Abstract

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 stateof- the-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. The Core will also provide support for all mouse procedures and maintenance of all breeding colonies.

Public Health Relevance

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
National Heart, Lung, and Blood Institute (NHLBI)
Type
Research Program Projects (P01)
Project #
5P01HL059407-14
Application #
8378204
Study Section
Heart, Lung, and Blood Initial Review Group (HLBP)
Project Start
Project End
Budget Start
2012-07-01
Budget End
2013-06-30
Support Year
14
Fiscal Year
2012
Total Cost
$219,723
Indirect Cost
$81,452

  Institution

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

  Publications

Giles, April R; Govindasamy, Lakshmanan; Somanathan, Suryanarayan et al. (2018) Mapping an Adeno-associated Virus 9-Specific Neutralizing Epitope To Develop Next-Generation Gene Delivery Vectors. J Virol 92:
Amengual, Jaume; Guo, Liang; Strong, Alanna et al. (2018) Autophagy Is Required for Sortilin-Mediated Degradation of Apolipoprotein B100. Circ Res 122:568-582
Giles, April R; Sims, Joshua J; Turner, Kevin B et al. (2018) Deamidation of Amino Acids on the Surface of Adeno-Associated Virus Capsids Leads to Charge Heterogeneity and Altered Vector Function. Mol Ther 26:2848-2862
Calcedo, Roberto; Somanathan, Suryanarayan; Qin, Qiuyue et al. (2017) Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for ?-1-antitrypsin deficiency. Proc Natl Acad Sci U S A 114:1655-1659
Ai, Jianzhong; Li, Jia; Gessler, Dominic J et al. (2017) Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery. Sci Rep 7:40336
Ai, Jianzhong; Tai, Phillip W L; Lu, Yi et al. (2017) Characterization of adenoviral transduction profile in prostate cancer cells and normal prostate tissue. Prostate 77:1265-1270
Greig, Jenny A; Limberis, Maria P; Bell, Peter et al. (2017) Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR+/- Rhesus Macaques. Hum Gene Ther Clin Dev 28:39-50
Greig, Jenny A; Limberis, Maria P; Bell, Peter et al. (2017) Nonclinical Pharmacology/Toxicology Study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a Mouse Model of Homozygous Familial Hypercholesterolemia. Hum Gene Ther Clin Dev 28:28-38
Ajufo, Ezim; Cuchel, Marina (2016) Recent Developments in Gene Therapy for Homozygous Familial Hypercholesterolemia. Curr Atheroscler Rep 18:22
Ibrahim, Salam; Somanathan, Suryanarayan; Billheimer, Jeffrey et al. (2016) Stable liver-specific expression of human IDOL in humanized mice raises plasma cholesterol. Cardiovasc Res 110:23-9

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