UW/FHCRC Program of Excellence in Gene Therapy - Overview
    - George Stamatoyannopoulos, MD, DrSci

The Program of Excellence in Gene Therapy (PEGT) at the University of Washington combines resources with the Fred Hutchinson Cancer Research Center to advance gene therapy of hematological disorders and gene therapy for two common inherited lung diseases. It is composed of five projects (two preclinical and three clinical), four core units, and a training program. Project 1 focuses on the development of new gene therapy approaches for hemophilia A and a-1 antitrypsin deficiency (preclinical). The objective of Project 2 is to develop gene targeting methods that can be used to correct genes in cells capable of reconstituting the hemopoietic system (preclinical). Project 3 is a clinical project on gene therapy of Cystic Fibrosis using AAV vectors. Project 4 conducts clinical studies on a novel inducible suicide gene based on the induction of apoptosis through oligimerization of the human Fas protein. Project 5 includes preclinical and clinical studies of gene therapy of sickle cell disease and b-thalassemia. Four core units support the research of the projects and include a clinical core, a hemopoietic cell procurement and processing resource core, a primate stem cell transplantation core, and an administrative core. The hemopoietic cell procurement and processing core unit and the primate transplantation core unit are core facilities for all of the PEGT and NHLBI investigators. As part of the administrative core, the training program trains scientists for successful, independent careers in gene therapy through formal courses, research seminars, participatory activities, laboratory and clinical research, and submission of applications for fellowship funding. 

- Project 1 - New Vectors for Gene Therapy of Hemophilia A and a-1 Antitrypsin Deficiency. A. Lieber

- Project 2 - Gene Targeting Approaches for Blood Diseases. D. W. Russell

- Project 3 -Gene Therapy for Cystic Fibrosis Using AAV Vectors. A. D. Miller

- Project 4 - A Novel Suicide Gene for Therapy of Graft Versus Host Disease. S. R. Riddell

- Project 5 - Pre-Clinical and Clinical Studies on Gene Therapy of Sickle Cell Disease and b -thalassemia. G. Stamatoyannopoulos

- National Service Cores: