Narcisi P, Wu Y, Tromp G, Earley J J, Richards A J, Pope F M, Kuivaniemi H
Department of Biochemistry and Molecular Biology, Jefferson Institute of Molecular Medicine, Thomas Jefferson University, Philadelphia, Pennsylvania 19107.
Am J Med Genet. 1993 May 15;46(3):278-83. doi: 10.1002/ajmg.1320460308.
The proposita described here was a 24-year-old woman with an acrogeric form of the Ehlers-Danlos syndrome including a massive dissecting aortic aneurysm. She was found to have a single-base mutation that substituted glutamic acid for glycine at amino acid position 1021 in the triple-helical domain of the type III procollagen. It is the most carboxy-terminal single-base mutation characterized to date in the COL3A1 gene. Analysis of medium and cell layer proteins from proposita's cultured skin fibroblasts showed that the mutant protein was poorly secreted, migrated more slowly on a polyacrylamide gel, and was partially unstable at +25 degrees C to brief digestion with trypsin.
本文所述的先证者是一名24岁女性,患有埃勒斯-当洛综合征的肢端型,包括巨大的主动脉夹层动脉瘤。她被发现有一个单碱基突变,该突变在III型前胶原三螺旋结构域的第1021位氨基酸处用谷氨酸替代了甘氨酸。这是迄今为止在COL3A1基因中鉴定出的最靠近羧基端的单碱基突变。对先证者培养的皮肤成纤维细胞的培养基和细胞层蛋白进行分析表明,突变蛋白分泌不良,在聚丙烯酰胺凝胶上迁移较慢,并且在25摄氏度下对胰蛋白酶的短暂消化部分不稳定。