Superti-Furga A, Eich G, Bucher H U, Wisser J, Giedion A, Gitzelmann R, Steinmann B
Department of Pediatrics, University of Zurich, Switzerland.
Eur J Pediatr. 1995 Mar;154(3):215-9. doi: 10.1007/BF01954274.
Achondroplasia, the most common form of chondrodysplasia, has been associated with mutations in the gene of the fibroblast growth factor receptor-3 (FGFR-3) on chromosome 4p. All 39 achondroplasia alleles studied so far carried point mutations which caused the same amino acid exchange, a substitution of glycine by arginine at position 380 (G380R) in the transmembrane domain of the receptor. We report on a newborn with achondroplasia who does not carry a G380R mutation but has a mutation causing substitution of a nearby glycine with a cysteine (G375C). This observation indicates allelic heterogeneity and confirms the role of mutations in the transmembrane domain of FGFR-3 in the pathogenesis of achondroplasia.
软骨发育不全是软骨发育异常最常见的形式,与位于4号染色体短臂上的成纤维细胞生长因子受体3(FGFR - 3)基因突变有关。迄今为止研究的所有39个软骨发育不全等位基因都携带点突变,这些突变导致相同的氨基酸交换,即受体跨膜结构域中第380位的甘氨酸被精氨酸取代(G380R)。我们报告了一名患有软骨发育不全的新生儿,其不携带G380R突变,但有一个导致附近甘氨酸被半胱氨酸取代(G375C)的突变。这一观察结果表明存在等位基因异质性,并证实了FGFR - 3跨膜结构域突变在软骨发育不全发病机制中的作用。