The UW/FHCRC Program of Excellence in Gene Therapy. The goal of this application from a Program of Excellence in Gene Therapy is to combine the resources of two Institutions, the University of Washington (UW) and the Fred Hutchinson Cancer Research Center (FHCRC), to advance gene therapy in two areas of interest of NHLBI-stem cell gene therapy of hematological disorders and gene therapy of two common inherited lung diseases. The program puts together an outstanding group of gene therapy investigators and utilizes several resources of the two Institutions. The PEGT is composed of six projects, four core units, a training program and a Coordinating and Date Core Unit. Two projects propose preclinical studies on new therapy strategies. Four projects plan critical trials. Project 1 focuses on the development of new gene therapy approaches for hemophilia A and alpha-1 anti-trypsin deficiency. 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. Project 3 is a clinical project on gene therapy of Cystic Fibrosis Project 4 clinical studies on a novel inducible suicide gene based on the induction of apoptosis through oligimerization of the human Fas protein. Project 5 proposes preclinical and clinical studies of gene therapy of sickle cell disease and beta thalassemia. Project 6 is a clinical trial in patients with Fanconi anemia. Four core units support the research of the projects. Core A is a clinical core. Core B is a hemopoietic Cell Procurement and Processing Resource. Core Unit C is a primate stem cell transplantation core. Core D is the administrative core unit of the PEGT. The Hemopoietic Cell Procurement and Processing Core Unit and the Primate Transplantation Core Unit are proposed as core facilities for all the PEGT and NHLBI investigators.
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