TBX3-相关疾病。

IF 1.7 4区 生物学 Q3 GENETICS & HEREDITY
Ziv Halperin, Karin Weiss
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引用次数: 0

摘要

TBX3的杂合致病变异可引起尺乳综合征(UMS)。典型的表型特征是上肢缺陷、大汗腺/乳腺发育不全、性腺功能减退和各种中线缺陷。然而,临床谱是高度可变的,一些个体可能表现为轻度或非典型的表现,没有四肢或乳房受累。最近的研究发现,在男性和女性中,垂体发育不全的发生率很高,促性腺激素和生长激素水平下降,在某些情况下,这是一个孤立的发现。我们描述了tbx3相关疾病的主要临床特征和分子基础,并对患者的治疗和管理提出建议。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
TBX3- Related Disorder.

Heterozygous pathogenic variants in TBX3 cause Ulnar-Mammary syndrome (UMS). The phenotype is classically characterized by upper limb defects, apocrine/mammary gland hypoplasia, hypogonadism, and various midline defects. However, the clinical spectrum is highly variable, and some individuals may present with a mild or atypical presentation without limb or mammary involvement. More recent studies identified a high rate of pituitary hypoplasia with decreased levels of gonadotropins and growth hormone in both males and females, in some cases as an isolated finding. We describe the main clinical features and molecular basis of the TBX3-related disorder and propose recommendations for the treatment and management of patients.

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来源期刊
CiteScore
3.50
自引率
5.00%
发文量
432
审稿时长
2-4 weeks
期刊介绍: The American Journal of Medical Genetics - Part A (AJMG) gives you continuous coverage of all biological and medical aspects of genetic disorders and birth defects, as well as in-depth documentation of phenotype analysis within the current context of genotype/phenotype correlations. In addition to Part A , AJMG also publishes two other parts: Part B: Neuropsychiatric Genetics , covering experimental and clinical investigations of the genetic mechanisms underlying neurologic and psychiatric disorders. Part C: Seminars in Medical Genetics , guest-edited collections of thematic reviews of topical interest to the readership of AJMG .
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