Maddox B K, Keene D R, Sakai L Y, Charbonneau N L, Morris N P, Ridgway C C, Boswell B A, Sussman M D, Horton W A, Bächinger H P, Hecht J T
Research Department, Shriners Hospital for Children, Portland, Oregon 97201, USA.
J Biol Chem. 1997 Dec 5;272(49):30993-7. doi: 10.1074/jbc.272.49.30993.
We have identified a novel missense mutation in a pseudoachondroplasia (PSACH) patient in one of the type III repeats of cartilage oligomeric matrix protein (COMP). Enlarged lamellar rough endoplasmic reticulum vesicles were shown to contain accumulated COMP along with type IX collagen, a cartilage-specific component. COMP was secreted and assembled normally into the extracellular matrix of tendon, demonstrating that the accumulation of COMP in chondrocytes was a cell-specific phenomenon. We believe that the intracellular storage of COMP causes a nonspecific aggregation of cartilage-specific molecules and results in a cartilage matrix deficient in required structural components leading to impaired cartilage growth and maintenance. These data support a common pathogenetic mechanism behind two clinically related chondrodysplasias, PSACH and multiple epiphyseal dysplasia.
我们在软骨寡聚基质蛋白(COMP)的III型重复序列之一中,鉴定出一名假性软骨发育不全(PSACH)患者存在一种新的错义突变。结果显示,扩大的板层状粗面内质网囊泡含有积聚的COMP以及IX型胶原蛋白(一种软骨特异性成分)。COMP正常分泌并组装到肌腱的细胞外基质中,表明COMP在软骨细胞中的积聚是一种细胞特异性现象。我们认为,COMP的细胞内储存导致软骨特异性分子的非特异性聚集,并导致软骨基质缺乏所需的结构成分,从而导致软骨生长和维持受损。这些数据支持了两种临床相关软骨发育异常疾病——PSACH和多发性骨骺发育异常背后的共同致病机制。