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.
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