Francomano C A, Streeten E A, Meyers D A, Pyeritz R E
Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, Maryland.
Am J Med Genet. 1988 Feb;29(2):457-62. doi: 10.1002/ajmg.1320290233.
The Marfan syndrome is an autosomal dominant connective tissue disorder with pleiotropic manifestations affecting skeletal, ocular and cardiovascular systems. Because the fibrillar collagens are major structural components of connective tissue, the hypothesis has long been set forth that the Marfan syndrome is a disorder of fibrillar collagen. We have investigated this hypothesis by performing linkage studies in 12 multiplex families with the Marfan syndrome, using restriction fragment length polymorphisms (RFLP's) associated with 3 genes encoding chains of fibrillar collagens. The data exclude linkage to all 3 candidate genes in 2 families and at least 1 of the candidates is excluded in 6 additional families. Each candidate was excluded in at least 3 families. In no case was strong evidence in favor of linkage of the Marfan syndrome to any of the 3 genes observed. These data speak against the hypothesis that mutations in one or more of these 3 fibrillar collagens cause the classic Marfan syndrome.
马凡综合征是一种常染色体显性遗传性结缔组织疾病,具有多效性表现,影响骨骼、眼部和心血管系统。由于纤维状胶原蛋白是结缔组织的主要结构成分,长期以来一直有人提出假说,认为马凡综合征是一种纤维状胶原蛋白疾病。我们通过对12个患有马凡综合征的多重家庭进行连锁研究来调查这一假说,使用与3个编码纤维状胶原蛋白链的基因相关的限制性片段长度多态性(RFLP)。数据排除了2个家庭与所有3个候选基因的连锁关系,另外6个家庭中至少排除了1个候选基因。每个候选基因在至少3个家庭中被排除。在任何情况下,均未观察到有力证据支持马凡综合征与这3个基因中的任何一个存在连锁关系。这些数据反驳了这一假说,即这3种纤维状胶原蛋白中的一种或多种发生突变会导致典型的马凡综合征。