Myeloid targeted gene therapy

The myeloid compartment of the immune system plays a crucial role in defence against infectious organisms, ensures homeostasis of the central nervous system, and is involved in keeping inflammatory processes in check. We develop novel technology for effective gene therapies targeting rare diseases that have a myeloid origin or can be targeted by myeloid-specific gene therapy in the fields of inborn immunodeficiency and neurodegeneration. Our programs rely on two pillars – novel gene addition gene therapy via lentiviral self-inactivated gene therapy vectors (for inborn myeloid immunodeficiency and inherited neurodegeneration), and innovative genome editing methods (for neuro-immunological diseases) and delivery methods for advanced cellular targeting.