导致痉挛性截瘫的 FARS2 假杂合错义变异和 Alu 介导的第 5 号外显子缺失 77.

IF 4.4 2区 医学 Q1 CLINICAL NEUROLOGY
Annals of Clinical and Translational Neurology Pub Date : 2024-11-01 Epub Date: 2024-09-28 DOI:10.1002/acn3.52195
Shu-Huai Lin, Jun-Hao Xie, Jun-Yi Jiang, Xin-Yu Yan, Chao-Yin Hong, Wan-Jin Chen, Ning Wang, Xiang Lin
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引用次数: 0

摘要

FARS2相关遗传性痉挛性截瘫(77型晚发痉挛性截瘫)是一种罕见的神经退行性疾病。在此,我们报告了一个常染色体隐性遗传痉挛性截瘫家族中的两个受影响的兄弟姐妹,他们的FARS2基因存在假同源错义变异和Alu介导的第5外显子缺失。两名患者都逐渐出现步态改变和双下肢无力。在我们的文献综述中,77 型痉挛性截瘫的临床表现具有高度异质性。我们的研究拓宽了FARS2复合杂合突变导致的SPG77的致病机制,并提供了FARS2缺失是由Alu元件介导的重组事件所致的有力证据。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

A pseudo-homozygous missense variant and Alu-mediated exon 5 deletion in FARS2 causing spastic paraplegia 77.

A pseudo-homozygous missense variant and Alu-mediated exon 5 deletion in FARS2 causing spastic paraplegia 77.

FARS2-associated hereditary spastic paraplegia, later onset spastic paraplegia type 77, is a rarely neurodegenerative disease. Here, we reported two affected siblings in an autosomal recessive spastic paraplegia family with a pseudo-homozygous missense variant and Alu-mediated exon 5 deletion in FARS2. Both patients gradually developed altered gaits and weakness in both lower limbs. In our literature review, spastic paraplegia type 77 shows high heterogeneity in clinical manifestations. Our study broadens the scope of pathogenic mechanisms of SPG77 resulting from compound heterozygous mutations in FARS2 and provides strong evidence that deletion in FARS2 due to recombination event mediated by Alu element.

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来源期刊
Annals of Clinical and Translational Neurology
Annals of Clinical and Translational Neurology Medicine-Neurology (clinical)
CiteScore
9.10
自引率
1.90%
发文量
218
审稿时长
8 weeks
期刊介绍: Annals of Clinical and Translational Neurology is a peer-reviewed journal for rapid dissemination of high-quality research related to all areas of neurology. The journal publishes original research and scholarly reviews focused on the mechanisms and treatments of diseases of the nervous system; high-impact topics in neurologic education; and other topics of interest to the clinical neuroscience community.
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