In X-linked recessive disorders, a few female gene carriers become symptomatic. Recent evidence implicates skewed X-chromosome inactivation in such female carriers. We studied the clinical features of eight female gene carriers of X-linked recessive spinal and bulbar muscular atrophy (SBMA), and evaluated the relationship between phenotype and genotype from the viewpoint of X-chromosome inactivation. Seven of eight cases were symptomatic, showing mild muscle weakness, frequent muscle cramps, slight elevation of the serum creatinine kinase level, or neurogenic changes on the electromyogram. Only one carrier was asymptomatic clinically. For the estimation of X-chromosome inactivation, the methylation status of the androgen receptor (AR) gene was determined by polymerase chain reaction-based assay. Highly skewed inactivation of the affected AR gene was found in the asymptomatic carrier, while symptomatic carriers had a random or lower inactivation pattern of the affected AR gene. These findings suggest that most female carriers of SBMA show some clinical abnormalities, and highly skewed inactivation of the affected X-chromosome seems to closely relate with escape of the manifestation in female carriers of SBMA.

译文

:在X连锁隐性疾病中,一些女性基因携带者有症状。最近的证据表明这种女性携带者中偏斜的X染色体失活。我们研究了X连锁隐性脊髓和延髓性肌萎缩症(SBMA)的八个女性基因携带者的临床特征,并从X染色体失活的角度评估了表型与基因型之间的关系。 8例中有7例是有症状的,表现出轻度的肌肉无力,频繁的肌肉痉挛,血清肌酐激酶水平的轻微升高或肌电图的神经源性改变。临床上仅一种携带者无症状。为了估计X染色体的失活,通过基于聚合酶链反应的测定来确定雄激素受体(AR)基因的甲基化状态。在无症状的携带者中发现受影响的AR基因高度失活,而有症状的携带者具有受影响的AR基因的随机或较低失活模式。这些发现表明,大多数SBMA女性携带者表现出某些临床异常,受影响的X染色体的高度偏斜失活似乎与SBMA女性携带者表现的逃避密切相关。

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