Hans-Peter

Kiem

M.D.

Publications

Research and/or clinical interests 

Dr. Hans-Peter Kiem is a world-renowned pioneer in stem-cell and gene therapy and in the development of new gene-editing technologies. His focus has been the development of improved treatment and curative approaches for patients with genetic and infectious diseases or cancer. For gene editing, his lab works on the design and selection of enzymes, known as nucleases, which include CRISPR/Cas. These enzymes function as molecular scissors that are capable of accurately disabling defective genes. By combining gene therapy’s ability to repair problem-causing genes and stem cells’ regenerative capabilities, he hopes to achieve cures of diseases as diverse as HIV, leukemia and brain cancer. With preclinical models of HIV, Dr. Kiem and his colleagues have demonstrated that they can modify a key viral entry gene and prevent it from working in transplanted blood stem cells. He also hopes to apply these technologies to cure genetic blood disorders such as Fanconi anemia and sickle cell disease. He is also pioneering in vivo gene therapy approaches to make gene therapy and gene editing more broadly available and accessible to patients and those living with HIV, especially in resource-limited settings.

Dr. Keim’s current projects include:

• Understanding basic hematopoietic stem cell (HSC) and transplantation biology and clonal composition of hematopoiesis after transplantation, especially the clonality of gene-modified HSCs.
• Developing and evaluating novel virus-based gene therapy technology and nuclease technology including megaTals, zinc finger nucleases and CRISPR/Cas technology to edit hematopoietic cells with the goal to improve the treatment for genetic diseases such as hemoglobinopathies, Fanconi anemia and immunodeficiencies. 
• Developing novel immunotherapies for patients with HIV and myeloid malignancies. 
• Developing novel approaches to protect hematopoietic stem and progenitor cells for selection in vivo. 
• Developing novel in vivo gene therapy approaches for genetic diseases, HIV and cancer. 
• Developing clinical gene therapy protocols for genetic and acquired diseases, including cancer. Current target diseases include Fanconi anemia, severe combined immunodeficiency, hemoglobinopathies, glioblastoma, and HIV.
• Developing less toxic conditioning hematopoietic cell transplantation protocols, especially for patients with nonmalignant diseases.