Developmental Genetics Unit, King Faisal Specialist Hospital and Research Center, Riyad 11211, Saudi Arabia.
J Med Genet. 2012 Jan;49(1):16-20. doi: 10.1136/jmedgenet-2011-100556. Epub 2011 Nov 25.
Split hand and foot malformation (SHFM) refers to a genetically heterogeneous developmental disorder of the hands and feet that presents as median ray deficiency of varying severity. 7q21.3 (SHFM1) is one of six loci described to date, and although DLX5 and DLX6 are compelling candidates in that locus, no intragenic mutations have been described in either of these genes.
The authors combined autozygome analysis and exome sequencing to study a consanguineous family with a highly unusual SHFM phenotype, where there is associated dorsalisation of the hands.
A novel missense mutation in a highly conserved residue of the homeobox domain of DLX5 was identified. Unlike previously reported position effect mutations in SHFM1, this first documented intragenic DLX5 mutation is also accompanied by abnormal dorsal-ventral patterning.
This study identified the first intragenic DLX5 mutation in SHFM and raises interesting possibilities about a dual role for DLX5 in limb development.
分裂手和脚畸形(SHFM)是一种手部和脚部的遗传异质性发育障碍,表现为中射线的不同程度缺失。7q21.3(SHFM1)是迄今为止描述的六个基因座之一,尽管 DLX5 和 DLX6 是该基因座中的有力候选基因,但这两个基因中都没有描述过基因内突变。
作者结合自交系分析和外显子组测序,研究了一个具有非常不寻常的 SHFM 表型的近亲家庭,该表型与手部的背侧化有关。
在 DLX5 的同源盒结构域的一个高度保守残基中发现了一个新的错义突变。与以前报道的 SHFM1 中的位置效应突变不同,这个首次记录的基因内 DLX5 突变也伴随着异常的背腹模式。
本研究鉴定了 SHFM 中的第一个基因内 DLX5 突变,并提出了 DLX5 在肢体发育中具有双重作用的有趣可能性。