Welcome to the Department of Pediatrics Research home page. Pediatric research, from molecular biology to clinical trials, is an important and exciting component of the Department‘s activity. With approx. $10M of research grants and contracts awarded in 2006, $6M in federal funding, the Department is one of the most research-active Pediatrics Departments in the United States.

Specific areas of research that attract national attention include cardiology, neurology, weight regulation, metabolism, oncology and stem cell research. The Department is also a leader in Child Health outcome research and an integral partner in the new OCTRI, one of 12 NIH-funded centers in the nation to promote translational research.

Our goal is to support all research that may improve the lives of our patients and the quality of the care we provide to them. We are particularly committed to supporting clinical scientists who have the potential to reconcile bench and bedside research, and further enhance DOP leadership in clinical and translational research (make the words "clinical and translational research" the link to the NIH website and delete "NIH translational site research.") At the same time, we are committed to continuing to support basic science research. After all, it is advancements in basic research that lead to new breakthroughs in clinical and translational research.

We encourage all interested in Pediatric research to explore this site and contact me and/or our research teams for further information on the exciting projects that are conducted by DOP scientists.

Markus Grompe, M.D.

Dr. Grompe received his medical degree (Dr. med.) in 1983 at the University of Ulm Medical School in Germany. His interest in Human Genetics started with a one year research project abroad during medical school. From 1984-1987 Dr. Grompe was trained in Pediatrics at Oregon Health Sciences University in Portland, Oregon, USA and then moved to Baylor College of Medicine in Houston, Texas. There he was a fellow sponsored by the Pediatric Scientist Training Program in the Institute for Molecular Genetics from 1987-1991 and worked on gene therapy for inherited diseases, particularly metabolic liver disorders. In 1991, Dr. Grompe joined the faculty at Oregon Health & Sciences University and he is currently Professor in the Departments of Molecular and Medical Genetics and Pediatrics. He is a recipient of the E. Mead Johnson award for pediatric research and the Merit Award of the Fanconi Anemia Research Foundation. He is involved in the clinical care of patients with genetic diseases as well as scientific investigation. In 2004 he became the first director of the newly founded Oregon Stem Cell Center. Since 2008 he is the holder of the Ray Hickey Chair and the Papé Family Pediatric Research Institute.
His research has focussed on the use of in vivo selection to enhance gene and cell therapy, particularly stem cell therapy. The two model diseases being studied are hereditary tyrosinemia type 1, a childhood liver disease and Fanconi Anemia (FA), a blood disorder. In 1996, his laboratory showed that gene therapy in combination in vivo selection could be used to replace > 90% of cells in a diseased mouse liver. Since then, his work has focussed on the biology of intra- and extrahepatic liver stem cells and their use in therapeutic liver repopulation. Recently, his group developed a method to replace the cells of mouse livers with human hepatocytes.
The Fanconi anemia pathway is involved in the maintenance of genomic stability and stem cell integrity in mammals. The Grompe Lab cloned a novel Fanconi Anemia gene, FANCD2, which has been shown to link the Fanconi Anemia pathway to BRCA1 and BRCA2, genes involved in familial breast cancer. Preclinical gene therapy experiments in murine models of FA have been used to demonstrate in vivo selection at the level of hematopoietic stem cells. Recently, the Grompe lab has discovered small molecules to prevent solid tumors in this disease.
For a complete list of scientific publications please click here.

Robert D. Steiner, M.D., FAAP, FACMG, joined OHSU in 1995 as Head of the Division of Pediatric Metabolism. He is currently Professor of Pediatrics and Vice Chair for Research in the Department of Pediatrics and has a joint appointment in the Department of Molecular and Medical Genetics.

Dr. Steiner is an internationally-recognized clinician and researcher in inborn errors of metabolism. In 1993 he was a member of a team of scientists who discovered the genetic cause of Smith Lemli Opitz Syndrome, a condition in which children are unable to make cholesterol. In March 2006, Dr. Steiner initiated the first-ever clinical trial to explore the safety and efficacy of injecting purified human fetal neural stem cells into the brain in an attempt to treat two forms of neuronal ceroid lipofuscinosis (NCL).

Dr. Steiner was instrumental in bringing together pediatric and child health clinical and translational research on campus to help OHSU win 1 of the first 12 prestigious NIH-funded Clinical and Translational Science Awards (CTSA) in the country. He is currently serving as 1 of 3 OHSU CTSA (OCTRI) Deputy Directors.