Vector Core The Vector Core at the University of Pennsylvania is an independent and full service laboratory providing reagents and expertise in the area of gene transfer. This facility called """"""""Penn Vector"""""""" is directed by Dr. Julie Johnston who manages 7 scientists in the area of AAV, adenovirus and lentiviral production. This program will focus on utilization of Penn vector for the production of AAV vectors that will service Projects I through III An independent Quality Control Program directed by Dr. Martin Lock performs a series of assays necessary for release of the vector for in vivo studies. This core will also analyze tissues for presence of vector genomes using TaqMan and PCR technology. Finally, the Vector Core will adapt existing methods of manufacturing and purification to the clinical candidate for a large scale production efficient enough to support large animals in a Phase 1 study. Assays necessary to support the ultimate clinical production will also be developed. The laboratory operates under the general principles of GLP including, documentation (batch record forms), SOPs, QA/QC, training and equipment validation, although it does not represent itself as being fully GLP or GMP. Lay description. All vectors used in the P01 will be. created and produced by the Vector Core.

Agency
National Institute of Health (NIH)
Institute
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Type
Research Program Projects (P01)
Project #
5P01HD057247-03
Application #
8056520
Study Section
Special Emphasis Panel (ZHD1)
Project Start
Project End
Budget Start
2010-04-01
Budget End
2011-03-31
Support Year
3
Fiscal Year
2010
Total Cost
$142,616
Indirect Cost
Name
University of Pennsylvania
Department
Type
DUNS #
042250712
City
Philadelphia
State
PA
Country
United States
Zip Code
19104
Ashley, Scott N; Somanathan, Suryanarayan; Hinderer, Christian et al. (2017) Alternative Start Sites Downstream of Non-Sense Mutations Drive Antigen Presentation and Tolerance Induction to C-Terminal Epitopes. J Immunol 198:4581-4587
Wang, Lili; Bell, Peter; Morizono, Hiroki et al. (2017) AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice. Mol Genet Metab 120:299-305
Yang, Yang; Wang, Lili; Bell, Peter et al. (2016) A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice. Nat Biotechnol 34:334-8
Bell, Peter; Wang, Lili; Chen, Shu-Jen et al. (2016) Effects of Self-Complementarity, Codon Optimization, Transgene, and Dose on Liver Transduction with AAV8. Hum Gene Ther Methods 27:228-237
Wang, Lili; Bell, Peter; Somanathan, Suryanarayan et al. (2015) Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids. Mol Ther 23:1877-87
Bissig-Choisat, Beatrice; Wang, Lili; Legras, Xavier et al. (2015) Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model. Nat Commun 6:7339
Mays, Lauren E; Wang, Lili; Lin, Jianping et al. (2014) AAV8 induces tolerance in murine muscle as a result of poor APC transduction, T cell exhaustion, and minimal MHCI upregulation on target cells. Mol Ther 22:28-41
Mikals, Kyle; Nam, Hyun-Joo; Van Vliet, Kim et al. (2014) The structure of AAVrh32.33, a novel gene delivery vector. J Struct Biol 186:308-17
Bryant, Laura M; Christopher, Devin M; Giles, April R et al. (2013) Lessons learned from the clinical development and market authorization of Glybera. Hum Gene Ther Clin Dev 24:55-64
Zhong, Li; Malani, Nirav; Li, Mengxin et al. (2013) Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction. Hum Gene Ther 24:520-5

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