Christoph Klein

Professor Christoph Klein is Chair of the Department of Pediatrics at Ludwig-Maximilians-University Munich, leading a research group focused on monogenic immunodeficiencies, gene therapy, and inflammatory bowel disease. His work bridges clinical pediatrics with cutting-edge molecular research, particularly in severe congenital neutropenia and very early onset inflammatory bowel disease (VEO-IBD). The Klein Lab operates within the Gene Center at LMU Munich, maintaining strong international collaborations.

His educational background spans multiple continents and disciplines: MD from LMU Munich (1991), PhD in Immunology from University Paris VI (2000), clinical training at Harvard Medical School (1995-2000), and philosophical studies at Munich and Harvard. His dual expertise in medicine and philosophy informs his holistic approach to rare pediatric diseases.

Professor Klein's research primarily investigates monogenic disorders of the immune system, with particular emphasis on severe congenital neutropenia (SCN), inflammatory bowel disease in children, and primary immunodeficiency disorders. His team has established innovative models using induced pluripotent stem cells combined with CRISPR/Cas9 technology to study neutrophil differentiation and function. The lab's work on IL-10 receptor deficiencies has led to novel therapeutic approaches for VEO-IBD patients through hematopoietic stem cell transplantation.

Analysis of Professor Klein's recent publications reveals a consistent focus on identifying genetic causes of rare pediatric immune disorders, particularly through whole genome sequencing approaches. His work bridges basic science with clinical applications, with significant contributions to understanding neutrophil biology, inflammatory bowel disease mechanisms, and gene therapy safety. The research demonstrates increasing sophistication in using stem cell models, proteomics, and systems biology to understand rare disease mechanisms.

• Khwarizmi International Award, Islamic Republic Iran (2016)
• Cusanus-Prize (2015)
• Member, American Society of Clinical Investigation (2014)
• Gottfried-Wilhelm-Leibniz Award, Deutsche Forschungsgemeinschaft (2010)
• William-Dameshek-Prize, American Society of Hematology (2011)
• Paul-Martini-Award (2011)

Professor Klein mentors multiple doctoral researchers and collaborates with a diverse team including Principal Investigator Daniel Kotlarz and Scientific Project Manager Regina Steck. His research is supported by numerous prestigious grants including an ERC advanced grant, SFB1054, SFB914, KFO 250, and the VEO-IBD Consortium funded by the Helmsley Charitable Trust. His work on Wiskott-Aldrich syndrome gene therapy, though demonstrating efficacy, also revealed important safety considerations regarding vector integration.

The Klein Lab operates within the Gene Center at LMU Munich, maintaining the Care-for-Rare alliance which provides rapid genomic sequencing for patients with severe congenital neutropenia worldwide. The lab has developed sophisticated models using induced pluripotent stem cells combined with CRISPR/Cas9 technology to study disease mechanisms and test potential therapies. Current research focuses on understanding the molecular basis of neutrophil disorders, developing novel diagnostic approaches for primary immunodeficiencies, and translating genetic findings into targeted therapies for children with rare diseases.