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微小角膜、虹膜和脉络膜缺损合并整体发育迟缓一例:由中国人 TENM3 致病性变异引起

Microcornea, iris and choroidal coloboma, and global developmental delay caused by TENM3 pathogenic variants in a Chinese patient.

机构信息

Department of Pediatrics, Fujian Provincial Maternity and Children's Hospital, Affiliated Hospital of Fujian Medical University, Fuzhou, China.

Beijing Chigene Translational Medicine Research Center Co., Ltd, Beijing, China.

出版信息

Mol Genet Genomic Med. 2022 Jun;10(6):e1948. doi: 10.1002/mgg3.1948. Epub 2022 Apr 9.

Abstract

BACKGROUND

Biallelic TENM3 pathogenic variants cause isolated or syndromic microphthalmia. Syndromic microphthalmia 15 (MCOPS15) is characterized by microphthalmia, coloboma, and developmental delay. Currently, only four cases of MCOPS15 have been reported and the clinical features varied among the patients indicating potential broad phenotypic spectrum.

METHODS

The present case was a 6-month-old male at diagnosis. The patient exhibited long philtrum, large ears, bilateral ptosis, and nystagmus. Ophthalmic tests showed that he had microcornea, iris and choroidal coloboma. The patient presented with global developmental delay (GDD). Trio-whole exome sequencing and genome copy number sequencing were conducted to explore the disease-causing mutations.

RESULTS

Exome sequencing and genome copy number sequencing showed the presence of L1471F and E661G compound mutations in TENM3, which were inherited from the mother and father, respectively. Sanger sequencing was conducted to verify association of the mutations with the disease in the present family.

CONCLUSION

Two TENM3 variants were identified in a patient with Syndromic microphthalmia 15 in the present study. However, further studies should be conducted to explore the pathogenicity of the variants.

摘要

背景

双等位基因 TENM3 致病变异可导致孤立性或综合征性小眼球症。综合征性小眼球症 15 型(MCOPS15)的特征为小眼球症、虹膜脉络膜缺损和发育迟缓。目前,仅报道了 4 例 MCOPS15 病例,且患者的临床表现存在差异,表明其存在潜在的广泛表型谱。

方法

本病例为诊断时 6 月龄男性。患儿存在长人中、大耳朵、双侧上睑下垂和眼球震颤。眼科检查显示患儿存在小角膜、虹膜和脉络膜缺损。患儿表现为全面发育迟缓(GDD)。对患儿及其父母进行了 trio-whole 外显子组测序和基因组拷贝数测序,以探讨致病突变。

结果

外显子组测序和基因组拷贝数测序显示,患儿携带分别来自母亲和父亲的 TENM3 基因 L1471F 和 E661G 复合杂合突变。对本家系进行了 Sanger 测序以验证突变与疾病的相关性。

结论

本研究在 1 例综合征性小眼球症 15 型患儿中鉴定出 2 种 TENM3 变异,但还需要开展进一步研究以探讨变异的致病性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0a8a/9184666/ca20a23cd2f4/MGG3-10-e1948-g001.jpg

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