Suppr超能文献

女孩远端型关节挛缩症源于父系体细胞核嵌合体遗传的 TNNI2 新型变异。

Distal arthrogryposis in a girl arising from a novel TNNI2 variant inherited from paternal somatic mosaicism.

机构信息

Department of Human Genetics, Yokohama City University Graduate School of Medicine, Yokohama, Japan.

Department of Obstetrics and Gynecology, Juntendo University, Tokyo, Japan.

出版信息

J Hum Genet. 2023 May;68(5):363-367. doi: 10.1038/s10038-022-01117-x. Epub 2023 Jan 12.

Abstract

TNNI2 at 11p15.5 encodes troponin I2, fast skeletal type, which is a member of the troponin I gene family and a component of the troponin complex. Distal arthrogryposis (DA) is characterized by congenital limb contractures without primary neurological or muscular effects. DA is inherited in an autosomal dominant fashion and is clinically and genetically heterogeneous. Exome sequencing identified a causative variant in TNNI2 [NM_003282.4:c.532T>C p.(Phe178Leu)] in a Japanese girl with typical DA2b. Interestingly, the familial study using Sanger sequencing suggested a mosaic variant in her healthy father. Subsequent targeted amplicon-based deep sequencing detected the TNNI2 variant with variant allele frequencies of 9.4-17.7% in genomic DNA derived from peripheral blood leukocytes, saliva, hair, and nails in the father. We confirmed a disease-causing variant in TNNI2 in the proband inherited from her asymptomatic father with its somatic variant. Our case demonstrates that careful clinical and genetic evaluation is required in DA.

摘要

TNNI2 在 11p15.5 编码快肌型肌钙蛋白 I2,它是肌钙蛋白 I 基因家族的成员,也是肌钙蛋白复合物的组成部分。远端型关节挛缩症(DA)的特征是先天性肢体挛缩,无原发性神经或肌肉影响。DA 以常染色体显性遗传方式遗传,临床表现和遗传具有异质性。外显子组测序在一名具有典型 DA2b 的日本女孩中发现了 TNNI2 [NM_003282.4:c.532T>C p.(Phe178Leu)]的致病变异。有趣的是,使用 Sanger 测序的家系研究表明她健康的父亲存在镶嵌变异。随后,基于靶向扩增子的深度测序在父亲的外周血白细胞、唾液、头发和指甲的基因组 DNA 中检测到 TNNI2 变异,其变异等位基因频率为 9.4-17.7%。我们在该先证者中证实了 TNNI2 中的致病变异,该变异来自无症状父亲,存在体细胞变异。我们的病例表明,在 DA 中需要进行仔细的临床和遗传评估。

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验