This Core will be directed by Dr. Wilson with Drs. Batshaw and Caplan serving as co-directors. The Core will provide basic administrafive support to manage the financial operations ofthe program project to include oversight of accounts, purchasing, and preparation and submission of reports to the institution, as well as to the NIH. An External Scientific Advisory Committee (ESAC) will be convened, which will include scientists knowledgeable about areas relevant to the grant including immunology, urea cycle disorders, AAV and vector safety assessment. An Ethics Advisory Board (EAB) will also be convened and chaired by Dr. Caplan. This Board will include a scienfist from the ESAC, as well as parents of children with inherited diseases and people with expertise in ethics, policy, and law. The ESAC and EAB will convene on an annual basis to review the progress ofthe science and help the team address important issues related to the ulfimate clinical applications of gene therapy. Issues that will be considered by the EAB include target cell populafions for early clinical trials, informed consent, inclusion and exclusion criteria, subject recruitment and communication with the community. The Core will also provide support in biostatisfics from Dr. Robert McCarter.

Public Health Relevance

(See Instructions): This Core will provide all administrative support for the implementation ofthe program. Both an External Scientific Advisory Committee and an Ethics Advisory Board will be convened.

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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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
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