INSR基因突变引起的家族性Rabson-Mendenhall综合征。

IF 3.1 2区 医学 Q1 ENDOCRINOLOGY & METABOLISM
Xuewen Yuan, Ziyang Zhu, Chao Liang
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引用次数: 0

摘要

背景:Rabson-Mendenhall综合征(RMS)是一种罕见的常染色体隐性遗传病,由胰岛素受体(INSR)基因突变引起,导致严重的胰岛素抵抗。RMS的临床表现包括多毛和黑棘皮病。病例介绍:一名3岁男性患者,颈部皮肤暗沉,无明显诱因,未出现多尿、烦渴症状。他和他的姐姐都表现出多毛症和黑棘皮症的症状。实验室调查显示胰岛素和c肽水平明显升高。基因检测发现了INSR基因的两个突变:第20外显子的c.3614C>T和第21外显子的c.3670G>A,后者是以前未在RMS中报道的新突变。他的妹妹也表现出类似的临床特征,并携带相同的突变。结果,两个兄弟姐妹都被诊断为RMS。结论:遗传自父亲的21外显子c.3670G>A突变可能通过破坏酪氨酸激酶活性而损害胰岛素受体功能,从而参与了谱系性RMS的发病机制。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Genealogical Rabson-Mendenhall syndrome caused by INSR gene mutation.

Rabson-Mendenhall syndrome (RMS) is a rare autosomal recessive disorder caused by mutations in the insulin receptor gene (INSR), leading to severe insulin resistance. Clinical manifestations of RMS include hypertrichosis and acanthosis nigricans. A 3-yr-old male patient presented with darkened skin on the neck, without any apparent precipitating factors, and did not exhibit symptoms of polyuria or polydipsia. Both the patient and his older sister displayed signs of hypertrichosis and acanthosis nigricans. Laboratory investigations revealed significantly elevated levels of insulin and C-peptide. Genetic testing identified two mutations in the INSR gene: c.3614C>T in exon 20 and c.3670G>A in exon 21, with the latter being a novel mutation previously unreported in RMS. His sister also exhibited similar clinical features and harbored the same mutations. Consequently, both siblings were diagnosed with RMS. The novel mutation c.3670G>A in exon 21, inherited from the father, is likely to impair insulin receptor function by disrupting tyrosine kinase activity, thereby contributing to the pathogenesis of genealogical RMS.

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来源期刊
CiteScore
9.80
自引率
0.00%
发文量
98
审稿时长
1 months
期刊介绍: The American Journal of Physiology-Endocrinology and Metabolism publishes original, mechanistic studies on the physiology of endocrine and metabolic systems. Physiological, cellular, and molecular studies in whole animals or humans will be considered. Specific themes include, but are not limited to, mechanisms of hormone and growth factor action; hormonal and nutritional regulation of metabolism, inflammation, microbiome and energy balance; integrative organ cross talk; paracrine and autocrine control of endocrine cells; function and activation of hormone receptors; endocrine or metabolic control of channels, transporters, and membrane function; temporal analysis of hormone secretion and metabolism; and mathematical/kinetic modeling of metabolism. Novel molecular, immunological, or biophysical studies of hormone action are also welcome.
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