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患有2型神经纤维瘤病和常染色体显性遗传性听力损失的家族:突变型NF2基因携带者的鉴定

Family with neurofibromatosis type 2 and autosomal dominant hearing loss: identification of carriers of the mutated NF2 gene.

作者信息

Bijlsma E K, Merel P, Fleury P, van Asperen C J, Westerveld A, Delattre O, Thomas G, Hulsebos T J

机构信息

Institute of Human Genetics, University of Amsterdam, Faculty of Medicine, The Netherlands.

出版信息

Hum Genet. 1995 Jul;96(1):1-5. doi: 10.1007/BF00214177.

Abstract

A family is presented in which neurofibromatosis type 2 (NF2) and autosomal dominant hearing loss segregate in an apparently independent way. The presence of the latter condition caused anxiety in all family members at risk for NF2 in whom hearing loss became apparent. Previously, we identified a G-->A transition in the donor splice site of exon 5 of the NF2 gene in a family member with proven NF2. As expected, the mutation was present in two other family members who fulfilled the diagnostic criteria for NF2. Four out of five family members at risk for NF2 developed hearing loss. Two of these had the G-->A transition. The mutation was absent in the two other individuals with hearing loss and in the fifth family member without hearing loss or other clinical symptoms. In this family, the identification of the underlying NF2 gene mutation excluded NF2 as the cause of hearing loss in two potential carriers of the mutated gene. On the other hand, it enabled the identification of two carriers of the NF2 gene mutation who did not fulfill the diagnostic criteria for NF2. They will have to be monitored very carefully for the development of NF2-associated tumors. The consistent association within this family of a relatively mild clinical phenotype with the NF2 mutation, supports earlier suggestions that intrafamilial variability is small in NF2.

摘要

本文报告了一个家系,其中2型神经纤维瘤病(NF2)和常染色体显性遗传性听力损失以明显独立的方式分离。后一种情况的存在使所有有NF2风险且出现听力损失的家庭成员感到焦虑。此前,我们在一名确诊为NF2的家庭成员中,在NF2基因第5外显子的供体剪接位点发现了一个G→A转换。正如预期的那样,该突变也存在于另外两名符合NF2诊断标准的家庭成员中。五名有NF2风险的家庭成员中有四名出现了听力损失。其中两名携带G→A转换突变。另外两名有听力损失的个体以及第五名没有听力损失或其他临床症状的家庭成员中不存在该突变。在这个家系中,潜在的NF2基因突变的发现排除了NF2是两名突变基因携带者听力损失原因的可能性。另一方面,它使得能够识别出两名未达到NF2诊断标准的NF2基因突变携带者。必须对他们进行非常仔细的监测,以防出现与NF2相关的肿瘤。这个家系中相对轻微的临床表型与NF2突变的一致关联,支持了先前关于NF2家系内变异性较小的观点。

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