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X连锁痉挛性截瘫(SPG2):单个基因位点的临床异质性。

X linked spastic paraplegia (SPG2): clinical heterogeneity at a single gene locus.

作者信息

Bonneau D, Rozet J M, Bulteau C, Berthier M, Mettey R, Gil R, Munnich A, Le Merrer M

机构信息

Unité de Génétique Médicale, Centre Hospitalier Universitaire, Poitiers, France.

出版信息

J Med Genet. 1993 May;30(5):381-4. doi: 10.1136/jmg.30.5.381.

Abstract

X linked hereditary spastic paraplegia is a rare condition that has been divided into two forms (the pure spastic form and the complicated form) as a function of clinical course and severity. A gene for pure hereditary spastic paraplegia (SPG2) has been mapped to the proximal long arm of the X chromosome (Xq21) by linkage to the DXS17 locus, while a gene for a complicated form of the disease has been mapped to the distal long arm by linkage to the DXS52 locus (Xq28). Here we report on the mapping of a gene for complicated hereditary spastic paraplegia to the Xq21 region by linkage to the probe S9 at the DXS17 locus (Z = 5 at theta = 0.04) in a three generation pedigree. Multipoint linkage analysis supports the distal location of the disease gene with respect to the DXYS1-DXS17 block (cen-DXYS1-DXS3-DXS17-SPG2-tel). The observation of a complicated form of spastic paraplegia mapping to Xq21 raises the difficult issue of variable phenotypic expression, allelic heterogeneity, or even close proximity of two genes for hereditary spastic paraplegia in this region. However, since our study provides clinical evidence for intrafamilial heterogeneity in complicated X linked spastic paraplegia, the present data support the hypothesis of variable clinical expression of a single gene at the SPG2 locus, as previously suggested for SPG1. Finally, we report here what we believe to be the first evidence of clinical expression in heterozygous carriers, a feature that is relevant to genetic counselling in at risk females.

摘要

X连锁遗传性痉挛性截瘫是一种罕见疾病,根据临床病程和严重程度可分为两种类型(单纯痉挛型和复杂型)。通过与DXS17位点连锁,已将纯遗传性痉挛性截瘫(SPG2)基因定位于X染色体长臂近端(Xq21),而通过与DXS52位点连锁(Xq28),已将该疾病复杂型的一个基因定位于长臂远端。在此,我们报告在一个三代家系中,通过与DXS17位点的探针S9连锁(在θ=0.04时Z=5),将复杂遗传性痉挛性截瘫基因定位于Xq21区域。多点连锁分析支持疾病基因相对于DXYS1-DXS17区域(着丝粒-DXYS1-DXS3-DXS17-SPG2-端粒)位于远端。观察到复杂型痉挛性截瘫定位于Xq21,这就提出了一个难题,即该区域存在可变的表型表达、等位基因异质性,甚至可能存在两个遗传性痉挛性截瘫基因紧密相邻的情况。然而,由于我们的研究为复杂型X连锁痉挛性截瘫的家系内异质性提供了临床证据,目前的数据支持之前针对SPG1所提出的假设,即在SPG2位点单个基因存在可变的临床表达。最后,我们在此报告我们认为是杂合子携带者临床表达的首个证据,这一特征与对有风险女性进行遗传咨询相关。

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