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Gene therapy is a recent break-through therapeutic field that has the potential to cure innate or acquired diseases by correcting or circumventing disease-causing genetic mutations. Our translational research is fully dedicated to the design, pre-clinical development, and clinical implementation of gene therapy treatments for rare incurable diseases, with a current focus on inborn errors of the immune system.
Our mission is to accelerate medical application of scientific discoveries arising from fundamental research, uniting expertise of basic and translational researchers as well as clinicians at the University Medicine Zurich hub.
Our current focus is hematopoietic stem cell (HSC) based ex vivo gene therapy, using two approaches: viral vector gene therapy, consisting of addition of a correct coding sequence of the mutated gene into the patient’s HSC genome by lentiviral vectors, and genome editing, allowing for targeted insertion of corrective sequences at a defined, ideally the endogenous locus of the mutated gene, or for excision of mutated DNA sequence.
We are currently developing innovative gene and cell therapies for rare diseases, thereby are also paving the grounds for the Medicine of tomorrow, providing proof-of-principle models and transferable new treatment tools for more frequent disorders.
Our research is supported by various national and international grant agencies including SNSF, DFG, Hartmann Müller Stiftung, Olga Mayenfisch Foundation, University Research Priority Program of the University of Zurich (URPP Itinerare), Clinical Research Priority Program of the Medical Faculty of the University of Zurich (CRPP ImmuGene), Wyss Translational Centre Zurich.
Julia Ackva
Dominik Buck, PhD student
Timon Menzi
PD Dr. Ute Modlich
Martina Nubiè, PhD student
Dr. Naz Leal Oburoglu, Postdoctoral researcher
Dr. Oleksandr Pastukhov
Simon Pöllmann, PhD student
Gilles Sartre, PhD student
Dr. Kah Mun Siow, Postdoctoral researcher
