The UW/FHCRC Program of Excellence in Gene Therapy. The goal of this competing application for 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 to NHLBI - stem cell gene therapy of hematological disorders and gene therapy of cystic fibrosis. The program puts together an outstanding group of gene therapy investigators and utilizes several resources of the two Institutions. The PEGT is composed of four clinical and projects, and three core units. Project by Miller focuses on the development of new gene therapy approaches for cystic fibrosis. The objective of Project by Stamatoyannopoulos is to develop gene therapy for beta thalassemia major. Project by Riddel proposes clinical studies on suicide gene-based induction of apoptosis through oligimerization of the human Fas protein. Project by Kiem is developing gene therapy for patients with Fanconi anemia. Core 9001 is the clinical core. Core 9002 is a National Core for hemopoietic cell procurement and processing. There is also the administration core of the PEGT.

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Research Project--Cooperative Agreements (U01)
Project #
2U01HL066947-06
Application #
6964571
Study Section
Special Emphasis Panel (ZHL1-CSR-I (M1))
Program Officer
Evans, Gregory
Project Start
2000-09-28
Project End
2010-08-31
Budget Start
2005-09-29
Budget End
2010-08-31
Support Year
6
Fiscal Year
2005
Total Cost
$2,348,469
Indirect Cost
Name
University of Washington
Department
Internal Medicine/Medicine
Type
Schools of Medicine
DUNS #
605799469
City
Seattle
State
WA
Country
United States
Zip Code
98195
Yannaki, Evangelia; Papayannopoulou, Thalia; Jonlin, Erica et al. (2012) Hematopoietic stem cell mobilization for gene therapy of adult patients with severe ?-thalassemia: results of clinical trials using G-CSF or plerixafor in splenectomized and nonsplenectomized subjects. Mol Ther 20:230-8
Yannaki, Evangelia; Stamatoyannopoulos, George (2010) Hematopoietic stem cell mobilization strategies for gene therapy of beta thalassemia and sickle cell disease. Ann N Y Acad Sci 1202:59-63
Kiem, Hans-Peter; Ironside, Christina; Beard, Brian C et al. (2010) A retroviral vector common integration site between leupaxin and zinc finger protein 91 (ZFP91) observed in baboon hematopoietic repopulating cells. Exp Hematol 38:819-22, 822.e1-3
Yannaki, Evangelia; Emery, David W; Stamatoyannopoulos, George (2010) Gene therapy for ?-thalassaemia: the continuing challenge. Expert Rev Mol Med 12:e31
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Berger, Carolina; Jensen, Michael C; Lansdorp, Peter M et al. (2008) Adoptive transfer of effector CD8+ T cells derived from central memory cells establishes persistent T cell memory in primates. J Clin Invest 118:294-305
Stirewalt, Derek L; Meshinchi, Soheil; Kopecky, Kenneth J et al. (2008) Identification of genes with abnormal expression changes in acute myeloid leukemia. Genes Chromosomes Cancer 47:8-20
Beard, Brian C; Dickerson, David; Beebe, Kate et al. (2007) Comparison of HIV-derived lentiviral and MLV-based gammaretroviral vector integration sites in primate repopulating cells. Mol Ther 15:1356-65
Halbert, Christine L; Lam, Siu-Ling; Miller, A Dusty (2007) High-efficiency promoter-dependent transduction by adeno-associated virus type 6 vectors in mouse lung. Hum Gene Ther 18:344-54
Berger, Carolina; Berger, Michael; Feng, Junli et al. (2007) Genetic modification of T cells for immunotherapy. Expert Opin Biol Ther 7:1167-82

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