Facultad de Medicina, Universidad Veracruzana, Ciudad Mendoza, Veracruz, México.
Laboratorio de Modelado Molecular y Bioinformática, Facultad de Ciencias Químico-Biológicas, Universidad Autónoma de Sinaloa, Culiacán, Sinaloa, México.
Int J Dermatol. 2020 Aug;59(8):969-977. doi: 10.1111/ijd.14952. Epub 2020 May 21.
Autosomal recessive congenital ichthyoses (ARCI) are inherited disorders produced by mutations in essential genes for the skin function. A low prevalence of this disease has been resported worldwide; however, in a recent study, we identified a large cluster of ARCI families who resided in the High Mountains Region from the Veracruz State, Mexico. Thus, we aimed to identify the causative mutation of ARCI and describe the high prevalence of this disease in this region.
We selected seven familiar trios and performed whole-exome sequencing to identify the mutation associated with ARCI. To validate the identified mutation, we performed Sanger sequencing in 62 patients, 30 unaffected relatives, and 100 healthy volunteers. Finally, we performed molecular modeling to investigate the possible functional consequences produced by the mutation.
We identified a novel homozygous mutation (c.1054C>G [p.Pro352Ala]) in the exon 7 of the TGM1 gene in all the patients. We calculated a prevalence rate of ARCI of 74:100,000 (1:1,348) in the studied communities. Molecular modeling revealed that the mutation leads to a nonconservative amino acid substitution, which is very probably damaging to the protein structure/function.
We report a novel mutation in the TGM1 gene in 62 Mexican patients. The unusually high frequency of this mutation suggests a founder effect; however, further haplotype analysis is necessary to corroborate this hypothesis. In this respect, to our knowledge, the prevalence of ARCI found in the studied communities is the highest observed worldwide.
常染色体隐性先天性鱼鳞病(ARCI)是由皮肤功能必需基因的突变引起的遗传性疾病。这种疾病的发病率很低,在全球范围内都有报道;然而,在最近的一项研究中,我们在墨西哥韦拉克鲁斯州的高山地区发现了一个大型 ARCI 家族聚集。因此,我们旨在确定 ARCI 的致病突变,并描述该地区这种疾病的高发病率。
我们选择了七个有亲缘关系的三胞胎家庭,并进行全外显子组测序以鉴定与 ARCI 相关的突变。为了验证鉴定出的突变,我们对 62 名患者、30 名未受影响的亲属和 100 名健康志愿者进行了 Sanger 测序。最后,我们进行了分子建模,以研究突变可能产生的功能后果。
我们在所有患者的 TGM1 基因外显子 7 中发现了一个新的纯合突变(c.1054C>G [p.Pro352Ala])。我们计算出研究社区中 ARCI 的患病率为 74:100,000(1:1,348)。分子建模显示,该突变导致非保守氨基酸取代,很可能对蛋白质结构/功能造成损害。
我们在 62 名墨西哥患者中报告了 TGM1 基因的一个新突变。该突变的异常高频提示存在一个奠基者效应;然而,进一步的单倍型分析是必要的,以证实这一假设。在这方面,据我们所知,在所研究的社区中发现的 ARCI 患病率是全球最高的。