Fabry nephropathy transcriptome. Fabry nephropathy transcriptome

Fabry nephropathy (FN) is a rare disorder caused by mutations in the alpha-galactosidase A gene. In this study we aim at providing a framework allowing selection of biomarkers and drug-targets. Two independent Fabry Nephropathy cohorts (FA.NO and CH.RO) were subjected to RNAseq from archival kidney biopsies taken prior and up to 10 years of Enzyme Replacement Therapy. Four compartments were laser capture microdissected (glomeruli, proximal tubuli, distal tubuli and arteries). We found several pathways that were consistently altered and that these kidney compartments’ transcriptional landscapes can be leveraged in the search for drug-targets and biomarkers. Overall design: RNAseq of lasermicrodissected (LCM) baseline and follow-up kidney biopsy compartments in Fabry Disease *** Submitter declares that, based on Norwegian legislation, submission of the raw-data files is not allowed. These can be made available upon request after individual ethics appliaction to the local IRB and after approval of the Data protection officer. ***