Abstract

Animal Models Core The Gene Therapy Program has access to animal facilities of importance to this P01. Dr. Wilson has managed mouse and nonhuman primate vivaria at Penn for the last 14 years. These facilities are state-ofthe- art, are operated under BSL2 and are AAALAC accredited. The University Laboratory Animal Resources (ULAR) provides clinical veterinarian support to both vivaria. The nonhuman primate facility is dedicated to Dr. Wilson's use, while the mouse vivarium was developed for the use of those faculty involved in gene therapy research. Dr. Claire Hankenson, (Assistant Professor of Laboratory Animal Medicine, Dept of Pathobiology and Senior Associate Director for Regulatory Affairs and Planning, ULAR) will serve as Director of the Core. The mouse oncogenicity study will be performed in a dedicated room within the mouse facility. All nonhuman primate experiments will be implemented by the Core with biological samples being provided to the Projects and other Cores. Lay description. A critical step in the pre-clinical development of gene therapy is the evaluation of the safety and efficacy of gene transfer in monkeys. This Core will perform these studies on behalf of the projects.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Type
Research Program Projects (P01)
Project #
5P01HD057247-02
Application #
7802301
Study Section
Special Emphasis Panel (ZHD1)
Project Start
2009-04-01
Project End
2012-03-31
Budget Start
2009-04-01
Budget End
2010-03-31
Support Year
2
Fiscal Year
2009
Total Cost
$140,042
Indirect Cost

  Institution

Name
University of Pennsylvania
Department
Type
DUNS #
042250712
City
Philadelphia
State
PA
Country
United States
Zip Code
19104

  Publications

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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