Hans-Peter Kiem

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.