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遗传性鱼鳞病作为罕见皮肤病的范例:基因组医学、发病机制与治疗

Inherited ichthyosis as a paradigm of rare skin disorders: Genomic medicine, pathogenesis, and management.

作者信息

Park Jason S, Saeidian Amir Hossein, Youssefian Leila, Kondratuk Katherine E, Pride Howard B, Vahidnezhad Hassan, Uitto Jouni

机构信息

Department of Dermatology and Cutaneous Biology, Thomas Jefferson University, Philadelphia, Pennsylvania; Geisinger Commonwealth School of Medicine, Scranton, Pennsylvania; Jefferson Institute of Molecular Medicine, Thomas Jefferson University, Philadelphia, Pennsylvania.

Department of Dermatology and Cutaneous Biology, Thomas Jefferson University, Philadelphia, Pennsylvania; Jefferson Institute of Molecular Medicine, Thomas Jefferson University, Philadelphia, Pennsylvania.

出版信息

J Am Acad Dermatol. 2023 Dec;89(6):1215-1226. doi: 10.1016/j.jaad.2022.08.012. Epub 2022 Aug 10.

Abstract

Great advances have been made in the field of heritable skin disorders using next-generation sequencing (NGS) technologies (ie, whole-genome sequencing, whole-exome sequencing, whole-transcriptome sequencing, and disease-targeted multigene panels). When NGS first became available, the cost and lack of access to these technologies were limiting factors; however, with decreasing sequencing costs and the expanding knowledge base of genetic skin diseases, fundamental awareness of NGS has become prudent. The heritable ichthyoses comprise a genotypically and phenotypically heterogeneous group of monogenic keratinization disorders characterized by persistent scaling, with at least 55 distinct genes currently implicated in causing nonsyndromic and syndromic forms of the disease. By providing a simplified overview of available NGS techniques and applying them in the context of ichthyosis, one of the most common genodermatoses, we hope to encourage dermatologists to offer, when appropriate, genetic testing earlier in patients with unsolved presentations. With the aid of NGS, dermatologists can provide diagnostic certainty in cases of suspected genodermatoses and offer potentially life-changing genome-guided and targeted therapies as they become available.

摘要

利用下一代测序(NGS)技术(即全基因组测序、全外显子组测序、全转录组测序和疾病靶向多基因检测板),遗传性皮肤病领域已取得了巨大进展。当NGS首次出现时,这些技术的成本和难以获取是限制因素;然而,随着测序成本的降低以及遗传性皮肤病知识库的不断扩大,对NGS的基本认识已变得十分必要。遗传性鱼鳞病是一组单基因角化障碍,在基因型和表型上具有异质性,其特征为持续性鳞屑,目前至少有55个不同基因与非综合征型和综合征型鱼鳞病的发病有关。通过简要概述现有的NGS技术,并将其应用于最常见的遗传性皮肤病之一鱼鳞病的背景中,我们希望鼓励皮肤科医生在适当的时候,对未明确诊断的患者尽早进行基因检测。借助NGS,皮肤科医生可以在疑似遗传性皮肤病的病例中提供明确诊断,并在有可用的潜在改变生命的基因组导向和靶向治疗方法时提供这些治疗。

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