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伴有新型BEST1突变的常染色体显性玻璃体视网膜脉络膜病变及已报道突变的综述

Autosomal Dominant Vitreoretinochoroidopathy With a Novel BEST1 Mutation and a Review of Reported Mutations.

作者信息

Komro Jack, Skender Sarah, Ross Bing X, Lin Xihui

机构信息

Ophthalmology, Ascension Macomb-Oakland Hospital/Ascension Eye Institute, Warren, USA.

Ophthalmology, Kresge Eye Institute/Wayne State University School of Medicine, Detroit, USA.

出版信息

Cureus. 2022 Dec 27;14(12):e32990. doi: 10.7759/cureus.32990. eCollection 2022 Dec.

DOI:10.7759/cureus.32990
PMID:36712704
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9878615/
Abstract

Here we describe a patient with atypical presentation of autosomal dominant vitreoretinochoroidopathy (ADVIRC) with a novel missense mutation in BEST1 gene and briefly review reported ADVIRC-associated genetic mutations. The patient is a 71-year-old African American female who presented with progressively worsening blurry vision bilaterally over the course of 40 years, with significant deterioration in both peripheral and central vision in the past five years. Her anterior segment exam was unremarkable. Fundoscopic examination showed confluent, demarcated areas of pigmentary chorioretinal atrophy in the mid-periphery of the retina with sparing of the macula in both eyes. Optical coherence tomography (OCT) of the lesions revealed flattening of the fovea with an elevation of the inner retinal structures and outer plexiform layer, and peripheral retinal thinning and loss of retinal structures with choroid hyperreflectivity, consistent with peripheral chorioretinal atrophy. Genetic testing identified a heterozygous c.830C>T, p.(T277M) mutation located on exon 7 of the BEST1 gene. This patient represents an atypical presentation of ADVIRC with more posterior involvement, and this case is associated with a novel missense mutation in the BEST1 gene.

摘要

在此,我们描述了一名患有常染色体显性遗传性玻璃体视网膜脉络膜病变(ADVIRC)非典型表现的患者,其BEST1基因存在一种新的错义突变,并简要回顾已报道的与ADVIRC相关的基因突变。该患者是一名71岁的非裔美国女性,在40年的病程中双侧视力逐渐模糊加重,在过去五年中外周和中心视力均显著下降。她的眼前节检查无异常。眼底检查显示双眼视网膜中周部有融合的、边界清晰的色素性脉络膜视网膜萎缩区域,黄斑未受累。病变的光学相干断层扫描(OCT)显示中央凹变平,视网膜内层结构和外丛状层抬高,周边视网膜变薄且视网膜结构丧失,脉络膜高反射,符合周边脉络膜视网膜萎缩。基因检测确定在BEST1基因第7外显子上存在一个杂合的c.830C>T,p.(T277M)突变。该患者代表了ADVIRC更靠后的非典型表现,且此病例与BEST1基因的一种新的错义突变相关。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/89bf/9878615/a76dbd3a718b/cureus-0014-00000032990-i02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/89bf/9878615/3e2d671664f6/cureus-0014-00000032990-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/89bf/9878615/a76dbd3a718b/cureus-0014-00000032990-i02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/89bf/9878615/3e2d671664f6/cureus-0014-00000032990-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/89bf/9878615/a76dbd3a718b/cureus-0014-00000032990-i02.jpg

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来自一个常染色体显性遗传性玻璃体视网膜脉络膜病变(ADVIRC)家族的诱导多能干细胞衍生视网膜色素上皮细胞中BEST1的定位错误。
Sci Rep. 2016 Sep 22;6:33792. doi: 10.1038/srep33792.
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Long-Term Macular Changes in the First Proband of Autosomal Dominant Vitreoretinochoroidopathy (ADVIRC) Due to a Newly Identified Mutation in BEST1.由于BEST1基因新发现的突变导致的常染色体显性遗传性玻璃体视网膜脉络膜病变(ADVIRC)首例先证者的长期黄斑变化
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ADVIRC is caused by distinct mutations in BEST1 that alter pre-mRNA splicing.常染色体显性遗传性黄斑营养不良(ADVIRC)由 BEST1 基因的不同突变引起,这些突变会改变前体信使核糖核酸(pre-mRNA)的剪接。
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Bestrophin, the product of the Best vitelliform macular dystrophy gene (VMD2), localizes to the basolateral plasma membrane of the retinal pigment epithelium.贝斯特蛋白,即贝斯特卵黄样黄斑营养不良基因(VMD2)的产物,定位于视网膜色素上皮细胞的基底外侧质膜。
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