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.
(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.
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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|
|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|
|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|
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|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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|Wang, Lili; Wang, Huan; Bell, Peter et al. (2012) Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector. Hum Gene Ther 23:533-9|
|Wang, Lili; Morizono, Hiroki; Lin, Jianping et al. (2012) Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome. Mol Genet Metab 105:203-11|
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