Translational and Functional Genomics Branch

Paul P. Liu, M.D., Ph.D.

Paul P. Liu
Senior Investigator
Translational and Functional Genomics Branch

Oncogenesis and Development Section

Deputy Scientific Director
Office of the Scientific Director

M.D. Capital Institute of Medicine, Beijing, China, 1982
Ph.D. University of Texas, 1991

phone (301) 402-2529
fax (301) 402-4929
Building 49, Room 3A26
BETHESDA, MD 20892-4442

Selected Publications

Dr. Liu's laboratory investigates the molecular mechanisms of leukemia, a cancer of blood cells. His group has a particular interest in the genetic control of hematopoiesis, or blood formation, through which hematopoietic stem cells differentiate into all mature blood cells in the circulatory system. Leukemia is an example of hematopoiesis gone awry. When it develops, the body produces large numbers of abnormal blood cells, or leukemia cells.

Leukemias are frequently associated with chromosome abnormalities such as translocations, inversions and deletions. One form of acute myeloid leukemia (AML) is associated with an inversion of chromosome 16. Dr. Liu's laboratory found that this inversion generates a fusion gene between the CBFB and the MYH11 genes.

To study CBFB-MYH11, Dr. Liu's group generated transgenic mouse models. Using these models, they demonstrated that the fusion gene blocks normal hematopoiesis, leading to AML susceptibility. In addition, they have identified CBFB-MYH11 target genes during leukemia development. His group was able to identify and study leukemia initiating cells, which may be responsible for relapse after treatments. 

Moreover, Dr. Liu's group is developing and testing novel anti-leukemia compounds that may lead to targeted therapy in the future. Dr. Liu's group also uses zebrafish as a model to study hematopoiesis, with genetic and genomic approaches. They use zebrafish to study the hematopoietic roles of transcription factor genes that are important for leukemia. Such studies enhance our understanding of leukemia.

Finally, Dr. Liu's group has an interest in modeling human diseases using induced pluripotent stem cells or iPSCs, with the hope of studying disease mechanisms and developing cell-based therapy. The primary focus of the iPSC studies has been the familial platelet disorder, a disease manifested by impaired platelet development and function, as well as increased risk of leukemia.


Dr. Liu received his medical degree and residency training in internal medicine in Beijing, China.  He then earned his Ph.D. in human genetics from the University of Texas M.D. Anderson Cancer Center in Houston. He received his postdoctoral research training at the University of Michigan before moving in 1993 to NIH's National Center for Human Genome Research, renamed the National Human Genome Research Institute (NHGRI) in 1995. 

He has remained at NHGRI - appointed initially as a senior staff fellow, then as a tenure track investigator and since 2001 as a tenured senior investigator. Dr. Liu has been the head of the Oncogenesis and Development Section in NHGRI since 1995. In May 2011, Dr. Liu was appointed as the deputy scientific director of NHGRI. The main focus of Dr. Liu's research over the years has been the mechanism of leukemia development at the molecular level, using genetic and genomic approaches.

Dr. Liu discovered that a CBFB-MYH11 fusion gene is the product of chromosome 16 inversion, a common chromosome abnormality in human acute myeloid leukemia. Using animal models, Dr. Liu has illustrated the importance of CBFB-MYH11 for leukemia development, and how this fusion gene works. His group has also made important discoveries regarding the normal functions of several important leukemia genes.

More recently, Dr. Liu's lab has focused on developing targeted treatments for leukemia. Dr. Liu has received several honors for his achievements, including NIH Director's Award and elections to the American Society for Clinical Investigation and the Association of American Physicians.

Scientific Summary

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Last Updated: January 6, 2015