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进一步描述 KDM5C 致病变异导致的女性表型:19 例新病例及文献复习。

Further delineation of the female phenotype with KDM5C disease causing variants: 19 new individuals and review of the literature.

机构信息

INSERM UMR1231, Equipe Génétique des Anomalies du Développement, Université de Bourgogne, Dijon, France.

Centre de Référence Maladies Génétique à Expression Cutanée, Centre Hospitalier Universitaire Dijon Bourgogne, Dijon, France.

出版信息

Clin Genet. 2020 Jul;98(1):43-55. doi: 10.1111/cge.13755. Epub 2020 May 29.

Abstract

X-linked intellectual disability (XLID) is a genetically heterogeneous condition involving more than 100 genes. To date, 35 pathogenic variants have been reported in the lysine specific demethylase 5C (KDM5C) gene. KDM5C variants are one of the major causes of moderate to severe XLID. Affected males present with short stature, distinctive facial features, behavioral disorders, epilepsy, and spasticity. For most of these variants, related female carriers have been reported, but phenotypic descriptions were poor. Here, we present clinical and molecular features of 19 females carrying 10 novel heterozygous variants affecting KDM5C function, including five probands with de novo variants. Four heterozygous females were asymptomatic. All affected individuals presented with learning disabilities or ID (mostly moderate), and four also had a language impairment mainly affecting expression. Behavioral disturbances were frequent, and endocrine disorders were more frequent in females. In conclusion, our findings provide evidence of the role of KDM5C in ID in females highlighting the increasing implication of XLID genes in females, even in sporadic affected individuals. Disease expression of XLID in females should be taken into consideration for genetic counseling.

摘要

X 连锁智力障碍 (XLID) 是一种遗传异质性疾病,涉及 100 多个基因。迄今为止,赖氨酸特异性去甲基酶 5C (KDM5C) 基因已报道 35 种致病性变异。KDM5C 变异是中度至重度 XLID 的主要原因之一。受影响的男性表现为身材矮小、特征性面部特征、行为障碍、癫痫和痉挛。对于这些变体中的大多数,已经报道了相关的女性携带者,但表型描述较差。在这里,我们介绍了携带 10 种影响 KDM5C 功能的新型杂合变异的 19 名女性的临床和分子特征,包括 5 名先证者携带新生变异。四名杂合子女性无症状。所有受影响的个体均表现为学习障碍或智力障碍(主要为中度),其中 4 人也存在主要影响表达的语言障碍。行为障碍频繁发生,女性内分泌紊乱更为常见。总之,我们的研究结果为 KDM5C 在女性智力障碍中的作用提供了证据,强调了 XLID 基因在女性中的作用日益增加,即使在散发性受影响的个体中也是如此。对于遗传咨询,应考虑女性 XLID 的疾病表现。

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