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COL7A1 基因外显子 87 跳跃导致显性营养不良型大疱性表皮松解症。

Exon 87 skipping of the COL7A1 gene in dominant dystrophic epidermolysis bullosa.

机构信息

Department of Dermatology, Kurume University School of Medicine, Asahimachi, Kurume, Fukuoka, Japan.

出版信息

J Dermatol. 2011 May;38(5):489-92. doi: 10.1111/j.1346-8138.2010.01008.x. Epub 2010 Sep 20.

Abstract

Dystrophic epidermolysis bullosa (DEB) is a rare, inherited, blistering disorder resulting from mutations in the COL7A1 gene, which encodes the anchoring fibrils, type VII collagen. We herein describe a further Japanese girl diagnosed with dominant DEB (DDEB). She had blisters sporadically and erosions healed with mild scarring and milia on the knees and pretibial regions. Severe pruritus was present at this time. Direct nucleotide sequencing of genomic DNA disclosed a heterozygous same splice-site mutation c.6900G>A in the COL7A1, which causes in-frame exon 87 skipping. So far, five different COL7A1 mutations leading to exon 87 skipping have been identified in rare forms of DEB: four DDEB pruriginosa and one pretibial DDEB. Therefore, a recent study suggested that exon 87 skipping in COL7A1 was related to the phenotype of DDEB pruriginosa. When she was 18 years old, however, the blister formation and pruritus markedly decreased. Therefore, her clinical symptoms were consistent to very mild DDEB but not to DDEB pruriginosa. Taken together, in-frame exon 87 skipping through c.6900G>A mutation may account for the mild skin features, rather than DDEB pruriginosa, in the present case.

摘要

营养不良型大疱性表皮松解症(DEB)是一种罕见的遗传性水疱性疾病,由 COL7A1 基因突变引起,该基因编码锚纤维,即 VII 型胶原。本文描述了另一位日本女孩患有显性 DEB(DDEB)。她膝盖和胫前区偶尔出现水疱,糜烂后有轻微瘢痕和粟粒疹。此时伴有严重瘙痒。对基因组 DNA 的直接核苷酸测序显示 COL7A1 中存在杂合相同剪接位点突变 c.6900G>A,导致内含子 87 跳跃。迄今为止,在罕见形式的 DEB 中已经发现了五种不同的 COL7A1 突变导致外显子 87 跳跃:四例瘙痒性 DDEB 和一例胫前 DDEB。因此,最近的一项研究表明,COL7A1 中的外显子 87 跳跃与瘙痒性 DDEB 的表型有关。然而,当她 18 岁时,水疱形成和瘙痒明显减少。因此,她的临床症状与非常轻微的 DDEB 一致,而不是瘙痒性 DDEB。总之,通过 c.6900G>A 突变的框内外显子 87 跳跃可能导致本病例皮肤特征较轻,而不是瘙痒性 DDEB。

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