Department of Developmental and Regenerative Biology, Mount Sinai School of Medicine, New York, NY 10029, USA.
South China Center for Biomedical Research, Fujian Key Laboratory of Developmental and Neuro Biology, College of Life Science, Fujian Normal University, Fuzhou, Fujian Province, PR China.
Dev Biol. 2014 Jul 15;391(2):170-81. doi: 10.1016/j.ydbio.2014.04.013. Epub 2014 Apr 28.
Congenital bony syngnathia, a rare but severe human birth defect, is characterized by bony fusion of the mandible to the maxilla. However, the genetic mechanisms underlying this birth defect are poorly understood, largely due to limitation of available animal models. Here we present evidence that transgenic expression of Bmp4 in neural crest cells causes a series of craniofacial malformations in mice, including a bony fusion between the maxilla and hypoplastic mandible, resembling the bony syngnathia syndrome in humans. In addition, the anterior portion of the palatal shelves emerged from the mandibular arch instead of the maxilla in the mutants. Gene expression assays showed an altered expression of several facial patterning genes, including Hand2, Dlx2, Msx1, Barx1, Foxc2 and Fgf8, in the maxillary and mandibular processes of the mutants, indicating mis-patterned cranial neural crest (CNC) derived cells in the facial region. However, despite of formation of cleft palate and ectopic cartilage, forced expression of a constitutively active form of BMP receptor-Ia (caBmprIa) in CNC lineage did not produce the syngnathia phenotype, suggesting a non-cell autonomous effect of the augmented BMP4 signaling. Our studies demonstrate that aberrant BMP4-mediated signaling in CNC cells leads to mis-patterned facial skeleton and congenital bony syngnathia, and suggest an implication of mutations in BMP signaling pathway in human bony syngnathia.
先天性骨性并颌畸形是一种罕见但严重的人类出生缺陷,其特征是下颌骨与上颌骨的骨性融合。然而,这种出生缺陷的遗传机制尚不清楚,这主要是由于可用的动物模型有限。在这里,我们提供的证据表明,Bmp4 在神经嵴细胞中的转基因表达会导致小鼠一系列颅面畸形,包括上颌骨和发育不良的下颌骨之间的骨性融合,类似于人类的骨性并颌畸形综合征。此外,在突变体中,腭板的前部分从下颌弓中出现,而不是从上颌中出现。基因表达分析表明,突变体上颌骨和下颌骨突中的几个面部模式形成基因(包括 Hand2、Dlx2、Msx1、Barx1、Foxc2 和 Fgf8)的表达发生改变,表明面部颅神经嵴(CNC)衍生细胞的模式发生改变。然而,尽管形成了腭裂和异位软骨,但在 CNC 谱系中强制表达组成型激活形式的 BMP 受体-Ia(caBmprIa)并没有产生并颌表型,这表明增强的 BMP4 信号传导具有非细胞自主效应。我们的研究表明,CNC 细胞中异常的 BMP4 介导的信号传导导致面部骨骼模式发生改变和先天性骨性并颌畸形,并提示 BMP 信号通路突变与人类骨性并颌畸形有关。