Suppr超能文献

视网膜色素变性自发性犬模型中的一个假定沉默变体。

A putative silencer variant in a spontaneous canine model of retinitis pigmentosa.

机构信息

Department of Veterinary Biosciences, University of Helsinki, Helsinki, Finland.

Department of Medical and Clinical Genetics, University of Helsinki, Helsinki, Finland.

出版信息

PLoS Genet. 2020 Mar 9;16(3):e1008659. doi: 10.1371/journal.pgen.1008659. eCollection 2020 Mar.

Abstract

Retinitis pigmentosa (RP) is the leading cause of blindness with nearly two million people affected worldwide. Many genes have been implicated in RP, yet in 30-80% of the RP patients the genetic cause remains unknown. A similar phenotype, progressive retinal atrophy (PRA), affects many dog breeds including the Miniature Schnauzer. We performed clinical, genetic and functional experiments to identify the genetic cause of PRA in the breed. The age of onset and pattern of disease progression suggested that at least two forms of PRA, types 1 and 2 respectively, affect the breed, which was confirmed by genome-wide association study that implicated two distinct genomic loci in chromosomes 15 and X, respectively. Whole-genome sequencing revealed a fully segregating recessive regulatory variant in type 1 PRA. The associated variant has a very recent origin based on haplotype analysis and lies within a regulatory site with the predicted binding site of HAND1::TCF3 transcription factor complex. Luciferase assays suggested that mutated regulatory sequence increases expression. Case-control retinal expression comparison of six best HAND1::TCF3 target genes were analyzed with quantitative reverse-transcriptase PCR assay and indicated overexpression of EDN2 and COL9A2 in the affected retina. Defects in both EDN2 and COL9A2 have been previously associated with retinal degeneration. In summary, our study describes two genetically different forms of PRA and identifies a fully penetrant variant in type 1 form with a possible regulatory effect. This would be among the first reports of a regulatory variant in retinal degeneration in any species, and establishes a new spontaneous dog model to improve our understanding of retinal biology and gene regulation while the affected breed will benefit from a reliable genetic testing.

摘要

色素性视网膜炎(RP)是导致失明的主要原因,全球有近 200 万人受到影响。许多基因与 RP 有关,但在 30-80%的 RP 患者中,遗传原因仍不清楚。一种类似的表型,进行性视网膜萎缩(PRA),影响许多犬种,包括迷你雪纳瑞。我们进行了临床、遗传和功能实验,以确定该品种 PRA 的遗传原因。发病年龄和疾病进展模式表明,至少有两种形式的 PRA,分别为 1 型和 2 型,分别影响该品种,这一点通过全基因组关联研究得到了证实,该研究分别在 15 号和 X 染色体上暗示了两个不同的基因组位置。全基因组测序揭示了 1 型 PRA 中一种完全分离的隐性调节变体。基于单倍型分析,相关变体具有非常近期的起源,位于 HAND1::TCF3 转录因子复合物的预测结合位点的调节位点内。荧光素酶测定表明,突变的调节序列增加了表达。通过定量逆转录酶 PCR 检测分析了六个最佳 HAND1::TCF3 靶基因的病例对照视网膜表达比较,结果表明受影响视网膜中 EDN2 和 COL9A2 的表达过度。EDN2 和 COL9A2 的缺陷以前与视网膜变性有关。总之,我们的研究描述了两种遗传上不同形式的 PRA,并确定了 1 型中一种完全穿透的变体,可能具有调节作用。这将是在任何物种的视网膜变性中首次报道调节变体之一,并建立了一个新的自发犬模型,以提高我们对视网膜生物学和基因调控的理解,同时受影响的品种将受益于可靠的基因测试。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/273b/7082071/e58f048d18fe/pgen.1008659.g002.jpg

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

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验