Administrative Core

行政核心(核心A)将主要负责总体科学和财务 P01的管理。这个P01的总体使命是组装一个综合的方案， 生物信息学和基因组学专业知识，不仅能够识别个体的致病变异 SCID和T细胞缺陷，但也利用基因组编辑作为一种真正个性化的方法来 治疗。P01将有三个研究项目：项目1，系统确定候选人 T细胞不足患者的致病变异；项目2。基因组编辑以创建， 验证和纠正导致T细胞功能不全的等位基因变异；以及项目3，功能分析 导致T细胞不足的等位基因变异。这些将得到三个核心的补充(包括 这个)：核心A，行政；核心B，信息学基础设施和生物信息学分析；以及 核心C，参与者注册和样本管理。行政核心将满足其 通过实施四个具体目标来承担责任：1)提供强有力的科学领导；2)提供 行政支助；3)对所有P01活动进行财政监督和管理；4)为所有 规划和评估P01的活动。这项工作将确保有效进展，优化利用 资源，以及加强P01调查人员和项目之间的协同作用。管理核心 包括一个由联合首席调查员以及项目和核心领导组成的指导委员会，该委员会 将负责审查进展和做出重大决策，并将常驻芝加哥大学 加州，旧金山(加州大学旧金山分校)。

The Administration Core (Core A) will have primary responsibility for overall scientific and financial management of the P01. The overall mission of this P01 is to assemble a comprehensive program that integrates bioinformatic and genomic expertise to enable not only identification of disease-causing variants in individuals with SCID and T cell deficiencies, but also to exploit genome editing as a truly personalized approach to treatment. The P01will have three research Projects: Project 1,Systematic identification of candidate disease-causing variants in patients with T cell insufficiency; Project 2. Genome editing to create, validate and correct allelic variants responsible for T cell insufficiency; and Project 3, Functional analysis of allelic variants responsible for T cell insufficiency. These will be complemented by three Cores (including this one): Core A, Administration; Core B, Informatics Infrastructure and Bioinformatics Analysis; and Core C, Participant Enrollment and Sample Management. The Administration Core will meet its responsibility by implementing four specific aims: 1) Provide strong scientific leadership; 2) Provide administrative support; 3) Provide fiscal oversight and management of all P01 activities; and 4) Facilitate all planning and evaluation activities for the P01. This work will ensure effective progress, optimal utilization of resources, and enhancement of the synergies among P01 investigators and Projects. The Administration Core includes a Steering Committee consisting the Co-Principal Investigators and the Project and Core Leads, which will be responsible for reviewing progress and making major decisions, and will be based at the University of California, San Francisco (UCSF).