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一种新型导致显性中间型腓骨肌萎缩症的动力蛋白 2 突变:病例报告。

A Novel Dynamin 2 Mutation Causing Dominant Intermediate Charcot-Marie-Tooth Neuropathy: Case Report.

机构信息

The University of Alabama at Birmingham, USA.

出版信息

J Investig Med High Impact Case Rep. 2022 Jan-Dec;10:23247096221117801. doi: 10.1177/23247096221117801.

Abstract

Dynamin 2 mutations are associated with Charcot-Marie-Tooth neuropathy. We report two siblings with a novel missense heterozygous point mutation (c.1609 G>A) in the highly conserved pleckstrin homology domain in exon 15 of Dynamin 2 presenting with progressive length-dependent sensorimotor polyneuropathy with mixed demyelinating and axonal features on electrodiagnostic studies. The previously unrecognized missense point mutation, which was inherited from their symptomatic but previously undiagnosed mother, was determined to be likely pathogenic based on a non-conservative amino acid substitution (p.Gly537Ser) that is predicted to damage secondary protein structure or function. This report emphasizes the importance of recognizing inherited neuropathies in clinical practice and evaluating suspected pathogenic gene variants initially classified to be of undetermined clinical significance in family cohorts. These cases add to the spectrum of pathogenic Dynamin 2 mutations associated with dominant-intermediate Charcot-Marie-Tooth neuropathy.

摘要

动力蛋白 2 突变与遗传性运动感觉神经病有关。我们报告了两例患有新型错义杂合点突变(c.1609 G>A)的同胞,该突变位于动力蛋白 2 的高度保守的 PH 结构域内 15 号外显子,表现为进行性长度依赖性感觉运动多发性神经病,电诊断研究显示脱髓鞘和轴索混合特征。该未被识别的错义点突变是从他们有症状但之前未被诊断的母亲那里遗传而来的,根据非保守氨基酸取代(p.Gly537Ser),该突变被认为可能破坏二级蛋白质结构或功能,被确定为可能致病的。本报告强调了在临床实践中识别遗传性神经病的重要性,并评估了最初被归类为家族队列中临床意义不明的疑似致病性基因变异。这些病例增加了与显性中间型遗传性运动感觉神经病相关的致病性动力蛋白 2 突变谱。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b43d/9527530/1745c197ec9b/10.1177_23247096221117801-fig1.jpg

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