Public Release: 

Gene therapy researcher selected as Howard Hughes Medical Institute investigator

Dr. Katherine High, of The Children's Hospital of Philadelphia, chosen on basis of patient-oriented research

Children's Hospital of Philadelphia

Philadelphia, Pa. - Katherine A. High, M.D., director of hematology research at The Children's Hospital of Philadelphia, has been named a Howard Hughes Medical Institute (HHMI) investigator. Dr. High is one of 12 physician-scientists recently selected by the prestigious research organization for major accomplishments in patient-oriented research.

"We are extremely pleased and proud of the fact that one of our physicians was honored by one of the world's leading biomedical research institutions," said Steven M. Altschuler, M.D., president and chief executive officer of The Children's Hospital of Philadelphia. "This appointment recognizes Dr. High's pioneering achievements in advancing gene therapy, a novel field of medicine."

Dr. High is internationally prominent for her studies of the molecular biology of the inherited bleeding disorder hemophilia. Over the past decade, she has investigated a gene transfer approach to treating hemophilia B, the form of hemophilia caused by a deficiency of blood clotting factor IX. That approach holds the potential of treating human disease at a fundamental level, by delivering therapeutic genes directly into a patient's cells.

In 1999, Dr. High's research team showed that gene therapy could achieve long-term improvement in dogs having naturally occurring hemophilia. Based on these studies, she and her collaborators have undertaken human gene therapy trials seeking to improve blood clotting in patients with severe hemophilia B. Even small increases in clotting factor in a patient's blood can improve hemophilia from a severe form to a much milder form, and result in great improvements in quality of life.

As an attending hematologist at Children's Hospital, Dr. High will continue to work at the Hospital and will become an employee of HHMI, which will provide a research budget of up to $1 million per year, plus funding for laboratory space. Dr. High will remain a professor of Pediatrics at the University of Pennsylvania School of Medicine.

After graduating from Harvard College and the University of North Carolina School of Medicine, Dr. High completed her residency at North Carolina Memorial Hospital, followed by a fellowship in hematology at Yale University School of Medicine. She was on the medical staff of Yale-New Haven Hospital, and then directed the Clinical Coagulation Laboratory at the University of North Carolina Hospitals before coming to The Children's Hospital of Philadelphia in 1992. She leads an NIH-funded laboratory and is active in many scientific societies, particularly the American Society of Hematology, the American Society for Gene Therapy and the International Society on Thrombosis and Hemostasis.


Established in 1953, the Howard Hughes Medical Institute is a nonprofit medical research organization that currently supports approximately 325 investigators throughout the United States. Based in Chevy Chase, Maryland, the Institute conducts medical research and supports science education in the United States and biomedical scientists in other countries. The most recent group of 12 investigators was chosen because their combination of scientific expertise and medical training holds great potential for translating basic science discoveries into useful medical treatments.

Founded in 1855 as the nation's first pediatric hospital, The Children's Hospital of Philadelphia is ranked today as the best pediatric hospital in the nation by a comprehensive Child magazine survey. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals and pioneering major research initiatives, Children's Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country, ranking second in National Institutes of Health funding. In addition, its unique family-centered care and public service programs have brought the 381-bed hospital recognition as a leading advocate for children and adolescents from before birth through age 19. For more information, visit

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