Department of Dermatology and Cutaneous Biology, Jefferson Institute of Molecular Medicine, and the PXE International Center of Excellence in Research and Clinical Care, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.
The Jackson Laboratory, Bar Harbor, Maine, USA.
J Invest Dermatol. 2019 Dec;139(12):2447-2457.e7. doi: 10.1016/j.jid.2019.04.023. Epub 2019 Jun 15.
Pseudoxanthoma elasticum, a prototype of heritable multisystem ectopic mineralization disorders, is caused by mutations in the ABCC6 gene encoding a putative efflux transporter, ABCC6. The phenotypic spectrum of pseudoxanthoma elasticum varies, and the correlation between genotype and phenotype has not been established. To identify genetic modifiers, we performed quantitative trait locus analysis in inbred mouse strains that carry the same hypomorphic allele in Abcc6 yet with highly variable ectopic mineralization phenotypes of pseudoxanthoma elasticum. Abcc6 was confirmed as a major determinant for ectopic mineralization in multiple tissues. Integrative analysis using functional genomics tools that included GeneWeaver, String, and Mouse Genome Informatics identified a total of nine additional candidate modifier genes that could influence the organ-specific ectopic mineralization phenotypes. Integration of the candidate genes into the existing ectopic mineralization gene network expands the current knowledge on the complexity of the network that, as a whole, governs ectopic mineralization in soft connective tissues.
弹性假黄瘤是一种遗传性多系统异位矿化疾病的原型,由编码假定外排转运蛋白 ABCC6 的 ABCC6 基因突变引起。弹性假黄瘤的表型谱存在差异,基因型与表型之间的相关性尚未建立。为了鉴定遗传修饰因子,我们在携带 Abcc6 相同功能缺失等位基因但具有高度可变的弹性假黄瘤异位矿化表型的近交系小鼠中进行了数量性状基因座分析。ABCC6 被确认为多种组织异位矿化的主要决定因素。使用包括 GeneWeaver、String 和 Mouse Genome Informatics 在内的功能基因组学工具进行的综合分析总共确定了另外 9 个候选修饰基因,它们可能影响器官特异性异位矿化表型。候选基因的整合到现有的异位矿化基因网络中,扩展了网络复杂性的现有知识,该网络作为一个整体,控制着软连接组织中的异位矿化。