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夏科-马里-图思X型:Cx32基因的一种新型突变与中枢传导减慢。

Charcot-Marie-Tooth type X: A novel mutation in the Cx32 gene with central conduction slowing.

作者信息

Seeman P, Mazanec R, Ctvrtecková M, Smilková D

机构信息

Department of Child Neurology, Second School of Medicine, Charles University Prague, V úvalu 84, Praha 5, Czech Republic.

出版信息

Int J Mol Med. 2001 Oct;8(4):461-8.

Abstract

Charcot-Marie-Tooth disease (CMT) is characterized by distal muscle weakness and wasting, often resulting in foot deformities and gait disturbances, distal sensory impairment and by more or less typical changes in sural nerve biopsy. CMT type 1 is also characterized by reduced nerve conduction velocities. For these demyelinating subtypes, most frequently a 1.5 Mb tandem duplication in chromosome 17p11.2-12 comprising the gene for the peripheral myelin protein 22 (PMP22) is observed (CMT1A), but point mutations in PMP22 have also rarely been reported. X-linked, dominant CMTX1 disease is the second most common type of these hereditary motor and sensory neuropathies (HMSN). Mutations in the X chromosomal gene Connexin32 (Cx32) synonymous gap junction beta-1 (GJB1) are detectable in most X-linked CMT families. We report a novel missense mutation--Tyr65His--in the first extracelullar domain of the Cx32 gene in a Czech CMTX1 family. The mutation was not detectable in 50 healthy controls. The clinical phenotype in both the male proband and his mother was moderate with pronounced peroneal weakness and foot drop. Nerve conduction velocities were intermediately decreased (31-38 m/s) in both patients and slowing of central acoustic conduction (BAEP) was found in both the son and the mother whereas visual central conduction slowing (VEP) was detectable only in the son.

摘要

夏科-马里-图思病(CMT)的特征为远端肌肉无力和萎缩,常导致足部畸形和步态障碍、远端感觉障碍以及腓肠神经活检出现或多或少的典型变化。1型CMT的特征还包括神经传导速度降低。对于这些脱髓鞘亚型,最常见的是在17号染色体p11.2-12区域观察到一个1.5 Mb的串联重复,其中包含外周髓鞘蛋白22(PMP22)基因(CMT1A),但PMP22的点突变也有极少报道。X连锁显性CMTX1病是这些遗传性运动和感觉神经病(HMSN)中第二常见的类型。在大多数X连锁CMT家族中可检测到X染色体基因连接蛋白32(Cx32,同义名为缝隙连接蛋白β-1,GJB1)的突变。我们报告了一个捷克CMTX1家族中Cx32基因第一个细胞外结构域的新型错义突变——Tyr65His。在50名健康对照中未检测到该突变。男性先证者及其母亲的临床表型为中度,伴有明显的腓骨肌无力和足下垂。两名患者的神经传导速度中度降低(31 - 38 m/s),儿子和母亲均发现中枢听觉传导(BAEP)减慢,而视觉中枢传导减慢(VEP)仅在儿子中可检测到。

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