Clinical translation stem cell gene therapy
Research Focus
We have developed a novel lentiviral self-inactivated (SIN) vector for treatment of hematopoietic stem cells to treat patients with an inborn immunodeficiency of phagocytes, chronic granulomatous disease (CGD). Our team is preparing an international first-in-man clinical trial to treat patients with autosomal-recessive CGD. The gene therapy medicinal product will be produced by our translational team under GMP (good manufacturing practice), at the Wyss Zurich Translational centre.
Group Members
Technician
Franziska Schenk