Division of Pediatric Nephrology, Boston Children's Hospital, Boston, Massachusetts, USA; Kidney Disease Initiative, Broad Institute, Cambridge, Massachusetts, USA.
Division of Nephrology, Department of Internal Medicine, Michigan Medicine, Ann Arbor, Michigan, USA.
Kidney Int. 2022 Jul;102(1):136-148. doi: 10.1016/j.kint.2021.10.041. Epub 2021 Dec 18.
Apolipoprotein L1 (APOL1)-associated focal segmental glomerulosclerosis (FSGS) is the dominant form of FSGS in Black individuals. There are no targeted therapies for this condition, in part because the molecular mechanisms underlying APOL1's pathogenic contribution to FSGS are incompletely understood. Studying the transcriptomic landscape of APOL1 FSGS in patient kidneys is an important way to discover genes and molecular behaviors that are unique or most relevant to the human disease. With the hypothesis that the pathology driven by the high-risk APOL1 genotype is reflected in alteration of gene expression across the glomerular transcriptome, we compared expression and co-expression profiles of 15,703 genes in 16 Black patients with FSGS at high-risk vs 14 Black patients with a low-risk APOL1 genotype. Expression data from APOL1-inducible HEK293 cells and normal human glomeruli were used to pursue genes and molecular pathways uncovered in these studies. We discovered increased expression of APOL1 and nine other significant differentially expressed genes in high-risk patients. This included stanniocalcin, which has a role in mitochondrial and calcium-related processes along with differential correlations between high- and low-risk APOL1 and metabolism pathway genes. There were similar correlations with extracellular matrix- and immune-related genes, but significant loss of co-expression of mitochondrial genes in high-risk FSGS, and an NF-κB-down regulating gene, NKIRAS1, as the most significant hub gene with strong differential correlations with NDUF family (mitochondrial respiratory genes) and immune-related (JAK-STAT) genes. Thus, differences in mitochondrial gene regulation appear to underlie many differences observed between high- and low-risk Black patients with FSGS.
载脂蛋白 L1(APOL1)相关局灶节段性肾小球硬化症(FSGS)是黑人中 FSGS 的主要形式。目前尚无针对这种疾病的靶向治疗方法,部分原因是 APOL1 对 FSGS 的致病作用的分子机制尚未完全了解。研究患者肾脏中 APOL1 FSGS 的转录组景观是发现对人类疾病具有独特或最相关的基因和分子行为的重要方法。我们假设由高风险 APOL1 基因型驱动的病理学反映在整个肾小球转录组中基因表达的改变,我们比较了 16 名高风险黑人 FSGS 患者和 14 名低风险 APOL1 基因型黑人患者的 15703 个基因的表达和共表达谱。APOL1 诱导的 HEK293 细胞和正常人类肾小球的表达数据用于研究这些研究中发现的基因和分子途径。我们发现高风险患者中 APOL1 和其他九个显著差异表达基因的表达增加。这包括参与线粒体和钙相关过程的 stanniocalcin,以及高风险和低风险 APOL1 与代谢途径基因之间的差异相关性。与细胞外基质和免疫相关基因也有类似的相关性,但高风险 FSGS 中存在线粒体基因共表达的显著丧失,以及 NF-κB 下调基因 NKIRAS1,作为与 NDUF 家族(线粒体呼吸基因)和免疫相关(JAK-STAT)基因具有强烈差异相关性的最重要的枢纽基因。因此,线粒体基因调控的差异似乎是高风险和低风险黑人 FSGS 患者之间许多差异的基础。