Lorenzetti D, Pareyson D, Sghirlanzoni A, Roa B B, Abbas N E, Pandolfo M, Di Donato S, Lupski J R
Istituto Nazionale Neurologico C. Besta, Milan.
Am J Hum Genet. 1995 Jan;56(1):91-8.
Hereditary neuropathy with liability to pressure palsies (HNPP) is an autosomal dominant disorder characterized by recurrent mononeuropathies. A 1.5-Mb deletion in chromosome 17p11.2-p12 has been associated with HNPP. Duplication of the same 1.5-Mb region is known to be associated with Charcot-Marie-Tooth disease type 1 (CMT1A), a more severe peripheral neuropathy characterized by symmetrically slowed nerve conduction velocity (NCV). The CMT1A duplication and HNPP deletion appear to be the reciprocal products of a recombination event involving a repeat element (CMT1A-REP) that flanks the 1.5-Mb region involved in the duplication/deletion. Patients from nine unrelated Italian families who were diagnosed with HNPP on the basis of clinical, electrophysiological, and histological evaluations were analyzed by molecular methods for DNA deletion on chromosome 17p. In all nine families, Southern analysis using a CMT1A-REP probe detected a reduced hybridization signal of a 6.0-kb EcoRI fragment mapping within the distal CMT1A-REP, indicating deletion of one copy of CMT1A-REP in these HNPP patients. Families were also typed with a polymorphic (CA)n repeat and with RFLPs corresponding to loci D17S122, D17S125, and D17S61, which all map within the deleted region. Lack of allelic transmission from affected parent to affected offspring was observed in four informative families, providing an independent indication for deletion. Furthermore, pulsed-field gel electrophoresis analysis of SacII-digested genomic DNA detected junction fragments specific to the 1.5-Mb HNPP deletion in seven of nine Italian families included in this study. These findings suggest that a 1.5-Mb deletion on 17p11.2-p12 is the most common mutation associated with HNPP.
遗传性压力易感性周围神经病(HNPP)是一种常染色体显性疾病,其特征为复发性单神经病。17号染色体p11.2 - p12区域的1.5兆碱基缺失与HNPP相关。已知相同的1.5兆碱基区域的重复与1型遗传性运动感觉神经病(CMT1A)相关,CMT1A是一种更严重的周围神经病,其特征为神经传导速度(NCV)对称减慢。CMT1A重复和HNPP缺失似乎是涉及一个重复元件(CMT1A - REP)的重组事件的相互产物,该重复元件位于与重复/缺失相关的1.5兆碱基区域两侧。对9个不相关的意大利家庭的患者进行了分析,这些患者根据临床、电生理和组织学评估被诊断为HNPP,采用分子方法检测17号染色体p上的DNA缺失。在所有9个家庭中,使用CMT1A - REP探针进行的Southern分析检测到位于远端CMT1A - REP内的一个6.0千碱基EcoRI片段的杂交信号减弱,表明这些HNPP患者中CMT1A - REP的一个拷贝缺失。这些家庭还用一个多态性(CA)n重复以及对应于位点D17S122、D17S125和D17S61的限制性片段长度多态性(RFLP)进行分型,这些位点均位于缺失区域内。在4个信息充分的家庭中观察到受累亲本向受累后代的等位基因传递缺失,这为缺失提供了独立的证据。此外,对用SacII消化的基因组DNA进行的脉冲场凝胶电泳分析在本研究纳入的9个意大利家庭中的7个中检测到了特定于1.5兆碱基HNPP缺失的连接片段。这些发现表明17p11.2 - p12上的1.5兆碱基缺失是与HNPP相关的最常见突变。