This Core will be directed by Dr. Wilson with Drs. Batshaw and Caplan serving as co-directors. The Core will provide basic administrative 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 scientist 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 ultimate clinical applications of gene therapy. Issues that will be considered by the EAB include target cell populations for early clinical trials, informed consent, inclusion and exclusion criteria, subject recruitment and communication with the community. The Core will also provide support in biostatistics from Dr. Robert McCarter.

Agency
National Institute of Health (NIH)
Institute
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Type
Research Program Projects (P01)
Project #
4P01HD057247-09
Application #
9060369
Study Section
Biobehavioral and Behavioral Sciences Subcommittee (CHHD)
Project Start
Project End
Budget Start
2016-04-01
Budget End
2017-03-31
Support Year
9
Fiscal Year
2016
Total Cost
Indirect Cost
Name
University of Pennsylvania
Department
Type
DUNS #
042250712
City
Philadelphia
State
PA
Country
United States
Zip Code
19104
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