Administrative Core

行政核心（核心A）将主要负责全面的科学和财务管理， 管理P01。P01的总体使命是组装一个综合性的程序， 生物信息学和基因组专业知识，不仅能够识别个体中的致病变异， 与SCID和T细胞缺陷，而且还利用基因组编辑作为一种真正个性化的方法， 治疗P01将有三个研究项目：项目1，候选人的系统识别 T细胞功能不全患者的致病变异;项目2。基因组编辑来创造， 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细胞功能不全的等位基因变异。这些将由三个核心（包括 核心A，管理;核心B，信息学基础设施和生物信息学分析; 核心C，受试者入组和样本管理。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 行政支持; 3）提供所有P01活动的财政监督和管理;以及4）促进所有 P01的规划和评估活动。这项工作将确保取得有效进展， 资源，并加强P01调查人员和项目之间的协同作用。行政核心 包括一个由共同主要研究者、项目负责人和核心负责人组成的指导委员会， 将负责审查进展情况和作出重大决定，并将设在纽约大学。 加州，旧金山弗朗西斯科（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).