硫酸乙酰肝素蛋白聚糖磷脂酰肌醇蛋白聚糖6(GPC6)的突变会损害软骨内骨化并导致隐性骨发育不全。
Mutations in the heparan-sulfate proteoglycan glypican 6 (GPC6) impair endochondral ossification and cause recessive omodysplasia.
作者信息
Campos-Xavier Ana Belinda, Martinet Danielle, Bateman John, Belluoccio Dan, Rowley Lynn, Tan Tiong Yang, Baxová Alica, Gustavson Karl-Henrik, Borochowitz Zvi U, Innes A Micheil, Unger Sheila, Beckmann Jacques S, Mittaz Lauréane, Ballhausen Diana, Superti-Furga Andrea, Savarirayan Ravi, Bonafé Luisa
机构信息
Division of Molecular Pediatrics, Centre Hospitalier Universitaire Vaudois, Lausanne, Switzerland.
出版信息
Am J Hum Genet. 2009 Jun;84(6):760-70. doi: 10.1016/j.ajhg.2009.05.002. Epub 2009 May 28.
Glypicans are a family of glycosylphosphatidylinositol (GPI)-anchored, membrane-bound heparan sulfate (HS) proteoglycans. Their biological roles are only partly understood, although it is assumed that they modulate the activity of HS-binding growth factors. The involvement of glypicans in developmental morphogenesis and growth regulation has been highlighted by Drosophila mutants and by a human overgrowth syndrome with multiple malformations caused by glypican 3 mutations (Simpson-Golabi-Behmel syndrome). We now report that autosomal-recessive omodysplasia, a genetic condition characterized by short-limbed short stature, craniofacial dysmorphism, and variable developmental delay, maps to chromosome 13 (13q31.1-q32.2) and is caused by point mutations or by larger genomic rearrangements in glypican 6 (GPC6). All mutations cause truncation of the GPC6 protein and abolish both the HS-binding site and the GPI-bearing membrane-associated domain, and thus loss of function is predicted. Expression studies in microdissected mouse growth plate revealed expression of Gpc6 in proliferative chondrocytes. Thus, GPC6 seems to have a previously unsuspected role in endochondral ossification and skeletal growth, and its functional abrogation results in a short-limb phenotype.
磷脂酰肌醇蛋白聚糖是一类糖基磷脂酰肌醇(GPI)锚定的、膜结合的硫酸乙酰肝素(HS)蛋白聚糖。尽管人们认为它们可调节HS结合生长因子的活性,但其生物学作用仅得到部分了解。果蝇突变体以及由磷脂酰肌醇蛋白聚糖3突变引起的伴有多种畸形的人类过度生长综合征(辛普森-戈拉比-贝梅尔综合征)突出了磷脂酰肌醇蛋白聚糖在发育形态发生和生长调节中的作用。我们现在报告,常染色体隐性骨发育不全是一种以短肢矮小身材、颅面畸形和不同程度的发育迟缓为特征的遗传疾病,定位于13号染色体(13q31.1-q32.2),由磷脂酰肌醇蛋白聚糖6(GPC6)中的点突变或较大的基因组重排引起。所有突变均导致GPC6蛋白截短,并消除HS结合位点和GPI相关膜结合结构域,因此预计会丧失功能。在显微切割的小鼠生长板中进行的表达研究显示Gpc6在增殖软骨细胞中表达。因此,GPC6似乎在软骨内骨化和骨骼生长中具有先前未被怀疑的作用,其功能缺失导致短肢表型。