Deere M, Sanford T, Ferguson H L, Daniels K, Hecht J T
Department of Pediatrics, University of Texas Medical School at Houston, 77225-0708, USA.
Am J Med Genet. 1998 Dec 28;80(5):510-3. doi: 10.1002/(sici)1096-8628(19981228)80:5<510::aid-ajmg14>3.0.co;2-f.
Pseudoachondroplasia (PSACH) is an autosomal dominant dwarfing condition characterized by disproportionate short stature, joint laxity, and early-onset osteoarthrosis. PSACH is caused by mutations in the gene encoding cartilage oligomeric matrix protein (COMP). We are reporting on mutations in COMP in 12 patients with PSACH, including ten novel mutations. Eleven of the mutations are in exons 17A, 17B, and 18A, which encode the calcium-binding domains, and one mutation is in exon 19, which encodes part of the carboxy-terminal globular domain. Two of the mutations identified are the common delGAC(1430-1444) in exon 17B, which accounts for 36% of identified PSACH mutations. This report increases the range of mutations in COMP that cause PSACH and provides additional evidence for the importance of the calcium-binding domains and the globular domain to the function of COMP.
假性软骨发育不全(PSACH)是一种常染色体显性遗传性侏儒症,其特征为身材比例失调、关节松弛和早发性骨关节炎。PSACH由编码软骨寡聚基质蛋白(COMP)的基因突变引起。我们报告了12例PSACH患者COMP基因的突变情况,其中包括10种新突变。11种突变位于外显子17A、17B和18A,这些外显子编码钙结合结构域,1种突变位于外显子19,该外显子编码羧基末端球状结构域的一部分。所鉴定出的两种突变是外显子17B中常见的delGAC(1430 - 1444),占已鉴定的PSACH突变的36%。本报告增加了导致PSACH的COMP基因突变范围,并为钙结合结构域和球状结构域对COMP功能的重要性提供了更多证据。