Gilchrist D, Schwarze U, Shields K, MacLaren L, Bridge P J, Byers P H
Department of Medicine, University of Alberta, Edmonton, Canada.
Am J Med Genet. 1999 Feb 12;82(4):305-11.
Ehlers-Danlos syndrome (EDS) type IV is an autosomal dominant connective tissue disorder. Early morbidity and mortality results from rupture of vessels and internal organs. A large kindred with EDS type IV was studied clinically, and the biochemical defects and underlying mutation in the COL3A1 gene that encodes the chains of type III procollagen were identified. A G-->A transition results in a single amino acid substitution, G571S, in the triple helical domain of the products of one COL3A1 allele. Although the clinical findings seen on examination are characteristic of EDS type IV, longevity is longer than that seen in many families and there is less pregnancy-associated morbidity or mortality than in some families. This suggests that some clinical aspects of EDS type IV may be related to the nature of the mutation and its effect on the behavior of the protein.
IV型埃勒斯-当洛综合征(EDS)是一种常染色体显性遗传性结缔组织疾病。早期发病和死亡是由血管和内脏破裂所致。对一个患有IV型EDS的大家族进行了临床研究,并确定了编码III型前胶原链的COL3A1基因中的生化缺陷和潜在突变。一个COL3A1等位基因产物的三螺旋结构域中发生了一个G→A转换,导致单个氨基酸替代,即G571S。尽管检查所见的临床特征是IV型EDS的特征,但寿命比许多家族中所见的更长,且与妊娠相关的发病率或死亡率比一些家族中更低。这表明IV型EDS的某些临床方面可能与突变的性质及其对蛋白质行为的影响有关。