Browse Institute for Stem Cell Biology and Regenerative Medicine

BioDr. Yusuke Nakauchi is a scientist specializing in stem cell biology, focusing on normal hematopoietic stem cells (HSCs), pre-leukemic HSCs, and leukemia stem cells (LSCs).

From 2005 to 2014, he trained and practiced as a clinical hematologist in Japan, where he witnessed allogeneic hematopoietic stem cell transplantation (HSCT) cure patients with otherwise fatal blood cancers—and saw many others suffer long-term complications from graft-versus-host disease (GVHD). Having saved many patients, yet lost others he could not save, he became convinced that overcoming the limitations of leukemia treatment would require a deeper understanding of both normal HSCs and the LSCs responsible for relapse.

This conviction led him to pursue basic research. In 2010, he began his PhD in the laboratory of Professor Hiromitsu Nakauchi at the University of Tokyo, where he developed allele-specific anti-HLA monoclonal antibodies as a novel therapeutic strategy for GVHD—the first work to demonstrate that anti-HLA antibodies could be used therapeutically in HLA-mismatched transplantation. This research also revealed the importance of detailed chimerism analysis for understanding early post-transplant changes in a patient's blood and HSCs, a theme he later continued at Stanford. As a graduate student, he also contributed to work on iPSC-derived T cell therapy and a large-scale single-cell study of HSC aging.

In 2014, building on this clinical and research foundation, Dr. Nakauchi joined Professor Ravindra Majeti's laboratory at Stanford University as a postdoctoral fellow. His research there centered on TET2, one of the most frequently mutated genes in acute myeloid leukemia (AML) and clonal hematopoiesis. Using CRISPR/Cas9 editing in human hematopoietic stem and progenitor cells, he developed a tractable model of TET2-mutant pre-leukemia, showing how TET2 loss reshapes epigenetic and transcriptional programs to drive clonal expansion—and how azacitidine plus vitamin C could restore normal function in TET2-mutant cells. His work now extends to antibody-based approaches to GVHD, single-cell analysis of CMML stem cells, and advanced in vivo mouse models, pursued through collaborations across and beyond Stanford.

Dr. Nakauchi's long-term goal is to bring personalized medicine to patients with AML and other blood cancers, believing that comparing normal HSCs, pre-leukemic HSCs, and LSCs at single-cell resolution is key to understanding—and ultimately preventing—leukemia. Drawing on years as a clinician who witnessed many patients lose their lives to AML, he is driven by the conviction that discoveries in stem cell biology will one day help cure and prevent the disease.