Welcome to the Kiem Lab
The main focus of our lab is to study stem cell biology/transplantation and gene therapy/editing with the goal of developing novel gene therapy-based treatment strategies for patients with genetic, infectious, or malignant diseases. Our research utilizes hematopoietic stem cells (HSCs), embryonic stem cells (ESCs), and induced pluripotent stem cells (iPSCs) as targets for gene transfer. HSCs are particularly useful in gene therapy and gene editing because of their ability to permanently reconstitute the hematopoietic and immune systems. However, in order to effectively modify HSCs for clinical use one must identify, isolate and expand HSCs and create vector systems or gene transfer tools that facilitate safe and efficient modification of HSCs.
Recent studies from the Kiem lab have examined the role of endothelial cells in iPSC-derived HSC specification and expansion. We have also used endothelial cell support to expand adult marrow HSCs and used novel small molecules for expansion. We have demonstrated successful ex vivo expansion of human cord blood cells when cultured in the presence of specific small molecules. We also have 4 active clinical gene therapy studies. One study aims at treating patients with glioblastoma and we have now treated 7 patients and have shown in our most recent manuscript improved survival in high-risk patients with glioblastoma. One clinical study involves the correction of the genetic defect in blood and marrow stem cells from patients with Fanconi anemia and we have now treated 2 patients. Two clinical studies involve gene therapy approaches for patients with HIV and lymphoma undergoing either primary chemotherapy or requiring an autologous HSC transplantation. The Kiem lab is also part of multiple collaborations to improve the efficiency by which HSCs can be cultured and transduced, while closely monitoring the gene modified clones through cutting-edge high-throughput retroviral integration site analysis.
The current research in our laboratory focuses on studies to: