在一个由 10 个家族组成的队列中,HR 中的新型致病变体是富贵病伴丘疹性病变的基础。

Kifayat Ullah, Sohail Ahmed, Nicole Cesarato, Xing Xiong, Maria Taj, Naushaba Manan, Maria Wehner, Muhammad Javed Khan, Hammal Khan, Sabba Mehmood, Muhammad Sharif Hasni, Dariusz Michna, Rehmana Waris, Henning Hamm, Regina C Betz, Wasim Ahmad, Imran Ullah
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引用次数: 0

摘要

毛囊角化性丘疹病(APL)是一种毛发异常,其特征是头皮和身体其他部位的毛发脱落。在少数病例中,脱发会伴随身体出现角化性丘疹。该病为常染色体隐性遗传。HR(无毛)基因的序列变异是导致这种毛发异常的原因。在此,我们介绍了具有 APL 临床表现的九个近亲家族和一个非近亲家族。为了确定致病变体,我们进行了全外显子组测序和/或直接桑格测序。研究发现了 7 个新的致病变体:c.794del;p. (Pro265Argfs*98)、c.2921-2936del;p. (Tyr974Leufs*16)、c.2889C>A;p. (Cys963*)、c.2689C>T;p. (Gln897*)、c.3186_3187dup;p.(Gln1063Profs*43)、c.560dup;p.(Tyr188Ilefs*131)、c.2203+5G>C、c.2776+5G>A,以及之前报道的这些家族中 HR 变异 c.1837C>T;p.(Arg613*)。这项研究不仅扩大了 HR 基因的突变范围,而且突出了不寻常的表型发现,将有助于为本地人群中出现各种脱发疾病的家族成员提供遗传咨询。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Novel pathogenic variants in HR underlie atrichia with papular lesions in a cohort of 10 families.

Atrichia with papular lesions (APL) is a hair abnormality characterized by loss of hair on the scalp and rest of the body. In a few cases, hair loss is accompanied by the appearance of keratotic papules on the body. It is inherited in an autosomal recessive manner. Sequence variants in the HR (hairless) gene are responsible for this hair abnormality. Here, we present nine consanguineous families and one nonconsanguineous family with clinical manifestations of APL. Whole exome followed by Sanger sequencing and/or direct Sanger sequencing was performed to identify pathogenic variants. The study revealed seven novel pathogenic variants c.794del;p.(Pro265Argfs*98), c.2921-2936del;p.(Tyr974Leufs*16), c.2889C>A;p.(Cys963*), c.2689C>T;p.(Gln897*), c.3186_3187dup;p.(Gln1063Profs*43), c.560dup;p.(Tyr188Ilefs*131), c.2203+5G>C, c.2776+5G>A, and the previously reported variant c.1837C>T;p.(Arg613*) in HR in these families. The study not only expands the mutational spectrum in the HR gene but also highlights the unusual phenotypic findings and will facilitate genetic counseling of families with members showing various types of hair loss disorders in the local population.

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