Gene therapy researcher selected as Howard Hughes Medical Institute investigator

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 Univer

Contact: John Ascenzi
Children's Hospital of Philadelphia

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