Administrative Core

行政核心 这个核心将由威尔逊博士指导，巴特肖博士和卡普兰博士担任联合主任。芯会 提供基本的行政支持，以管理该计划项目的财务运作，包括 监督账户、采购、编写和向机构提交报告，以及 国家卫生研究院将召集一个外部科学咨询委员会（ESAC），其中包括科学家 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。伦理咨询委员会（EAB）也将召集和主持博士。 卡普兰该委员会将包括一名来自ESAC的科学家，以及遗传性疾病儿童的父母。 疾病和具有道德、政策和法律方面专门知识的人。ESAC和EAB将在一个 每年审查科学进展，帮助团队解决与科学有关的重要问题。 基因治疗的最终临床应用。EAB将考虑的问题包括目标细胞 用于早期临床试验的人群、知情同意、入选和排除标准、受试者招募和 与社区的沟通。核心还将提供支持。在生物统计学博士罗伯特 麦卡特Joy Cavagnaro博士将担任FDA相关问题的顾问。 Lay描述。该核心将为方案的实施提供所有行政支持。两 将召集外部科学咨询委员会和伦理咨询委员会。

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.