Traynis Ilana, Bernstein Jonathan A, Gardner Phyllis, Schrijver Iris
Department of Pathology, L235 Stanford University Medical Center, 300 Pasteur Drive, Stanford, CA 94305, USA.
Cleft Palate Craniofac J. 2012 Jan;49(1):104-8. doi: 10.1597/10-217. Epub 2011 Apr 27.
Craniosynostosis is characterized by premature fusion of one or more cranial sutures and is associated with mutations in fibroblast growth factor receptor (FGFR) genes. Here we describe a novel mutation (1084+1G>A) in the FGFR2 gene of a patient with isolated bicoronal synostosis. We detected two isoforms that result from the mutation and are characterized, respectively, by exon skipping and the use of a cryptic splice site. Interestingly, the alternatively spliced forms of FGFR2 appear to induce fusion of the cranial sutures suggesting that the mutation acts via a gain-of-function mechanism rather than a loss of protein functionality.
颅缝早闭的特征是一条或多条颅缝过早融合,并且与成纤维细胞生长因子受体(FGFR)基因突变有关。在此,我们描述了一名孤立性双冠状缝早闭患者FGFR2基因中的一种新型突变(1084+1G>A)。我们检测到由该突变产生的两种异构体,分别以外显子跳跃和使用隐蔽剪接位点为特征。有趣的是,FGFR2的可变剪接形式似乎会诱导颅缝融合,这表明该突变通过功能获得机制而非蛋白质功能丧失起作用。