David A. Williams, MD




blood formation

blood stem cell biology

Fanconi anemia and bone marrow failure

gene therapy

genetic blood disorders

Clinical Titles

Chief, Division of Hematology/Oncology

Director of Translational Research

Academic Appointment

Leland Fikes Professor of Pediatrics, Harvard Medical School

Board Certification(s)



Medical/Dental School
Medicine, Indiana University School of Medicine, Indianapolis, IN (1979)


Pediatrics, Cincinnati's Children's Hospital Medical Center, Cincinnati, OH (1980)


Pediatrics, Cincinnati's Children's Hospital Medical Center, Cincinnati, OH (1982)


Hematology/Oncology, Children's Hospital Boston/Dana-Farber Cancer Inst, Boston, MA (1985)


Dr. Williams major research interests are in the study of blood stem cell biology, blood formation, leukemia and the treatment of genetic blood diseases using gene therapy. He has published over 250 peer-reviewed manuscripts and textbook chapters. He was an investigator of the HHMI for 16 years. He has been continuously funded by the NIH since 1986. His laboratory co-discovered and cloned Interleukin-11 (Neumega TM) and carried out developmental studies that assisted in obtaining FDA approval of this drug. His laboratory also described and patented the use of fibronectin in retrovirus-mediated gene transfer, now nearly universally used (as Retronectin TM) in clinical gene therapy trials and basic research utilizing gene transfer. His most recent focus has been on exploring the role of Rho GTPases in hematopoietic stem cell biology. He and Dr. Yi Zheng have developed a new small molecule inhibitor of Rac GTPases that is being investigated collaboratively in cancer therapies. Thus, work in the Williams laboratory has led to multiple patents and product licenses for new therapies in human diseases. He also leads several gene therapy trials in immunodeficiency and neurologic disease.