Ferner Rosalie E
Department of Neurology, Guy's and St. Thomas' Hospitals, Guy's Hospital, London, UK.
Lancet Neurol. 2007 Apr;6(4):340-51. doi: 10.1016/S1474-4422(07)70075-3.
Historically, neurofibromatosis 1 (NF1) has been inextricably linked with neurofibromatosis 2 (NF2). Both are inherited autosomal-dominant neurocutaneous disorders that have high de novo mutation rates and carry a high risk of tumour formation. However, they are clinically and genetically distinct diseases and should be considered as seperate entities. NF1 is a common disease that mainly affects the skin and peripheral nervous system and causes characteristic bony dysplasia. By contrast, NF2 is a rare disorder with a relative paucity of skin manifestations and high-grade malignancy is unusual. Neurological symptoms are the predominant problem and the cardinal sign is bilateral vestibular schwannomas. In this Review, I discuss the pertinent diagnostic, clinical, and genetic symptoms of NF1 and NF2. I also examine the current views on the pathogenesis of these neurocutaneous disorders in the wake of advances in molecular genetics and the development of mouse models of disease.
从历史上看,神经纤维瘤病1型(NF1)一直与神经纤维瘤病2型(NF2)紧密相连。两者都是常染色体显性遗传的神经皮肤疾病,具有高新发突变率且携带高肿瘤形成风险。然而,它们是临床和遗传上截然不同的疾病,应被视为独立的实体。NF1是一种常见疾病,主要影响皮肤和周围神经系统,并导致特征性骨发育异常。相比之下,NF2是一种罕见疾病,皮肤表现相对较少,高级别恶性肿瘤并不常见。神经症状是主要问题,主要体征是双侧前庭神经鞘瘤。在本综述中,我将讨论NF1和NF2相关的诊断、临床和遗传症状。我还将在分子遗传学取得进展以及疾病小鼠模型得到发展之后,审视关于这些神经皮肤疾病发病机制的当前观点。