Breakthrough Gene Therapy Treats Hemophilia B
Interview: Katherine A. High, MD,
professor of pediatrics, University of Pennsylvania School of Medicine
director, Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia
Investigator, Howard Hughes Medical Institute
Hemophilia is a genetic disorder in which the patientís blood lacks the all-important clotting factor.
Physician and hemophilia researcher Katherine A. High, MD, chats with Sound Medicineís Dr. David Crabb about the encouraging results of a recent study that injected patients with the correct form of the defective gene.
It may be the first instance of gene therapy successfully used to treat a well-known disease.
Dr. High is a professor of pediatrics at the University of Pennsylvania School of Medicine and director of the Center for Cellular and Molecular Therapeutics at Children's Hospital of Philadelphia. In addition, she is an investigator with Howard Hughes Medical Institute.