The Animal Models Core will provide technical support to Projects I, II and III to allow for the evaluation of various in vivo strategies for adeno-associated virus-based gene therapy for ornithine transcarbamylase deficiency (OTCD). The Gene Therapy Program at the University of Pennsylvania has extensive experience with the in vivo characterization of vectors in various murine models of OTCD. In the context of this P01 application, the Animal Models Core will provide full support at all levels of the in vivo gene transfer studies including study design;animal acquisition;breeding of OTC and FRG mice, management of in-life phase of studies including organization of the study, technical support for procedures and harvesting of samples;and necropsy. The Core will maintain four colonies that include the spf[ash], spf, OTC-KO and FRG mice to support the experimental needs of Projects I, II and III.

Public Health Relevance

The Animal Models Core is pivotal to the success of this POI. The Core will expand and maintain the spf, spf[ash], OTC and FRG mouse colonies that are critical for the experiments of all three projects of this program The Core has the technical expertise to perform all animal-related experiments that have been designed to address the barriers to effective gene transfer in humans in an effort to bring AAVS to the clinic.

National Institute of Health (NIH)
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Research Program Projects (P01)
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Pediatrics Subcommittee (CHHD)
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University of Pennsylvania
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