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LMOD2 相关性扩张型心肌病,在婴儿晚期发病。

LMOD2-related dilated cardiomyopathy presenting in late infancy.

机构信息

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, Texas, USA.

Department of Pediatrics, Section of Cardiology, Texas Children's Hospital, Houston, Texas, USA.

出版信息

Am J Med Genet A. 2022 Jun;188(6):1858-1862. doi: 10.1002/ajmg.a.62699. Epub 2022 Feb 21.

Abstract

Leiomodin-2 (LMOD2) is an important regulator of the thin filament length, known to promote elongation of actin through polymerization at pointed ends. Mice with Lmod2 deficiency die around 3 weeks of age due to severe dilated cardiomyopathy (DCM), resulting from decreased heart contractility due to shorter thin filaments. To date, there have been three infants from two families reported with biallelic variants in LMOD2, presenting with perinatal onset DCM. Here, we describe a third family with a child harboring a previously described homozygous frameshift variant, c.1243_1244delCT (p.L415Vfs*108) with DCM, presenting later in infancy at 9 months of age. Family history was relevant for a sibling who died suddenly at 1 year of age after being diagnosed with cardiomegaly. LMOD2-related cardiomyopathy is a rare form of inherited cardiomyopathy resulting from thin filament length dysregulation and should be considered in genetic evaluation of newborns and infants with suspected autosomal recessive inheritance or sporadic early onset cardiomyopathy.

摘要

肌球蛋白结合蛋白 2(LMOD2)是调节细肌丝长度的重要调控因子,已知可通过在端点聚合来促进肌动蛋白的伸长。由于细肌丝变短导致心肌收缩力下降,Lmod2 缺陷的小鼠在 3 周龄左右死亡,死于严重的扩张型心肌病(DCM)。迄今为止,已有来自两个家族的 3 名婴儿被报道存在 LMOD2 的双等位基因突变,表现为围产期起病的 DCM。在这里,我们描述了第三个家族,其孩子携带先前描述的纯合移码变异,c.1243_1244delCT(p.L415Vfs*108),患有 DCM,在婴儿期 9 个月时出现较晚。家族史与一个兄弟姐妹有关,该兄弟姐妹在 1 岁时被诊断为心脏扩大后突然死亡。由 LMOD2 引起的心肌病是一种罕见的遗传性心肌病,由细肌丝长度失调引起,在对疑似常染色体隐性遗传或散发性早发心肌病的新生儿和婴儿进行基因评估时,应考虑这种心肌病。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4613/9117498/a0cd14da5016/nihms-1797335-f0001.jpg

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LMOD2-related dilated cardiomyopathy presenting in late infancy.LMOD2 相关性扩张型心肌病,在婴儿晚期发病。
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