蓝缅因绵羊结性大疱性表皮松解症的纯合子LAMB3移码变异。

IF 2 3区 生物学 Q3 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
Anna Letko, Liesbeth Harkema, Karianne Peterson, Reinie Dijkman, Cord Drögemüller
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引用次数: 0

摘要

大疱性表皮松解症(EB)是一组以皮肤脆弱和起泡为特征的遗传性皮肤病。在这里,四只蓝缅因羔羊,由一只公羊所生,很早就被诊断出患有EB。由于临床症状的严重性,羔羊不得不被安乐死。受影响的羔羊表现为马蹄脱落,头部、口腔、关节上方皮肤和瘤胃柱出现多处溃疡。组织病理学表现为突然的表皮下裂,基底膜正上方表皮脱离,溃疡符合交界性EB (JEB)。2例进行了全基因组测序(WGS)以确定遗传原因。基因组分析采用常染色体隐性遗传假设,确定了最可能致病的lam3基因(NC_056065.1:g.73166198delG)的纯合1 bp缺失。人类和狗的JEB的隐性形式是由LAMB3基因的变异引起的,该基因编码层粘连蛋白332的β3亚基,层粘连蛋白332是表皮基膜的关键成分。据推测,羊移码变体引入了一个过早终止密码子,并破坏了外显子20的供体剪接位点。该变异等位基因在两个测序病例中均为纯合子,在三个未受影响的近亲属中为杂合子,在1075只其他品种的无亲缘关系对照绵羊中不存在。这项研究强调了遗传调查在兽医诊断中的重要性,并代表了绵羊中与lamb3相关的隐性EB的首次报道。这些发现使基因检测能够为育种策略提供信息,并为人类与lamb3相关的JEB提供第二个自发的大型动物模型。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
A homozygous LAMB3 frameshift variant in junctional epidermolysis bullosa-affected Bleu du Maine sheep.

Epidermolysis bullosa (EB) is a group of inherited skin disorders characterized by skin fragility and blistering. Here, four Bleu du Maine lambs, sired by one ram, were diagnosed with EB very early in life. Due to the severity of the clinical signs, the lambs had to be euthanized. The affected lambs exhibited hoof sloughing and multiple ulcerations on the head, oral cavity, skin over the joints, and the ruminal pillars. Histopathology showed abrupt subepidermal clefts, epidermal detachment directly above the basal membrane, and ulcerations consistent with junctional EB (JEB). Two cases underwent whole-genome sequencing (WGS) to identify the genetic cause. Genomic analyses with the hypothesis of autosomal recessive inheritance identified the most likely pathogenic homozygous 1-bp deletion in the LAMB3 gene (NC_056065.1:g.73166198delG). Recessive forms of JEB in humans and dogs are caused by variants in LAMB3 gene, which encodes β3 subunit of laminin 332, a critical component of the epidermal basal membrane. The ovine frameshift variant putatively introduces a premature stop codon and disrupts the donor splice site of exon 20. The variant allele was homozygous in both sequenced cases and heterozygous in three unaffected close relatives and was absent in 1075 unrelated control sheep of various other breeds. This study highlights the importance of genetic investigation in veterinary diagnostics of and represents the first report of a LAMB3-related recessive EB in sheep. The findings enable genetic testing to inform breeding strategies and provide a second spontaneous large animal model for LAMB3-related JEB in humans.

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来源期刊
Journal of Applied Genetics
Journal of Applied Genetics 生物-生物工程与应用微生物
CiteScore
4.30
自引率
4.20%
发文量
62
审稿时长
6-12 weeks
期刊介绍: The Journal of Applied Genetics is an international journal on genetics and genomics. It publishes peer-reviewed original papers, short communications (including case reports) and review articles focused on the research of applicative aspects of plant, human, animal and microbial genetics and genomics.
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