Department of Vascular Surgery, Nanjing Drum Tower Hospital, The Affiliated Hospital of Nanjing University Medical School, Nanjing, Jiangsu 210008, P.R. China.
Center for Translational Medicine, Nanjing University Medical School, Nanjing, Jiangsu 210093, P.R. China.
Mol Med Rep. 2017 Nov;16(5):6620-6625. doi: 10.3892/mmr.2017.7410. Epub 2017 Aug 31.
The etiology of thoracic aortic aneurysm and dissection (TAAD) is complex and heterogeneous. Emerging evidence has demonstrated that genetic causes may be a consideration in early‑onset TAAD. Owing to overlapping clinical phenotypes and the genetic heterogeneity of TAAD, it is challenging for clinicians to make a molecular diagnosis of TAAD, particularly in those who present with non‑specific syndromic features. In order to identify the causative mutation in two young patients with acute type B aortic dissection without syndromic features, whole exome sequencing (WES) was performed in the present study. A missense mutation (c.G6953A:p.C2318Y) and a nonsense mutation (c.C4786T:p.R1596X) were identified in the fibrillin 1 gene in patients T287 and T267, respectively. The present study emphasized the necessity of genetic testing for young patients with type B aortic dissection. WES is a timely, robust and inexpensive technique for molecular diagnosis, particularly for TAAD caused by numerous genes. Genetic diagnosis of Marfan syndrome could aid in periodic surveillance, prophylactic surgical measures, and genetic counseling.
胸主动脉瘤和夹层(TAAD)的病因复杂且具有异质性。新出现的证据表明,遗传原因可能是早发性 TAAD 的一个考虑因素。由于 TAAD 的临床表现重叠和遗传异质性,临床医生很难对 TAAD 做出分子诊断,特别是对于那些表现出非特异性综合征特征的患者。为了确定两名无综合征特征的急性 B 型主动脉夹层年轻患者的致病突变,本研究对其进行了全外显子组测序(WES)。在患者 T287 和 T267 中,纤维连接蛋白 1 基因中分别发现了一个错义突变(c.G6953A:p.C2318Y)和一个无义突变(c.C4786T:p.R1596X)。本研究强调了对 B 型主动脉夹层年轻患者进行基因检测的必要性。WES 是一种及时、强大且廉价的分子诊断技术,特别是对于由众多基因引起的 TAAD。马凡综合征的基因诊断有助于定期监测、预防性手术措施和遗传咨询。