文献检索文档翻译深度研究
Suppr Zotero 插件Zotero 插件
邀请有礼套餐&价格历史记录

新学期,新优惠

限时优惠:9月1日-9月22日

30天高级会员仅需29元

1天体验卡首发特惠仅需5.99元

了解详情
不再提醒
插件&应用
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
高级版
套餐订阅购买积分包
AI 工具
文献检索文档翻译深度研究
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利:CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2025

基因突变导致小眼症。

Mutations in ALDH1A3 cause microphthalmia.

机构信息

Department of Genetics, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia.

出版信息

Clin Genet. 2013 Aug;84(2):128-31. doi: 10.1111/cge.12184. Epub 2013 May 27.


DOI:10.1111/cge.12184
PMID:23646827
Abstract

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.

摘要

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。

相似文献

[1]
Mutations in ALDH1A3 cause microphthalmia.

Clin Genet. 2013-5-27

[2]
Mutations in ALDH1A3 represent a frequent cause of microphthalmia/anophthalmia in consanguineous families.

Hum Mutat. 2014-8

[3]
A homozygous mutation in a consanguineous family consolidates the role of ALDH1A3 in autosomal recessive microphthalmia.

Clin Genet. 2014-9

[4]
Novel mutations in ALDH1A3 associated with autosomal recessive anophthalmia/microphthalmia, and review of the literature.

BMC Med Genet. 2018-9-10

[5]
Novel splice-site and missense mutations in the ALDH1A3 gene underlying autosomal recessive anophthalmia/microphthalmia.

Br J Ophthalmol. 2014-2-25

[6]
Incomplete penetrance of biallelic ALDH1A3 mutations.

Eur J Med Genet. 2016-4

[7]
ALDH1A3 loss of function causes bilateral anophthalmia/microphthalmia and hypoplasia of the optic nerve and optic chiasm.

Hum Mol Genet. 2013-4-15

[8]
ALDH1A3 mutations cause recessive anophthalmia and microphthalmia.

Am J Hum Genet. 2013-1-9

[9]
A missense mutation in ALDH1A3 causes isolated microphthalmia/anophthalmia in nine individuals from an inbred Muslim kindred.

Eur J Hum Genet. 2013-7-24

[10]
VSX2 mutations in autosomal recessive microphthalmia.

Mol Vis. 2011

引用本文的文献

[1]
Genotypic and phenotypic spectrum of anophthalmia/microphthalmia in families from Khyber Pakhtunkhwa, Pakistan.

J Hum Genet. 2025-8-18

[2]
Clinical and genetic analysis further delineates the phenotypic spectrum of ALDH1A3-related anophthalmia and microphthalmia.

Eur J Hum Genet. 2023-10

[3]
Identification of 4 novel human ocular coloboma genes ANK3, BMPR1B, PDGFRA, and CDH4 through evolutionary conserved vertebrate gene analysis.

Genet Med. 2022-5

[4]
Role of carotenoids and retinoids during heart development.

Biochim Biophys Acta Mol Cell Biol Lipids. 2020-1-22

[5]
Genetic architecture of retinoic-acid signaling-associated ocular developmental defects.

Hum Genet. 2019-7-29

[6]
Genetics of anophthalmia and microphthalmia. Part 1: Non-syndromic anophthalmia/microphthalmia.

Hum Genet. 2019-2-14

[7]
Genetics of anophthalmia and microphthalmia. Part 2: Syndromes associated with anophthalmia-microphthalmia.

Hum Genet. 2018-10-30

[8]
Novel mutations in ALDH1A3 associated with autosomal recessive anophthalmia/microphthalmia, and review of the literature.

BMC Med Genet. 2018-9-10

[9]
Retinoic Acid Maintains Function of Neural Crest-Derived Ocular and Craniofacial Structures in Adult Zebrafish.

Invest Ophthalmol Vis Sci. 2018-4-1

[10]
Novel compound heterozygous mutations of ALDH1A3 contribute to anophthalmia in a non-consanguineous Chinese family.

Genet Mol Biol. 2017

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

推荐工具

医学文档翻译智能文献检索