Wessex Regional Genetics Laboratory, Salisbury District Hospital, Salisbury, Wiltshire, UK.
Am J Med Genet A. 2010 Dec;152A(12):3124-8. doi: 10.1002/ajmg.a.33567.
We describe a patient with striking generalized symmetrical enchondromatosis of the tubular bones and a de novo duplication of chromosome 12p11.23 to 12p11.22. The PTHLH gene within this region encodes a ligand for PTHR1: mutations in the gene encoding this receptor are associated with some cases of Ollier disease, several skeletal dysplasias including Blomstrand, Eiken, and Jansen and down-regulation of PTHLH expression in brachydactyly type E. Our findings suggest that abnormal PTHLH-PTHR1 signaling may underly this unusual form of enchondromatosis and indicate that unlike most cases of Ollier disease it is dominantly inherited.
我们描述了一位患者,其管状骨存在显著的对称性软骨瘤病,且 12p11.23 到 12p11.22 染色体发生了从头重复。该区域内的 PTHLH 基因编码了 PTHR1 的配体:该受体基因的突变与一些 Ollier 病、包括 Blomstrand、Eiken 和 Jansen 在内的几种骨骼发育不良以及 E 型短指(趾)症的 PTHLH 表达下调有关。我们的发现表明,异常的 PTHLH-PTHR1 信号可能是这种不寻常形式的软骨瘤病的基础,并表明与大多数 Ollier 病病例不同,它是显性遗传的。