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一个 17q24.3 重复序列被鉴定存在于一个带有短指-并指畸形的大型中国家族中。

A 17q24.3 duplication identified in a large Chinese family with brachydactyly-anonychia.

机构信息

Department of Obstetrics/Gynecology, Joint Laboratory of Reproductive Medicine (SCU-CUHK), Key Laboratory of Obstetric, Gynecologic and Pediatric Diseases and Birth Defects of Ministry of Education, West China Second University Hospital, Sichuan University, Chengdu, China.

State Key Laboratory of Biotherapy and Cancer Center, West China Hospital, Sichuan University and Collaborative Innovation Center, Chengdu, China.

出版信息

Mol Genet Genomic Med. 2020 Sep;8(9):e1392. doi: 10.1002/mgg3.1392. Epub 2020 Jun 25.

Abstract

BACKGROUND

Brachydactyly (BD) is a rare autosomal dominant inherited disease characterized by shortness of the fingers and/or toes, which has been classified into the subtypes A-E. However, the exact cause and mechanism of BD remain to be illuminated. Here, we aim to reveal the clinical and genetic characteristics of a subtype of BD, brachydactyly-anonychia.

METHODS

In this study, a large Chinese family with three members affected by brachydactyly-anonychia was investigated. Both whole-exome sequencing and microarray-based comparative genomic hybridization (CGH) were performed on this family and the results of copy number variation (CNV) were verified by quantitative real-time PCR (qPCR).

RESULTS

All the affected individuals showed short fingers and toes as well as missing nails; and the absence of middle phalanges in figure II-V of the upper and lower extremities was observed by X-ray examination. A duplication involving in the region of 17q24.3 was detected by CGH. The results of qPCR also represented this duplication in 17q24.3 in all the patients.

CONCLUSION

In summary, our findings suggest that 17q24.3 duplication is the genetic cause of brachydactyly-anonychia in this family, which support the prior report that brachydactyly-anonychia is associated with 17q24.3 duplication, and further indicates the pathogenic correlation between BD and CNVs.

摘要

背景

短指(BD)是一种罕见的常染色体显性遗传疾病,其特征为手指和/或脚趾短小,已分为 A-E 亚型。然而,BD 的确切病因和机制仍不清楚。在此,我们旨在揭示一种短指-无甲亚型的临床和遗传特征。

方法

本研究对一个受短指-无甲影响的中国大家庭进行了研究。对该家系进行了全外显子组测序和基于微阵列的比较基因组杂交(CGH),并通过实时定量 PCR(qPCR)验证了拷贝数变异(CNV)的结果。

结果

所有受影响的个体均表现为手指和脚趾短小,且无指甲;X 射线检查显示上肢和下肢 II-V 指(趾)骨中节缺失。CGH 检测到 17q24.3 区域的重复。qPCR 的结果也在所有患者的 17q24.3 中显示出这种重复。

结论

总之,我们的研究结果表明,17q24.3 重复是该家系短指-无甲的遗传原因,支持先前关于短指-无甲与 17q24.3 重复相关的报道,并进一步表明 BD 与 CNVs 之间存在致病相关性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/147f/7507485/c3e0ca24df90/MGG3-8-e1392-g001.jpg

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