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Institute for Regenerative Medicine, University of Zurich;
Somatic Gene Therapy, University Children`s Hospital Zurich
Institute for Regenerative Medicine
University Children`s Hospital Zurich
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Our translational research is focused on inborn errors of the immune system, spanning from analysis of molecular pathophysiology to development of new therapeutic concepts and therapeutic correction by clinical gene therapy. Our group is involved as principal investigator in two large collaborative EU-FP7 projects – CELL-PID (development of next-generation of LV-SIN gene therapy vectors for X-linked Chronic Granulomatous Disease [CGD]) and NET4CGD (clinical phase I/II gene therapy study for the treatment of X-CGD). Technical expertise in the field of inborn errors of innate immunity: CGD diagnostics (detection and quantification of reactive oxygen species (ROS) formation in various cellular systems), CGD gene therapy monitoring (functional assays including neutrophil extracellular trap [NET]-formation and pathogen killing) and monitoring of vector insertion and clonal evolution (analysis of clonality, vector copy number analysis), gene therapy vector development and vector testing in cell lines, iPSCs and animal models. Furthermore, we focus on the analysis of hyperinflammatory pathophysiology observed in CGD.