Passos-Bueno M R, Sertié A L, Richieri-Costa A, Alonso L G, Zatz M, Alonso N, Brunoni D, Ribeiro S F
Departamento de Biologia, Instituto de Biociências, Universidade de São Paulo, Brazil.
Am J Med Genet. 1998 Jul 7;78(3):237-41.
Dominant mutations in three fibroblast growth factor receptor genes (FGFRs1-3) cause Crouzon, Jackson-Weiss, Pfeiffer, and Apert syndromes. In the present study, 50 Brazilian patients with these four syndromes (27 Apert, 17 Crouzon, 5 Pfeiffer, and 1 Jackson-Weiss patients) were screened for mutations in the FGFR1-3 genes. Except for one, all the Apert patients had either S252W (n = 16) or P253R (n = 10) mutations. The remaining Apert case is atypical with a mutation altering the splice site of FGFR2 exon IIIc. The Pfeiffer patients had mutations in one of the FGFR genes: three in FGFR2, one in FGFR1, and one in FGFR3. In contrast, only 8 of the 17 Crouzon patients studied had a mutation in either FGFR2 (n = 7) or FGFR3 locus (n = 1). Mutations in the FGFR2 locus account for most (93%) of our syndromic craniosynostotic cases, whereas 5% had mutations in the FGFR3 locus and only 2% had mutations in the FGFR1 gene. Except for one, all the other mutations were reported previously in craniosynostotic patients from other populations. Interestingly, the mutation C278F, previously described in Crouzon and Pfeiffer cases, was here identified in a familial case with Jackson-Weiss. Also, unexpectedly, a common mutation altering the splice site of the FGFR2 exon IIIc was found in one Apert and two Pfeiffer patients. In addition, we identified a new mutation (A337P) in the FGFR2 exon IIIc associated with Crouzon phenotype.
三个成纤维细胞生长因子受体基因(FGFRs1 - 3)的显性突变会导致克鲁宗综合征、杰克逊 - 韦斯综合征、 Pfeiffer综合征和阿佩尔综合征。在本研究中,对50名患有这四种综合征的巴西患者(27名阿佩尔综合征患者、17名克鲁宗综合征患者、5名Pfeiffer综合征患者和1名杰克逊 - 韦斯综合征患者)进行了FGFR1 - 3基因突变筛查。除了1例患者外,所有阿佩尔综合征患者都有S252W(n = 16)或P253R(n = 10)突变。其余1例阿佩尔综合征病例是非典型的,其突变改变了FGFR2外显子IIIc的剪接位点。Pfeiffer综合征患者在FGFR基因之一中存在突变:FGFR2中有3例,FGFR1中有1例,FGFR3中有1例。相比之下,在研究的17例克鲁宗综合征患者中,只有8例在FGFR2(n = 7)或FGFR3位点(n = 1)有突变。FGFR2位点的突变占我们综合征性颅缝早闭病例的大多数(93%),而5%的病例在FGFR3位点有突变,只有2%的病例在FGFR1基因中有突变。除了1例患者外,所有其他突变先前在其他人群的颅缝早闭患者中都有报道。有趣的是,先前在克鲁宗综合征和Pfeiffer综合征病例中描述的C278F突变,在1例杰克逊 - 韦斯综合征家族病例中被发现。此外,意外的是,在1例阿佩尔综合征患者和2例Pfeiffer综合征患者中发现了一个改变FGFR2外显子IIIc剪接位点的常见突变。此外,我们在FGFR2外显子IIIc中鉴定出一个与克鲁宗综合征表型相关的新突变(A337P)。