Horton W A
Research Department, Portland Shriners Hospital, Oregon, USA.
Endocrinol Metab Clin North Am. 1996 Sep;25(3):683-97. doi: 10.1016/s0889-8529(05)70347-9.
Considerable progress has been made in delineating the molecular genetic basis of the human chondrodysplasias. Two genes emerge as harboring mutations found in patients with the most common disorders. Mutations in the type II collagen gene account for most spondyloepiphyseal dysplasia and spondyloepiphyseal dysplasia-like clinical disorders, whereas mutations in the fibroblast growth factor receptor 3 gene are responsible for achondroplasia, thanatophoric dysplasia, and hypochondroplasia. A substantial portion of remaining patients have mutations of the genes encoding cartilage oligomeric matrix protein or diastrophic dysplasia sulfate transporter.
在阐明人类软骨发育不全的分子遗传基础方面已取得了相当大的进展。有两个基因因在患有最常见疾病的患者中发现存在突变而备受关注。II型胶原蛋白基因突变是大多数脊椎骨骺发育不良和脊椎骨骺发育不良样临床疾病的病因,而成纤维细胞生长因子受体3基因突变则导致软骨发育不全、致死性发育不良和软骨发育低下。其余相当一部分患者存在编码软骨寡聚基质蛋白或硫酸酯转运蛋白的基因突变。