CORE A: Vector Core Core Director: Julie Johnston, Ph.D. The Vector Core is a critical resource for University of Pennsylvania (UPenn) and other investigators requiring viral based vectors for basic and translational studies for the development of gene therapies for acquired and inherited disease. Since its establishment, one of the main objectives of the Core has been to provide advanced vector technologies to facilitate the translation of gene therapy research into clinical treatment for cystic fibrosis (CF). The Vector Core has enjoyed a long-standing relationship with the CF research community, providing services to NIDDK P30 Molecular Therapy and CF center members for over 20 years (1993?present), supporting CF Foundation researchers for 18 years (1992?2011) and serving as the NHLBI Gene Therapy Resource Program (GTRP) Preclinical Core since its inception in 2007. During this time, the Core has offered novel serotype AAV vectors, variously pseudotyped lentiviral vectors and adenoviral vectors for the development of gene therapies for CF and other lung disorders. The Core has also maintained close ties to key laboratories involved in the development of next generation vectors and acts as the distribution center for novel materials to the research community. In support of this RFA, the Core will generate research grade, GMP process comparable grade and GMP/clinical grade vectors (the latter to be offered in May 2015) to support basic and clinical CF research by center members located in one of 3 institutions/1 consortium (University of Pennsylvania, University of Massachusetts, Johns Hopkins, UK Gene Therapy CF Consortium) comprising our multi-site CF center.

National Institute of Health (NIH)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Center Core Grants (P30)
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Special Emphasis Panel (ZDK1)
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University of Pennsylvania
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