Lamberg S I, Dorfman A
J Clin Invest. 1973 Oct;52(10):2428-33. doi: 10.1172/JCI107433.
Increased amounts of hyaluronic acid accumulate in fibroblasts cultured from patients with Marfan's disease, an autosomal dominant disorder. In the recessive Hurler's disease, the storage of glycosaminoglycan (GAG) is due to impaired degradation. This study examines the kinetics of GAG accumulation in Marfan's disease in order to determine whether the mechanism of accumulation differs from that in Hurler's disease.Marfan-derived fibroblasts incorporated [(14)C]acetate or [(14)C]glucosamine into GAG to a level 4-6 times greater than control fibroblasts. Sugar analyses, electrophoretic mobility, and enzyme susceptibility studies showed that the isolated material was hyaluronic acid. There were no differences in activity of a variety of glycosidases between Marfan and control fibroblasts, nor were there differences in the ability to degrade prelabeled hyaluronate by cell-free extracts. Finally, chase experiments showed parallel rates of loss of labeled GAG from control fibroblasts and fibroblasts from Marfan patients. It appears that hyaluronic acid was accumulating in greater amounts in the fibroblasts from patients with Marfan's disease because of a greater rate of synthesis as opposed to a decreased rate of breakdown.
在从患有马凡氏综合征(一种常染色体显性疾病)的患者体内培养的成纤维细胞中,透明质酸的含量会增加。在隐性的Hurler病中,糖胺聚糖(GAG)的蓄积是由于降解受损所致。本研究检测了马凡氏综合征中GAG蓄积的动力学,以确定其蓄积机制是否与Hurler病不同。源自马凡氏综合征患者的成纤维细胞将[(14)C]乙酸盐或[(14)C]葡糖胺掺入GAG的水平比对照成纤维细胞高4至6倍。糖类分析、电泳迁移率和酶敏感性研究表明,分离出的物质为透明质酸。马凡氏综合征患者的成纤维细胞与对照成纤维细胞之间,多种糖苷酶的活性没有差异,无细胞提取物降解预先标记的透明质酸盐的能力也没有差异。最后,追踪实验表明,对照成纤维细胞和马凡氏综合征患者的成纤维细胞中标记GAG的丢失速率是平行的。看来,马凡氏综合征患者的成纤维细胞中透明质酸蓄积量更多是因为合成速率更高,而非分解速率降低。