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Case Report: A Novel Truncating Variant of NR0B1 Presented With X-Linked Late-Onset Adrenal Hypoplasia Congenita With Hypogonadotropic Hypogonadism

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单位: [1]Huazhong Univ Sci & Technol, Union Hosp, Tongji Med Coll, Dept Cardiol, Wuhan, Peoples R China [2]Huazhong Univ Sci & Technol,Tongji Hosp,Tongji Med Coll,Dept Pulm & Crit Care Med,Wuhan,Peoples R China [3]Natl Minist Hlth Peoples Republ China, Key Lab Resp Dis, Wuhan, Peoples R China [4]Huazhong Univ Sci & Technol, Union Hosp, Tongji Med Coll, Dept Endocrinol, Wuhan, Peoples R China [5]Distinct HealthCare, Dept Internal Med, Wuhan, Peoples R China [6]Huazhong Univ Sci & Technol, Union Hosp, Tongji Med Coll, Dept Nephrol, Wuhan, Peoples R China [7]Natl Clin Res Ctr Resp Dis, Wuhan, Peoples R China
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关键词: adrenal hypoplasia congenita hypogonadotropic hypogonadism DAX1 X-linked recessive

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Nuclear receptor subfamily 0 group B member 1 gene (NR0B1) encodes an orphan nuclear receptor that plays a critical role in the development and regulation of the adrenal gland and hypothalamic-pituitary-gonadal axis. In this study, we report a novel mutation in NR0B1 that led to adult-onset adrenal hypoplasia congenita (AHC) and pubertal development failure in a male adult. Clinical examinations revealed hyponatremia, elevated adrenocorticotropic hormone levels, reduced testosterone and gonadotropin levels, and hyper-responses to gonadotropin-releasing hormone and human chorionic gonadotropin stimulation tests. Whole-exome sequencing and Sanger sequencing were performed to identify the potential causes of AHC. Candidate variants were shortlisted based on the X-linked recessive models. Sequence analyses identified a novel hemizygous variant of c.1034delC in exon 1 of NR0B1 at Xp21.2, resulting in a frameshift mutation and premature stop codon formation. The c.1034delC/p.Pro345Argfs*27 in the NR0B1 gene was detected in the hemizygous state in affected males and in the heterozygous state in healthy female family carriers. These results expand the clinical features of AHC as well as the mutation profile of the causative gene NR0B1. Further studies are needed to elucidate the biological effects of the mutation on the development and function of the adrenal gland and the hypothalamic-pituitary-gonadal axis.

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出版当年[2021]版:
大类 | 3 区 医学
小类 | 3 区 内分泌学与代谢
最新[2025]版:
大类 | 3 区 医学
小类 | 3 区 内分泌学与代谢
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出版当年[2020]版:
Q1 ENDOCRINOLOGY & METABOLISM
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Q1 ENDOCRINOLOGY & METABOLISM

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第一作者单位: [1]Huazhong Univ Sci & Technol, Union Hosp, Tongji Med Coll, Dept Cardiol, Wuhan, Peoples R China
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