The success of somatic gene therapy is largely dependent on the development of gene transfer vehicles that can safely and efficiently deliver their genetic payload to target cells. It is of equal importance that investigators interested in evaluating new and established gene transfer techniques have at their disposal both properly equipped facilities and the guidance of scientists trained in gene therapy research. The vector core facility was born out of this understanding and is committed to meet the challenges presented by somatic gene transfer. While the primary goal of the core facility is to provide investigators with the methods and materials necessary to establish viral-directed gene therapy protocols for cystic fibrosis and other inherited disorders, a significant effort is reserved for research and development. This latter component will primarily focus on establishing AAV as a tool for somatic gene transfer. Housed within the Wistar Institute, the core facility occupies 1250 sq ft of independent laboratory space with direct ties to the main IHGT laboratory. The central location of the core relative to the University of Pennsylvania campus ensures easy access by investigators from various disciplines and academic Departments. Specific services are listed below: (1) Provide expert consultation and guidance of interested investigators on the use of recombinant viruses for targeted transgene delivery; (2) Instruct the investigator on the theoretical and technical aspects of vector construction, virus production and functional analysis; (3) Assemble, organized, and make available libraries of vectors and recombinant viruses with relevant supporting information; (4) Provide investigators participating in the center access to BL2+ facilities for experiments involving recombinant viruses; (5) Perform simple repetitive analyses such as plaque assays for adenoviruses or helper virus assays for retroviruses and adenoviruses; (6) When appropriate provide a comprehensive service for vector/virus production, including vector construction, virus production, and functional analyses.

Project Start
Project End
Budget Start
Budget End
Support Year
Fiscal Year
Total Cost
Indirect Cost
University of Pennsylvania
United States
Zip Code
Bals, R; Weiner, D J; Meegalla, R L et al. (2001) Salt-independent abnormality of antimicrobial activity in cystic fibrosis airway surface fluid. Am J Respir Cell Mol Biol 25:21-5
Gao, G P; Engdahl, R K; Wilson, J M (2000) A cell line for high-yield production of E1-deleted adenovirus vectors without the emergence of replication-competent virus. Hum Gene Ther 11:213-9
Chirmule, N; Propert, K; Magosin, S et al. (1999) Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther 6:1574-83
Zuckerman, J B; Robinson, C B; McCoy, K S et al. (1999) A phase I study of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator gene to a lung segment of individuals with cystic fibrosis. Hum Gene Ther 10:2973-85
Bals, R; Weiner, D J; Moscioni, A D et al. (1999) Augmentation of innate host defense by expression of a cathelicidin antimicrobial peptide. Infect Immun 67:6084-9
Chirmule, N; Truneh, A; Haecker, S E et al. (1999) Repeated administration of adenoviral vectors in lungs of human CD4 transgenic mice treated with a nondepleting CD4 antibody. J Immunol 163:448-55
Jiang, Q; Mak, D; Devidas, S et al. (1998) Cystic fibrosis transmembrane conductance regulator-associated ATP release is controlled by a chloride sensor. J Cell Biol 143:645-57
Jooss, K; Turka, L A; Wilson, J M (1998) Blunting of immune responses to adenoviral vectors in mouse liver and lung with CTLA4Ig. Gene Ther 5:309-19
Chirmule, N; Hughes, J V; Gao, G P et al. (1998) Role of E4 in eliciting CD4 T-cell and B-cell responses to adenovirus vectors delivered to murine and nonhuman primate lungs. J Virol 72:6138-45
Goldman, M J; Lee, P S; Yang, J S et al. (1997) Lentiviral vectors for gene therapy of cystic fibrosis. Hum Gene Ther 8:2261-8

Showing the most recent 10 out of 12 publications