In 1989, Lynn and David Frohnmayer founded the Fanconi Anemia Research Fund (FARF), determined and desperate to find a cure for their three daughters diagnosed with Fanconi anemia (FA). Since then, their vision has grown into a vibrant community of FA families, researchers, clinicians, donors, fundraisers, staff, and volunteers, all dedicated to improving outcomes for people with FA.
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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
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World-Renown Experts Take On the Root of the Problem The long-awaited genetic revolution for rare genetic diseases has arrived and with it, the potential to cure diseases like FA within our lifetimes using state-of-the-art gene therapy (gene replacement) and gene...
My name is Blue Mohr and I’m a 27-year-old living with Fanconi anemia (FA). I’m from Austin, Texas, though I am currently living in Washington, D.C. where I’m pursuing a Master of Public Health degree from the George Washington University with concentrations in epidemiology, cancer, and public health communication and marketing.