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Centrum für Chronische Immundefizienz - CCI

Research Group
Genome Engineering

CCI Junior Group Leader: Dr. Claudio Mussolino

The goals of the research group Genome Engineering are the further development of designer nuclease platforms and their applications for primary and secondary immunodeficiencies.

Patients with congenital and acquired immunodeficiencies suffer from frequent and severe infections. The therapeutic options for these patients include blood stem cell transplantation, to replace the defective immune system with a healthy one provided by the donor stem cells. However, this approach is not suitable e.g. for HIV patients because the virus would infect the new immune system again.

The aim of the research group Genome Engineering is to modify blood stem cells of affected patients in such a way that the derived immune cells are resistant to HIV. Since the virus enters the target cells (T cells, macrophages) via binding to the CCR5 co-receptor, selective disruption of the gene encoding this receptor with designer nucleases renders these cells resistant to virus infection. The research group Genome Engineering develops novel and safer types of designer nucleases, such as TALENs (transcription activator-like effector nucleases) and CRISPR/Cas based nucleases. The long-term goal is the development of long-lasting therapeutic approaches for HIV patients on the basis of targeted gene editing in blood stem cells and the adaptation of this methodology to other acquired and congenital disorders of the immune system.  


Dr. Claudio Mussolino
CCI Junior Group Leader

Institute for Transfusion Medicine and Gene Therapy &
Center for Chronic Immunodeficiency
at Center for Translational Cell Research
Breisacher Straße 115
79106 Freiburg

Phone +49 761 270 77738
Fax +49 761 270 77744