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NR2F1数据库:112个变异体和84名患者支持完善博世-布恩斯特拉-沙夫视神经萎缩综合征的临床概要。

NR2F1 database: 112 variants and 84 patients support refining the clinical synopsis of Bosch-Boonstra-Schaaf optic atrophy syndrome.

作者信息

Billiet Benjamin, Amati-Bonneau Patrizia, Desquiret-Dumas Valérie, Guehlouz Khadidja, Milea Dan, Gohier Philippe, Lenaers Guy, Mirebeau-Prunier Delphine, den Dunnen Johan T, Reynier Pascal, Ferré Marc

机构信息

Département d'Ophtalmologie, Centre Hospitalier Universitaire d'Angers, Angers, France.

Unité MITOVASC, Équipe Mitolab, SFR ICAT, INSERM, CNRS, Université d'Angers, Angers, France.

出版信息

Hum Mutat. 2022 Feb;43(2):128-142. doi: 10.1002/humu.24305. Epub 2021 Dec 9.

Abstract

Pathogenic variants of the nuclear receptor subfamily 2 group F member 1 gene (NR2F1) are responsible for Bosch-Boonstra-Schaaf optic atrophy syndrome (BBSOAS), an autosomal dominant disorder characterized by optic atrophy associated with developmental delay and intellectual disability, but with a clinical presentation which appears to be multifaceted. We created the first public locus-specific database dedicated to NR2F1. All variants and clinical cases reported in the literature, as well as new unpublished cases, were integrated into the database using standard nomenclature to describe both molecular and phenotypic anomalies. We subsequently pursued a comprehensive approach based on computed representation and analysis suggesting a refinement of the BBSOAS clinical description with respect to neurological features and the inclusion of additional signs of hypotonia and feeding difficulties. This database is fully accessible for both clinician and molecular biologists and should prove useful in further refining the clinical synopsis of NR2F1 as new data is recorded.

摘要

核受体亚家族2组F成员1基因(NR2F1)的致病变异是博施-布恩斯特拉-沙夫视神经萎缩综合征(BBSOAS)的病因,这是一种常染色体显性疾病,其特征为视神经萎缩,并伴有发育迟缓及智力残疾,但其临床表现似乎具有多面性。我们创建了首个专门针对NR2F1的公共位点特异性数据库。文献中报道的所有变异和临床病例,以及新的未发表病例,均使用标准命名法整合到数据库中,以描述分子和表型异常。随后,我们采用了基于计算机表征和分析的综合方法,建议在神经学特征方面对BBSOAS临床描述进行细化,并纳入肌张力减退和喂养困难等更多体征。临床医生和分子生物学家均可完全访问该数据库,随着新数据的记录,该数据库应会有助于进一步完善NR2F1的临床概要。

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