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在中国一个 Sheldon-Hall 综合征(DA2B)家系中首次发现的 MYH3 突变。

A MYH3 mutation identified for the first time in a Chinese family with Sheldon-Hall syndrome (DA2B).

机构信息

Metabolic Bone Disease and Genetic Research Unit, Department of Osteoporosis and Bone Disease, Shanghai Jiao Tong University Affiliated Six People's Hospital, 600 Yi-Shan Rd., Shanghai, 200233, PR China.

Department of Orthopedic Surgery, Shanghai Jiao Tong University Affiliated Six People's Hospital, 600 Yi-Shan Rd., Shanghai, 200233, PR China.

出版信息

Neuromuscul Disord. 2018 May;28(5):456-462. doi: 10.1016/j.nmd.2018.03.002. Epub 2018 Mar 8.

Abstract

Sheldon-Hall syndrome is the most common type of distal arthrogryposis syndromes, also known as distal arthrogryposis 2B (DA2B). Sheldon-Hall syndrome is caused by mutations in the TPM2, TNNI2, TNNT3 or MYH3 gene and characterized by ulnar deviation, camptodactyly, overlapping fingers and scoliosis from birth. We investigated a Chinese family with multiple members who clinically presented with distal arthrogryposis of the hands. In total, 261 subjects including one proband and ten family members from the non-consanguineous Chinese family and 250 healthy volunteers were included and had their genomic DNA extracted. A novel missense mutation in exon 13 of the MYH3 gene, c.1160A > G (p.Tyr387Cys), was identified in the proband and his father through whole-exome sequencing. The proband and six affected family members were confirmed to carry this mutation by Sanger sequencing, although the mutation was not detected in the four unaffected individuals or 250 volunteers. This is the first report of a novel MYH3 mutation being identified as the cause of DA2B in a Chinese family. Our findings confirm that MYH3 gene mutations can be a pathogenic cause of DA2B in Asian patients. This study increases the mutational spectrum in MYH3 and aids genetic counseling and prenatal diagnosis.

摘要

谢尔登-霍尔综合征是最常见的远端型关节挛缩症类型,也称为远端型关节挛缩症 2B(DA2B)。谢尔登-霍尔综合征是由 TPM2、TNNI2、TNNT3 或 MYH3 基因突变引起的,其特征为出生时即出现尺骨偏斜、掌侧挛缩、手指重叠和脊柱侧凸。我们研究了一个有多个成员的中国家族,这些成员临床上均表现为手部远端型关节挛缩。共有 261 名受试者,包括一名先证者和十名非近亲中国家族成员以及 250 名健康志愿者,提取了他们的基因组 DNA。通过全外显子组测序,在先证者及其父亲中发现了 MYH3 基因第 13 外显子中的一个新错义突变 c.1160A>G(p.Tyr387Cys)。通过 Sanger 测序,先证者和六名受影响的家族成员证实携带该突变,尽管该突变未在四名未受影响的个体或 250 名志愿者中检测到。这是首例报道 MYH3 突变可引起中国家族的 DA2B。我们的研究结果证实,MYH3 基因突变可成为亚洲患者 DA2B 的致病原因。本研究增加了 MYH3 的突变谱,有助于遗传咨询和产前诊断。

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