Department of Molecular & Human Genetics, Baylor College of Medicine, Houston, TX, USA.
Chair and Department of Genetics and Pharmaceutical Microbiology, Poznan University of Medical Sciences, Poznan, Poland.
Eur J Med Genet. 2022 Jun;65(6):104519. doi: 10.1016/j.ejmg.2022.104519. Epub 2022 May 6.
Alveolar capillary dysplasia with misalignment of pulmonary veins (ACDMPV) is a rare lethal lung developmental disorder in neonates due to heterozygous loss-of-function of the mesenchymal transcription factor gene, FOXF1. Interestingly, unlike ACDMPV-causing point mutations in FOXF1 that can be inherited from the mother or father, causative copy-number variant (CNV) deletions arise de novo and almost exclusively on chromosome 16 inherited from the mother (n = 50 vs. n = 3). Here, we describe a fourth case of a de novo paternal CNV deletion encompassing FOXF1, its neighboring long non-coding RNA gene FENDRR, and their distant lung-specific enhancer, identified in a 21-week-old fetus with tetralogy of Fallot, gastrointestinal and genitourinary abnormalities, a single umbilical artery, and patchy histopathological findings of ACDMPV in autopsy lung. We also review the ACDMPV-causative CNV deletions detected prenatally and propose that the majority of paternal deletions manifest with more severe additional non-lung abnormalities.
肺泡毛细血管发育不良伴肺静脉错位(ACDMPV)是一种罕见的新生儿致死性肺发育障碍,由间充质转录因子基因 FOXF1 的杂合功能丧失引起。有趣的是,与 FOXF1 导致 ACDMPV 的点突变不同,这些点突变可以从母亲或父亲遗传,而致病拷贝数变异(CNV)缺失是从头发生的,几乎只发生在从母亲遗传的 16 号染色体上(n=50 对 n=3)。在这里,我们描述了第四例 FOXF1 及其邻近的长非编码 RNA 基因 FENDRR 及其遥远的肺特异性增强子的从头发生的父系 CNV 缺失,该缺失发生在一名 21 周大的法洛四联症胎儿中,该胎儿伴有胃肠道和泌尿生殖系统异常、单脐动脉以及尸检肺中 ACDMPV 的斑片状组织病理学发现。我们还回顾了产前检测到的 ACDMPV 致病 CNV 缺失,并提出大多数父系缺失表现出更严重的额外非肺异常。