Shotelersuk Vorasuk, Punyashthiti Rachaneekorn
Section on Medical Genetics and Metabolism, Department of Pediatrics, King Chulalongkorn Memorial Hospital, Bangkok 10330, Thailand.
Int J Mol Med. 2002 Jan;9(1):81-4.
Pseudoachondroplasia (PSACH) is an autosomal dominant disorder characterized by disproportionate short stature and precocious osteoarthritis. Radiographic manifestations include epiphyseal, metaphyseal and vertebral abnormalities. Mutations in the cartilage oligomeric matrix protein (COMP) have been identified to cause PSACH. Most of them affect one of the eight calcium-binding domains of COMP. We describe a clinically and radiologically typical PSACH 4-year-old girl and her 31-year-old father. A novel mutation, 1345-1347CCC deletion in exon 13, of COMP was identified in both patients. The deletion would be expected to result in the loss of the conserved proline at codon 449 from the sixth calcium-binding domain. This result further supports that COMP is the only gene, discovered to date, responsible for PSACH across different populations and that the calcium-binding domains are important to the function of the normal COMP.
假性软骨发育不全(PSACH)是一种常染色体显性疾病,其特征为身材不成比例矮小和早熟性骨关节炎。影像学表现包括骨骺、干骺端和椎体异常。已确定软骨寡聚基质蛋白(COMP)突变会导致PSACH。其中大多数影响COMP的八个钙结合结构域之一。我们描述了一名临床和放射学表现典型的4岁PSACH女童及其31岁的父亲。在两名患者中均鉴定出COMP基因外显子13中的一个新突变,即1345 - 1347CCC缺失。该缺失预计会导致第六个钙结合结构域中第449位密码子处保守脯氨酸的缺失。这一结果进一步支持COMP是迄今为止发现的唯一一个在不同人群中导致PSACH的基因,并且钙结合结构域对正常COMP功能很重要。