Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, Texas.
Department of Pediatrics/Genetics Division, University of Tennessee Health Science Center Memphis, Memphis, Tennessee.
Hum Mutat. 2017 Oct;38(10):1365-1371. doi: 10.1002/humu.23282. Epub 2017 Jul 10.
Pathogenic variants in genes encoding components of the BRG1-associated factor (BAF) chromatin remodeling complex have been associated with intellectual disability syndromes. We identified heterozygous, novel variants in ACTL6A, a gene encoding a component of the BAF complex, in three subjects with varying degrees of intellectual disability. Two subjects have missense variants affecting highly conserved amino acid residues within the actin-like domain. Missense mutations in the homologous region in yeast actin were previously reported to be dominant lethal and were associated with impaired binding of the human ACTL6A to β-actin and BRG1. A third subject has a splicing variant that creates an in-frame deletion. Our findings suggest that the variants identified in our subjects may have a deleterious effect on the function of the protein by disturbing the integrity of the BAF complex. Thus, ACTL6A gene mutation analysis should be considered in patients with intellectual disability, learning disabilities, or developmental language disorder.
编码 BRG1 相关因子 (BAF) 染色质重塑复合物成分的基因中的致病性变异与智力残疾综合征有关。我们在三个智力残疾程度不同的患者中发现了 ACTL6A 基因的杂合性、新的变异,该基因编码 BAF 复合物的一个成分。两个患者有影响肌动蛋白样结构域内高度保守氨基酸残基的错义变异。先前报道酵母肌动蛋白中同源区域的错义突变是显性致死的,并与人类 ACTL6A 与 β-肌动蛋白和 BRG1 的结合受损有关。第三个患者有一个剪接变异,导致框架内缺失。我们的研究结果表明,我们在患者中发现的变异可能通过扰乱 BAF 复合物的完整性对蛋白质的功能产生有害影响。因此,在智力残疾、学习障碍或发育性语言障碍的患者中应考虑 ACTL6A 基因突变分析。