Research Focus: Gene- and cell therapy
Gene therapy is a recent break-through therapeutic field that has the potential to cure innate or acquired diseases. The translational research of the group 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. The innovative gene and cell therapies currently developed for rare diseases are also paving the grounds for the medicine of tomorrow, providing proof-of-principle models and transferable new treatment tools for more frequent disorders.
First generation gene therapy using a gamma-retroviral vector to correct hematopoietic stem cells has been performed several years ago within a phase I/II gene therapy trial to treat patients with an inborn immunodeficiency of phagocytes, chronic granulomatous disease (CGD) (PMID: 25981636, 25245086, 16582916).
Another phase I/II clinical trial for X-linked CGD using an efficiency and safety improved lentiviral self-inactivated (SIN) vector is currently ongoing within the EU-FP7 program (www.NET4CGD.eu, PMID: 25245086, 21045810). In these novel types of vectors, the expression of the therapeutic gene is driven by an internal tissue-specific promotor to avoid potential side effects at the level of hematopoietic stem cells.
We have developed a third generation lentiviral SIN vector for autosomal-recessive p47phox- deficient CGD, which is currently translated into clinical application within a first-in-man clinical trial (EU-FP7 CELL-PID, CRPP ImmuGene, PMID: 20445581, 22311747, 23489116),
CRISPR/Cas mediated genome editing gene therapy for patients with immunodeficiency, and at a later phase, potentially diseases of the skin, blood, metabolic and nerve system are in preparation (CRPP ImmuGene).
Our research is supported by:
Various national and international grant agencies including EU-FP7, Clinical Research Priority Program of the Medical Faculty of Zurich University (CRPP ImmuGene), CGD Society UK, Fondazione Ettore e Valeria Rossi, Gottfried und Julia Bangerter-Rhyner-Stiftung, Hochspezialisierte Medizin Schwerpunkt Immunologie (HSM-2-Immunologie).