Research is the answer to one day making FA a treatable, manageable disease. Here, you'll discover the most recent strides in FA research and activities funded by FCF. Every quarter, we'll bring you updates on newly funded grants, ongoing projects, and significant milestones.
Directory
Hans-Peter Kiem
Director, Cell and Gene Therapy Program | MD | Seattle, USA
Dr. Kiem’s research has focused on stem cell and transplantation biology, cell and gene therapy and the development and use of novel gene editing technologies. The overall goal has been the development of improved treatment approaches for patients with genetic and infectious diseases and cancer. Dr. Kiem has extensive experience training students and postdoctoral fellows and has mentored more than 50 trainees in his lab over the past 20 years. Many of his trainees now hold tenured faculty positions in the US and in Germany. Dr. Kiem is the Sponsor of 4 clinical gene therapy studies (HIV, glioblastoma, and Fanconi anemia) and he is the PI or Co-PI of many R01 or P01 grants including a Martin Delaney Consortium grant to study HIV cure strategies (defeatHIV). Dr. Kiem has also served on the NIH Recombinant DNA Advisory Committee (RAC) for 5 years and the last year as Chair. He is currently the Chair of the Stem Cell Committees for both the American Society for Gene and Cell Therapy (ASGCT) and the American Society of Hematology.
The current research in our laboratory focuses on studies to:
Understand basic hematopoietic stem cell (HSC) and transplantation biology and clonal composition of hematopoiesis after transplantation, especially the clonality of gene-modified HSCs
Develop and evaluate 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 and infectious diseases and cancer.
Develop novel ways to derive HSCs from induced pluripotent stem cells (iPSCs) and to expand HSCs to facilitate gene therapy and stem cell transplantation
Develop clinical gene therapy protocols for genetic and acquired diseases, including cancer. Current target diseases include Fanconi anemia, severe combined immunodeficiency, hemoglobinopathies, glioblastoma, and HIV
Develop less toxic hematopoietic cell transplantation protocols especially for patients with nonmalignant diseases
FA Research Projects
The Latest
News & Events
I am a poet, a writer, and a storyteller. I’m also a student, and an older brother to a young man with FA and special needs. I’m a DACA recipient. I’m here. I’m present. I am more than my current mental state, and more than this diagnosis.
People with rare diseases like FA often rely on a combination of healthcare programs and insurance coverage. Any change that reduces access to care or increases out-of-pocket costs can have real-world consequences for our community.