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古巴弗里德里希共济失调患者家系的非常见特征。

Uncommon features in Cuban families affected with Friedreich ataxia.

机构信息

Center of Medical Genetics, Holguín, Cuba.

出版信息

Neurosci Lett. 2010 Mar 19;472(2):85-9. doi: 10.1016/j.neulet.2010.01.045. Epub 2010 Jan 28.

Abstract

This report describes two families who presented with autosomal recessive ataxia. By means of Polymerase Chain Reaction (PCR) molecular testing we identified expansions in the gene encoding Frataxin (FTX) that is diagnostic of Friedreich ataxia. A history of reproductive loss in the two families, prominent scoliosis deformity preceding the onset of ataxic gait, the presence of a sensitive axonal neuropathy, as well as the common origin of ancestors are unusual features of these families. These cases illustrate the importance of molecular diagnosis in patients with a recessive ataxia. The origin of the expanded gene and the GAA repeat size in the normal population are issues to be further investigated. The molecular diagnosis of Friedreich ataxia is now established in Cuba.

摘要

本报告描述了两个常染色体隐性共济失调的家系。通过聚合酶链反应(PCR)分子检测,我们鉴定出编码 Frataxin(FTX)的基因突变,该突变是弗里德里希共济失调的诊断标志。两个家系均有生殖损失史,共济失调步态前出现明显的脊柱侧凸畸形,存在敏感的轴索性神经病,以及祖先的共同起源,这些都是这些家系的不常见特征。这些病例说明了对隐性共济失调患者进行分子诊断的重要性。扩展基因的起源和正常人群中 GAA 重复大小是有待进一步研究的问题。弗里德里希共济失调的分子诊断现已在古巴建立。

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