Di Rocco Federico, Biosse Duplan Martin, Heuzé Yann, Kaci Nabil, Komla-Ebri Davide, Munnich Arnold, Mugniery Emilie, Benoist-Lasselin Catherine, Legeai-Mallet Laurence
INSERM U781, Université Paris Descartes, Sorbonne Paris Cité, Institut Imagine, Hopital Necker-Enfants malades, Paris, France.
Hum Mol Genet. 2014 Jun 1;23(11):2914-25. doi: 10.1093/hmg/ddu004. Epub 2014 Jan 12.
FGFR3 gain-of-function mutations lead to both chondrodysplasias and craniosynostoses. Achondroplasia (ACH), the most frequent dwarfism, is due to an FGFR3-activating mutation which results in impaired endochondral ossification. The effects of the mutation on membranous ossification are unknown. Fgfr3(Y367C/+) mice mimicking ACH and craniofacial analysis of patients with ACH and FGFR3-related craniosynostoses provide an opportunity to address this issue. Studying the calvaria and skull base, we observed abnormal cartilage and premature fusion of the synchondroses leading to modifications of foramen magnum shape and size in Fgfr3(Y367C/+) mice, ACH and FGFR3-related craniosynostoses patients. Partial premature fusion of the coronal sutures and non-ossified gaps in frontal bones were also present in Fgfr3(Y367C/+) mice and ACH patients. Our data provide strong support that not only endochondral ossification but also membranous ossification is severely affected in ACH. Demonstration of the impact of FGFR3 mutations on craniofacial development should initiate novel pharmacological and surgical therapeutic approaches.
成纤维细胞生长因子受体3(FGFR3)功能获得性突变可导致软骨发育不全和颅缝早闭。软骨发育不全(ACH)是最常见的侏儒症,由FGFR3激活突变引起,导致软骨内骨化受损。该突变对膜内骨化的影响尚不清楚。模仿ACH的Fgfr3(Y367C/+)小鼠以及对ACH和FGFR3相关颅缝早闭患者的颅面分析为解决这一问题提供了契机。通过研究颅盖骨和颅底,我们观察到在Fgfr3(Y367C/+)小鼠、ACH患者以及FGFR3相关颅缝早闭患者中,软骨异常以及软骨结合过早融合,导致枕骨大孔形状和大小改变。Fgfr3(Y367C/+)小鼠和ACH患者还存在冠状缝部分过早融合以及额骨未骨化间隙。我们的数据有力支持了不仅软骨内骨化,而且膜内骨化在ACH中也受到严重影响。FGFR3突变对颅面发育影响的证明应会引发新的药物和手术治疗方法。