Center for Medical Genetics, Antwerp University Hospital/University of Antwerp, Antwerp, Belgium.
Department of Human Genetics, Radboud University Medical Center, Nijmegen, The Netherlands.
Adv Exp Med Biol. 2021;1348:265-272. doi: 10.1007/978-3-030-80614-9_12.
Meester-Loeys syndrome is an X-linked form of syndromic thoracic aortic aneurysm, characterized by the involvement of multiple organ systems. More specifically, the cardiovascular, skeletal, craniofacial, cutaneous and neurological systems are affected. Clear clinical overlap with Marfan syndrome and Loeys-Dietz syndrome is observed. Aortic dissections occur typically at young ages and are most often observed in males. Meester-Loeys syndrome is caused by loss-of-function mutations in BGN, encoding the small leucine-rich proteoglycan biglycan. Although functional consequences of these mutations remain largely elusive, increased TGF-β signaling has been observed. Novel insights will provide opportunities for preventive therapeutic interventions.
马方综合征和李-法美尼综合征的临床表现有明显重叠。Meester-Loeys 综合征是一种 X 连锁的综合征性胸主动脉瘤,其特征为多器官系统受累。确切地说,心血管系统、骨骼系统、颅面、皮肤和神经系统均受影响。主动脉夹层通常发生于年轻时,且多见于男性。Meester-Loeys 综合征是由编码小富含亮氨酸的蛋白聚糖 biglycan 的 BGN 基因突变引起的。尽管这些突变的功能后果仍难以捉摸,但已观察到 TGF-β信号的增强。新的认识将为预防性治疗干预提供机会。