Twigg Stephen R F, Healy Chris, Babbs Christian, Sharpe Jacqueline A, Wood William G, Sharpe Paul T, Morriss-Kay Gillian M, Wilkie Andrew O M
Weatherall Institute of Molecular Medicine, John Radcliffe Hospital, Oxford, United Kingdom.
Dev Dyn. 2009 Feb;238(2):331-42. doi: 10.1002/dvdy.21790.
Muenke syndrome, defined by heterozygosity for a Pro250Arg substitution in fibroblast growth factor receptor 3 (FGFR3), is the most common genetic cause of craniosynostosis in humans. We have used gene targeting to introduce the Muenke syndrome mutation (equivalent to P244R) into the murine Fgfr3 gene. A rounded skull and shortened snout (often skewed) with dental malocclusion was observed in a minority of heterozygotes and many homozygotes. Development of this incompletely penetrant skull phenotype was dependent on genetic background and sex, with males more often affected. However, these cranial abnormalities were rarely attributable to craniosynostosis, which was only present in 2/364 mutants; more commonly, we found fusion of the premaxillary and/or zygomatic sutures. We also found decreased cortical thickness and bone mineral densities in long bones. We conclude that although both cranial and long bone development is variably affected by the murine Fgfr3(P244R) mutation, coronal craniosynostosis is not reliably reproduced.
孟克综合征由成纤维细胞生长因子受体3(FGFR3)中Pro250Arg替代的杂合性所定义,是人类颅缝早闭最常见的遗传原因。我们利用基因靶向技术将孟克综合征突变(等同于P244R)引入小鼠Fgfr3基因。在少数杂合子和许多纯合子中观察到圆头和短口鼻(常偏斜)以及牙齿咬合不正。这种不完全显性的颅骨表型的发展取决于遗传背景和性别,雄性更常受影响。然而,这些颅骨异常很少归因于颅缝早闭,仅在2/364个突变体中存在;更常见的是,我们发现上颌前部和/或颧骨缝融合。我们还发现长骨的皮质厚度和骨矿物质密度降低。我们得出结论,尽管小鼠Fgfr3(P244R)突变对颅骨和长骨发育均有不同程度的影响,但冠状缝颅缝早闭并不能可靠地重现。