鱼鳞病患者中的有丝分裂重组导致 KRT10 显性突变的回复。

Mitotic recombination in patients with ichthyosis causes reversion of dominant mutations in KRT10.

机构信息

Department of Dermatology, Yale University School of Medicine, New Haven, CT 06510, USA.

出版信息

Science. 2010 Oct 1;330(6000):94-7. doi: 10.1126/science.1192280. Epub 2010 Aug 26.

Abstract

Somatic loss of wild-type alleles can produce disease traits such as neoplasia. Conversely, somatic loss of disease-causing mutations can revert phenotypes; however, these events are infrequently observed. Here we show that ichthyosis with confetti, a severe, sporadic skin disease in humans, is associated with thousands of revertant clones of normal skin that arise from loss of heterozygosity on chromosome 17q via mitotic recombination. This allowed us to map and identify disease-causing mutations in the gene encoding keratin 10 (KRT10); all result in frameshifts into the same alternative reading frame, producing an arginine-rich C-terminal peptide that redirects keratin 10 from the cytokeratin filament network to the nucleolus. The high frequency of somatic reversion in ichthyosis with confetti suggests that revertant stem cell clones are under strong positive selection and/or that the rate of mitotic recombination is elevated in individuals with this disorder.

摘要

体细胞野生型等位基因的丢失可导致肿瘤等疾病特征。相反,疾病相关突变的体细胞丢失可使表型逆转;然而,这些事件很少观察到。在这里,我们表明,斑驳性鱼鳞病是一种严重的、散发性的人类皮肤疾病,与数千个正常皮肤的回复克隆有关,这些克隆是通过有丝分裂重组从 17q 染色体的杂合性丢失中产生的。这使我们能够定位和鉴定编码角蛋白 10(KRT10)的基因突变;所有突变均导致移码到相同的选择性阅读框,产生富含精氨酸的 C 末端肽,使角蛋白 10 从细胞角蛋白丝网络转移到核仁。斑驳性鱼鳞病中体细胞逆转的高频率表明,回复干细胞克隆受到强烈的正选择,或者患有这种疾病的个体的有丝分裂重组率升高。

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