KIEM LAB -- Kiem Lab

Welcome to the Kiem Lab

The Laboratory of Dr. Hans-Peter Kiem is located at the Fred Hutchinson Cancer Research Center, in the Clinical Research Division, in Seattle, Washington.

We are part of the University of Washington's Molecular and Cell Biology Graduate Program


Expertise and Research Interests

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:

  • Understand basic hematopoietic stem cell (HSC) biology and clonal composition of hematopoiesis after transplantation of gene-modified HSCs
  • Analyze the integration site pattern of different retroviruses and the safety in various clinical settings
  • Utilize novel nuclease technology including megaTals, zinc finger nucleases and CRISPR/Cas technology to edit hematopoietic cells.
  • Develop novel ways to derive HSCs from induced pluripotent stem cells (iPSCs) and to expand HSCs to facilitate gene therapy and transplantation
  • Develop clinical gene therapy protocols for genetic and acquired diseases, including cancer and in particular Fanconi anemia, glioblastoma, and HIV
  • Develop less toxic hematopoietic cell transplantation protocols for patients with nonmalignant diseases
  • Develop improved vector and delivery systems for gene therapy and gene editing