Administrative Core

管理核心 这一核心将由威尔逊博士执导，巴特肖和卡普兰博士担任联合导演。核心将会 提供基本的行政支持，以管理计划项目的财务运作，包括 监督帐目、采购、编写和向机构提交报告，以及 美国国立卫生研究院。将召集一个外部科学咨询委员会(ESAC)，其中将包括科学家 了解与赠款相关的领域，包括免疫学、尿素循环障碍、AAV和 病媒安全评估。道德咨询委员会(EAB)也将由Dr。 卡普兰。该委员会将包括来自ESAC的一名科学家以及遗传病儿童的父母 疾病和具有伦理、政策和法律专业知识的人。ESAC和EAB将于 每年审查科学进展，并帮助团队解决与 基因治疗的终极临床应用。EAB将考虑的问题包括目标小区 早期临床试验的人群、知情同意、纳入和排除标准、受试者招募和 与社区沟通。该中心还将为罗伯特博士的生物统计学提供支持 麦卡特。喜悦·卡瓦纳罗博士将担任美国食品和药物管理局相关问题的顾问。 版面描述。该核心将为该方案的实施提供所有行政支持。两者都有 将召集一个外部科学咨询委员会和一个道德操守咨询委员会。

Administrative Core 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 of the 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 of the 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. Dr. Joy Cavagnaro will serve as a consultant regarding FDA-related issues. Lay description. This Core will provide all administrative support for the implementation of the program. Both an External Scientific Advisory Committee and an Ethics Advisory Board will be convened.