Beijing Tongren Eye Center, Beijing Tongren Hospital, Capital Medical University, Beijing Ophthalmology & Visual Sciences Key Lab, 100730, Beijing, China.
NO.1 Dong Jiao Min Xiang, 100730, Beijing, China.
Orphanet J Rare Dis. 2023 Jul 21;18(1):200. doi: 10.1186/s13023-023-02816-0.
Congenital cataract is a leading cause of treatable childhood blindness and both clinically and genetically heterogeneous. Among the already characterized phenotypes, coralliform cataract is a rare special form of congenital cataracts. Although previous studies had shown that mutations in the γD-crystallin (CRYGD) can result in congenital coralliform cataracts, no conclusive genotype-phenotype correlation might be drawn. Here we aimed to identify the spectrum and frequency of CRYGD gene mutations in congenital coralliform cataracts of Chinese origin.
The medical records of 392 Chinese families with congenital cataracts were reviewed between January 2011 and December 2021. The families, clinically documented to have congenital coralliform cataracts, were screened for mutations in candidate CRYGD gene. The genomic DNA of all subjects was extracted from peripheral blood leukocytes. PCR amplified and direct sequencing were performed to identify the disease-causing mutation.
A total of 12 families with coralliform cataracts were recruited in this study in the past 10 years, accounting for 3.1% of the families with congenital cataracts. Of the 12 families, all affected individuals presented with bilateral non-progressive coralliform cataracts since birth, with the best-corrected Snellen visual acuities ranging from 20/200 to 20/25. A recurrent c.70 C > A (p. P24T) mutation in CRYGD was identified in 10 families (83.3%) with congenital cataract, which co-segregated with all affected individuals and was not observed in unaffected family members or ethnically matched normal controls.
The coralliform cataract is characterized by being bilateral, non-progressive and present at birth. A recurrent p.P24T CRYGD mutation occurs independently in 83.3% of the Chinese families with congenital coralliform cataracts and most likely represents a mutational hot spot, which underscore the relations between coralliform cataract and p.P24T CRYGD.
先天性白内障是可治疗的儿童失明的主要原因,且在临床上和遗传上均具有异质性。在已经确定的表型中,珊瑚状白内障是一种罕见的先天性白内障特殊形式。尽管先前的研究表明,γD-晶体蛋白(CRYGD)的突变可导致先天性珊瑚状白内障,但可能无法得出明确的基因型-表型相关性。在此,我们旨在鉴定中国先天性珊瑚状白内障患者 CRYGD 基因突变的谱和频率。
回顾了 2011 年 1 月至 2021 年 12 月期间 392 个先天性白内障中国家庭的病历。对临床上确诊为先天性珊瑚状白内障的家庭进行候选 CRYGD 基因突变筛查。所有受试者的外周血白细胞基因组 DNA 均通过 PCR 扩增和直接测序进行鉴定,以确定致病突变。
在过去的 10 年中,这项研究共招募了 12 个具有珊瑚状白内障的家庭,占先天性白内障家庭的 3.1%。在这 12 个家庭中,所有受影响的个体均从出生起就表现出双侧非进行性珊瑚状白内障,最佳矫正视力从 20/200 到 20/25 不等。在 10 个(83.3%)先天性白内障家庭中发现了 CRYGD 中的一个重复 c.70C > A(p. P24T)突变,该突变与所有受影响的个体共分离,且未在未受影响的家庭成员或种族匹配的正常对照中观察到。
珊瑚状白内障的特征是双侧、非进行性和出生时存在。在 83.3%的中国先天性珊瑚状白内障家庭中,独立发生了一个重复的 p.P24T CRYGD 突变,这很可能是一个突变热点,突出了珊瑚状白内障与 p.P24T CRYGD 之间的关系。