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常染色体隐性神经退行性共济失调的罕见类型。

Rare forms of autosomal recessive neurodegenerative ataxia.

作者信息

Koenig Michel

机构信息

Institut de Génétique et de Biologie Moléculaire et Céllulaire, CNRS/INSERM/Université Louis-Pasteur, Illkirch, France.

出版信息

Semin Pediatr Neurol. 2003 Sep;10(3):183-92. doi: 10.1016/s1071-9091(03)00027-5.

Abstract

There has been a recent explosion in knowledge regarding the genetic basis of several autosomal recessive ataxias. This article summarizes current information regarding rare forms of recessive ataxias. Friedreich's ataxia and ataxia telangiectasia are dealt with in other articles in this issue. The rarer recessive ataxias can be clinically classified as sensory and spinocerbellar ataxias, cerebellar ataxia with sensory-motor polyneuropathy, and purely cerebellar ataxias. Examples of the first category include ataxia with isolated vitamin E deficiency, abetalipoproteinemia, Refsum's disease, infantile-onset spinocerebellar ataxia, and ataxia with blindness and deafness. Examples of ataxia with sensory-motor polyneuropathy include ataxia with oculomotor apraxia 1 and 2 and spinocerebellar ataxia with neuropathy 1. Examples of purely cerebellar ataxia include autosomal recessive spastic ataxia of Charlevoix-Saguenay and ataxia with hypogonadotropic hypogonadism. This review summarizes the clinical and genetic features of these entities and concludes that the pathogenic basis of such ataxias at this time appear to involve two broad types of processes: free-radical injury and defects of DNA single- or double-strand break repair.

摘要

最近,关于几种常染色体隐性共济失调的遗传基础的知识有了迅猛增长。本文总结了有关罕见形式隐性共济失调的当前信息。本期其他文章中讨论了弗里德赖希共济失调和共济失调毛细血管扩张症。较罕见的隐性共济失调在临床上可分为感觉性和脊髓小脑性共济失调、伴有感觉运动性多神经病的小脑性共济失调以及单纯小脑性共济失调。第一类的例子包括孤立性维生素E缺乏性共济失调、无β脂蛋白血症、雷夫叙姆病、婴儿期起病的脊髓小脑性共济失调以及伴有失明和失聪的共济失调。伴有感觉运动性多神经病的共济失调的例子包括伴有动眼神经失用症1型和2型的共济失调以及伴有神经病的脊髓小脑性共济失调1型。单纯小脑性共济失调的例子包括常染色体隐性遗传性沙勒沃-萨格奈痉挛性共济失调和伴有低促性腺激素性性腺功能减退的共济失调。本综述总结了这些疾病的临床和遗传特征,并得出结论,目前此类共济失调的致病基础似乎涉及两大类过程:自由基损伤和DNA单链或双链断裂修复缺陷。

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