Brison Nathalie, Tylzanowski Przemko, Debeer Philippe
Laboratory of Skeletal Development and Joint Disorders, University of Leuven, Herestraat 49, 3000 Leuven, Belgium.
Eur J Med Genet. 2012 Jan;55(1):1-7. doi: 10.1016/j.ejmg.2011.06.003. Epub 2011 Jul 2.
Synpolydactyly (SPD) is a rare congenital limb disorder caused by mutations in the HOXD13 gene, a homeobox transcription factor crucial for autopod development. The hallmarks of SPD are the webbing between the third and the fourth finger and the fourth and the fifth toe, with a partial or complete digit duplication in the syndactylous web. Different classes of HOXD13 mutations are involved in the pathogenesis of synpolydactyly, but an unequivocal genotype-phenotype correlation cannot always be achieved due to the lack of structure-function data of HOXD13. Mutations in DNA binding or polyalanine tract domains of HOXD13 result in predictable clinical outcomes. However, mutations outside of these domains cause a broad variety of clinical features that complicate the differential diagnosis. In this review, we summarize the different classes of HOXD13 mutations causing synpolydactyly phenotypes with respect to their underlying pathogenic mechanism of action. In addition, we emphasize the importance of the chicken embryo as an animal model system for the study of (limb) development and potential genotype-phenotype correlations in SPD or other human malformation syndromes.
多指(趾)并指(趾)畸形(SPD)是一种罕见的先天性肢体疾病,由HOXD13基因突变引起,HOXD13是一种对肢体末端发育至关重要的同源框转录因子。SPD的特征是第三和第四指以及第四和第五趾之间有蹼,在并指(趾)的蹼中有部分或完全的指(趾)重复。不同类型的HOXD13突变参与了多指(趾)并指(趾)畸形的发病机制,但由于缺乏HOXD13的结构-功能数据,基因型与表型之间的确切相关性并不总是能够实现。HOXD13的DNA结合域或聚丙氨酸 tracts 域中的突变会导致可预测的临床结果。然而,这些域之外的突变会导致各种各样的临床特征,使鉴别诊断变得复杂。在这篇综述中,我们总结了导致多指(趾)并指(趾)畸形表型的不同类型的HOXD13突变及其潜在的致病作用机制。此外,我们强调鸡胚作为动物模型系统在研究(肢体)发育以及SPD或其他人类畸形综合征中潜在的基因型-表型相关性方面的重要性。