一种新的双等位基因变异进一步描述了prdx3相关的常染色体隐性小脑性共济失调。

IF 1.6 4区 医学 Q3 CLINICAL NEUROLOGY Neurogenetics Pub Date : 2023-01-01 DOI:10.1007/s10048-022-00701-9
Misbahuddin M Rafeeq, Muhammad Umair, Muhammad Bilal, Alaa Hamed Habib, Ahmed Waqas, Ziaullah M Sain, Mohammad Zubair Alam, Raja Hussain Ali
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引用次数: 4

摘要

小脑共济失调(CAs)包括一组罕见的神经系统疾病,其特点是广泛的表型和遗传异质性。在过去的几年里,我们对CA病因学的理解有了显著的提高,并导致了许多共济失调相关基因的发现。在这里,我们描述了一个受影响的个体从一个近亲家庭分离隐性神经发育障碍。先证者表现出全面发育迟缓、小脑萎缩、张力减退、语言障碍、张力障碍和深度听力障碍等特征。全外显子组测序和Sanger测序显示一个双等位无义变异(c.496A > T;PRDX3基因外显子5中的p.Lys166*)在家族中完美分离。这是第三个将PRDX3基因变异与小脑性共济失调联系起来的报道。此外,相关听力障碍进一步描述了PRDX3相关基因的表型。
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A novel biallelic variant further delineates PRDX3-related autosomal recessive cerebellar ataxia.

Cerebellar ataxias (CAs) comprise a rare group of neurological disorders characterized by extensive phenotypic and genetic heterogeneity. In the last several years, our understanding of the CA etiology has increased significantly and resulted in the discoveries of numerous ataxia-associated genes. Herein, we describe a single affected individual from a consanguineous family segregating a recessive neurodevelopmental disorder. The proband showed features such as global developmental delay, cerebellar atrophy, hypotonia, speech issues, dystonia, and profound hearing impairment. Whole-exome sequencing and Sanger sequencing revealed a biallelic nonsense variant (c.496A > T; p.Lys166*) in the exon 5 of the PRDX3 gene that segregated perfectly within the family. This is the third report that associates the PRDX3 gene variant with cerebellar ataxia. In addition, associated hearing impairment further delineates the PRDX3 associated gene phenotypes.

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来源期刊
Neurogenetics
Neurogenetics 医学-临床神经学
CiteScore
3.90
自引率
0.00%
发文量
24
审稿时长
6 months
期刊介绍: Neurogenetics publishes findings that contribute to a better understanding of the genetic basis of normal and abnormal function of the nervous system. Neurogenetic disorders are the main focus of the journal. Neurogenetics therefore includes findings in humans and other organisms that help understand neurological disease mechanisms and publishes papers from many different fields such as biophysics, cell biology, human genetics, neuroanatomy, neurochemistry, neurology, neuropathology, neurosurgery and psychiatry. All papers submitted to Neurogenetics should be of sufficient immediate importance to justify urgent publication. They should present new scientific results. Data merely confirming previously published findings are not acceptable.
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