Group for Advanced Molecular Investigation, Graduate Program in Health Sciences, Center for Biological and Health Sciences, Pontifícia Universidade Católica do Parana, Curitiba, PR, Brazil.
Am J Med Genet A. 2011 May;155A(5):1152-6. doi: 10.1002/ajmg.a.33942. Epub 2011 Apr 11.
Split hand/foot malformation (SHFM) is characterized by underdeveloped or absent central digital rays, clefts of hands and feet, and variable syndactyly of the remaining digits. SHFM is a heterogeneous condition caused by abnormalities at one of multiple loci, including SHFM1 (SHFM1 at 7q21-q22), SHFM2 (Xq26), SHFM3 (FBXW4/DACTYLIN at 10q24), SHFM4 (TP63 at 3q27), and SHFM5 (DLX1 and DLX 2 at 2q31). SHFM3 is unique in that it is caused by submicroscopic tandem chromosome duplications of FBXW4/DACTYLIN. In order to show that array-based comparative genomic hybridization should be considered an essential aspect of the genetic analysis of patients with SHFM, we report on a family with two brothers who have ectrodactyly. Interestingly, both also have ocular abnormalities. Their sister and both parents are healthy. DNA of all five family members was analyzed using oligonucleotide-based DNA microarray and quantitative PCR. The two affected brothers were found to have a small duplication of approximately 539 kb at 10q24.32. The patients' sister and father do not have the microduplication, but qPCR showed that mother's DNA carries the duplication in 20% of blood lymphocytes. In this family, two children were affected with ectrodactyly having a duplication over the SHFM3 locus. The mother, who shows no clinical features of ectrodacytyly, is a mosaic for the same duplication. Therefore, we demonstrate that somatic/gonadal mosaicism is a mechanism that gives rise to SHFM. We also suggest that ocular abnormalities may be part of the clinical description of SHFM3.
先天性并指(趾)畸形(split hand/foot malformation,SHFM)的特征为中央指(趾)骨缺失或发育不全、手和足裂、剩余指(趾)的蹼状融合。SHFM 是一种由多个基因座异常引起的异质性疾病,包括 SHFM1(7q21-q22 上的 SHFM1)、SHFM2(Xq26 上的)、SHFM3(10q24 上的 FBXW4/DACTYLIN 上的)、SHFM4(3q27 上的 TP63)和 SHFM5(2q31 上的 DLX1 和 DLX2)。SHFM3 的独特之处在于其由 FBXW4/DACTYLIN 的亚显微串联染色体重复引起。为了表明基于阵列的比较基因组杂交应该成为 SHFM 患者遗传分析的重要方面,我们报告了一个有两个患有外指(趾)畸形兄弟的家庭。有趣的是,他们两人还伴有眼部异常。他们的妹妹和父母都健康。使用寡核苷酸 DNA 微阵列和定量 PCR 分析了所有五名家庭成员的 DNA。结果发现,这两个受影响的兄弟在 10q24.32 上有一个约 539kb 的小重复。患者的妹妹和父亲没有微重复,但 qPCR 显示母亲的 DNA 在 20%的血淋巴细胞中携带重复。在这个家庭中,两个孩子患有外指(趾)畸形,在 SHFM3 基因座上有重复。没有外指(趾)畸形临床特征的母亲是该重复的嵌合体。因此,我们证明了体/性腺嵌合是导致 SHFM 的一种机制。我们还建议眼部异常可能是 SHFM3 临床描述的一部分。