor neuron involvement reminiscent of amyotrophic lateral sclerosis evolves with disease progression. We identified a candidate gene, C20orf54, by studying a consanguineous family with multiple affected individuals and subsequently demonstrated that mutations in this gene were the cause of disease in other, unrelated families.
Brown-Vialetto-Van Laere 综合征是一种罕见的神经疾病,其发病年龄和临床病程具有变异性。主要特征是进行性桥脑延髓麻痹和双侧感觉神经性耳聋。随着疾病的进展,会出现一种复杂的神经表型,具有上下运动神经元受累的混合表现,类似于肌萎缩侧索硬化症。我们通过研究一个有多个受影响个体的近亲家族,确定了一个候选基因 C20orf54,随后证明该基因的突变是其他不相关家族疾病的原因。