Background
A renowned researcher and physician, Dr. Kiem has extensive experience in hematopoietic stem cell transplantation and cellular immunotherapy. He has trained and mentored more than 50 students and postdoctoral fellows over the past two decades.
Dr. Kiem’s research focuses on extracting stem cells from patients, modifying them at the genetic level and returning them to the body to treat a wide range of conditions, including cancer, genetic diseases such as sickle cell disease and infections like HIV. He is currently sponsoring multiple clinical gene therapy studies, including a first-of-its-kind trial using gene-modified stem cells to correct a gene associated with Fanconi anemia, a rare blood disorder that frequently leads to leukemia and other cancers.
His lab is a leader in gene-editing technologies, including the design of gene-cutting enzymes such as CRISPR/Cas, which act as molecular scissors to precisely disable or repair faulty genes. By combining gene therapy with the regenerative capacity of stem cells, Dr. Kiem aims to develop curative treatments for diseases such as HIV, leukemia, sickle cell disease 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 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.
In addition to caring for patients and conducting research, he also serves as chair of the American Society for Gene and Cell Therapy’s Stem Cell Committee and the American Society of Hematology’s Stem Cell Committee.
Area of Clinical Practice
Blood and marrow transplantation, cellular immunotherapy, hematopoietic stem cell transplantation, gene therapy, graft-versus-host disease, cellular immunotherapy
