Department of Genetics, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia.
Clin Genet. 2013 Aug;84(2):128-31. doi: 10.1111/cge.12184. Epub 2013 May 27.
Microphthalmia is an important inborn error of eye development that can be associated with multisystem involvement. Anophthalmia is more severe and rarer. Single mutations in an expanding list of genes are known to cause this spectrum of anomaly. In one branch of a multiplex family with microphthalmia and anophthalmia, autozygome analysis excluded all known microphthalmia genes at the time of doing this study. Exome sequencing and autozygome filtration identified a novel homozygous variant in ALDH1A3. Subsequently, we identified another homozygous variant in 2 of the 10 probands with microphthalmia we specifically screened for mutations in ALDH1A3. Interestingly, the other branch of the original family was found to segregate anophthalmia/syndactyly with a novel homozygous SMOC1 variant. Our data support the very recent and independent identification of ALDH1A3 as a disease gene in microphthalmia. Locus heterogeneity should be considered in consanguineous families even for extremely rare phenotypes.
小眼症是一种重要的眼发育先天错误,可与多系统受累相关。无眼症则更为严重且罕见。目前已知一系列基因的单个突变可导致这种异常。在一个具有小眼症和无眼症的多重家族的一个分支中,进行本研究时,单体型分析排除了所有已知的小眼症基因。外显子组测序和单体型过滤鉴定出 ALDH1A3 中的一个新的纯合变异。随后,我们在专门筛选 ALDH1A3 突变的 10 名小眼症先证者中的 2 名中鉴定出另一个纯合变异。有趣的是,原始家族的另一个分支被发现与一种新的纯合 SMOC1 变异分离出无眼症/并指畸形。我们的数据支持 ALDH1A3 作为小眼症疾病基因的最近且独立鉴定。即使对于极其罕见的表型,在近亲家族中也应考虑基因座异质性。
Clin Genet. 2013-5-27
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