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ASH to Honor Esteemed Gene Therapy Researcher

September 1, 2013

Distinguished gene therapy researcher Katherine A. High, MD, will be honored by the American Society of Hematology (ASH) with the 2013 E. Donnall Thomas Lecture and Prize for her efforts to uncover the molecular workings of hemophilia and to create new genetic-based therapies to treat it. High is a Howard Hughes Medical Institute Investigator, the William H. Bennett Professor of Pediatrics at the University of Pennsylvania School of Medicine, and an attending physician at the Children's Hospital of Philadelphia.

High will give her lecture, "Sailing to Ithaca: Gene Therapy's Odyssey from Investigational Agent to Therapeutic Product," at the 55th ASH Annual Meeting and Exposition on December 9, 2013, in New Orleans. The talk will encompass the use of gene therapies for the treatment of inherited disorders, including a retrospective on the therapeutic successes and persistent challenges of laboratory and small-scale clinical studies conducted over the last two decades. High will touch on several aspects of gene therapy development and science.

High will also highlight our enhanced understanding of adeno-associated virus (AAVs) vectors and the nuances inherent in their interactions with the human immune system. In gene therapy, AAVs are employed as delivery vehicles, or vectors, to carry the actual genetic material that triggers a desired therapeutic response, such as the production of factor IX in hemophilia B patients. Investigators now often opt to use AAVs because they can deliver genetic material into living cells to sustain therapeutic effect without causing disease and with less risk for triggering an immune response.

"Dr. High is unquestionably a leader in gene therapy and has made remarkable, ground-breaking contributions to the field, demonstrating creativity, determination, and the ability to overcome numerous obstacles," said ASH President Janis L. Abkowitz, MD, of the University of Washington. "Through her countless discoveries, Dr. High has transformed the notion of utilizing genetically engineered mechanisms for treatment of incurable inherited disorders from a distant vision to reality."

ASH is the world's largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment and prevention of disorders affecting the blood. For more than 50 years, ASH has led the development of hematology as a discipline by promoting research, patient care, education, training and advocacy in hematology.


Source:, August 14, 2013