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KLHL7的BTB结构域中双等位基因c.181_183delTGT与Crisponi/CISS1样和Bohring-Opitz样综合征的重叠表型相关。

Bi-allelic c.181_183delTGT in BTB domain of KLHL7 is associated with overlapping phenotypes of Crisponi/CISS1-like and Bohring-Opitz like syndrome.

作者信息

Kanthi Anil, Hebbar Malavika, Bielas Stephanie L, Girisha Katta M, Shukla Anju

机构信息

Department of Medical Genetics, Kasturba Medical College, Manipal, Manipal Academy of Higher Education, Manipal, India.

Department of Human Genetics, University of Michigan, Ann Arbor, MI, USA.

出版信息

Eur J Med Genet. 2019 Jun;62(6):103528. doi: 10.1016/j.ejmg.2018.08.009. Epub 2018 Aug 22.

Abstract

Biallelic pathogenic variants in KLHL7 are known to result in Crisponi syndrome (CS)/cold-induced sweating syndrome type 1 (CISS1) like phenotype and Bohring-Opitz-like syndrome. In this report, a trio whole-exome sequencing (WES) was performed in proband with cold-induced sweating, microcephaly, facial dysmorphism, spasticity, failure to thrive, pigmentary abnormalities of the retina, hypoplasia of corpus callosum and periventricular nodular heterotopia. A novel homozygous in-frame deletion was identified in exon 2 of KLHL7, affecting the BTB domain of the protein. Our findings expand the clinical and molecular spectrum of KLHL7-related disorders.

摘要

已知KLHL7双等位基因致病变异会导致类似克里斯波尼综合征(CS)/1型冷诱导出汗综合征(CISS1)的表型以及类似博林-奥皮茨综合征。在本报告中,对一名患有冷诱导出汗、小头畸形、面部畸形、痉挛、生长发育迟缓、视网膜色素沉着异常、胼胝体发育不全和脑室周围结节性异位症的先证者进行了三人全外显子测序(WES)。在KLHL7的第2外显子中发现了一种新的纯合框内缺失,影响该蛋白的BTB结构域。我们的研究结果扩展了KLHL7相关疾病的临床和分子谱。

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