Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
Eur J Hum Genet. 2013 Apr;21(4):474-7. doi: 10.1038/ejhg.2012.171. Epub 2012 Sep 19.
Alveolar capillary dysplasia with misalignment of pulmonary veins (ACD/MPV) is a rare developmental lung disorder that is uniformly lethal. Affected infants die within the first few weeks of their life despite aggressive treatment, although a few cases of late manifestation and longer survival have been reported. We have shown previously that mutations and deletions in FOXF1 are a cause of this disorder. Although most of the cases of ACD/MPV are sporadic, there have been infrequent reports of familial cases. We present a family with five out of six children affected with ACD/MPV. DNA analysis identified a missense mutation (c.416G>T; p.Arg139Leu) in the FOXF1 gene that segregated in the three affected siblings tested. The same variant is also present as a de novo mutation in the mother and arose on her paternally derived chromosome 16. The two tested affected siblings share the same chromosome 16 haplotype inherited from their maternal grandfather. Their single healthy sibling has a different chromosome 16 haplotype inherited from the maternal grandmother. The results are consistent with paternal imprinting of FOXF1 in human.
肺静脉异位连接性/肺静脉发育不良(ACD/MPV)是一种罕见的肺发育障碍,具有一致性致死性。尽管进行了积极的治疗,但受影响的婴儿仍会在生命的头几周内死亡,尽管有一些病例表现较晚且存活时间较长。我们之前已经表明,FOXF1 的突变和缺失是导致这种疾病的原因。虽然大多数 ACD/MPV 病例是散发性的,但也有罕见的家族病例报告。我们报告了一个有六个孩子中有五个患有 ACD/MPV 的家庭。DNA 分析确定了 FOXF1 基因中的一个错义突变(c.416G>T;p.Arg139Leu),在三个受影响的兄弟姐妹中进行了测试,该突变与疾病共分离。在母亲和她从父亲那里继承的第 16 号染色体上也存在相同的变体,是一个新生突变。两个受测试的患病兄弟姐妹从他们的外祖父那里继承了相同的 16 号染色体单倍型。他们唯一健康的兄弟姐妹从他们的祖母那里继承了不同的 16 号染色体单倍型。结果与人类 FOXF1 的父系印迹一致。