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中文摘要
翻译
开发用于SCID-X1的基于FV的整合基因疗法需要有效整合我们的基因。 建议在五个研究所开展基础和实用研究工作,并支持核心服务 (西雅图儿童研究所(SCR 1);弗雷德哈钦森癌症研究所(FHCRC); 华盛顿州立大学(WSU)、华盛顿大学(UW)和宾夕法尼亚大学(U of Penn))。行政核心的总体目标是通过协调 我们的PPG计划的行政,财政和组织方面。具体而言,核心将: 为财政资源管理、支出和预算跟踪提供行政支助; 协调会议和沟通,并在调查人员之间传播关键信息, 更广泛的科学界;安排外部咨询委员会的年度实地访问;并确保 我们在进行研究时符合最高的道德标准。核心活动将与科学 和管理所有合作机构的工作人员,并在这些机构之间架起桥梁, 确保项目顺利运行。我们的行政人员已经善于处理 这样一个复杂的组合程序所创造的。罗林斯博士和基姆博士一直在合作 5年以上基因治疗相关研究。他们将共同领导核心A, 有效地指导和协调整个项目工作流程。为了实现这一目标,核心A的关键人员将 位于各自的机构,SCR 1和FHCRC。 相关性: 成功开发基于FV载体的SCID-X1基因治疗需要整合我们的研究 在五个方面。行政核心将通过协调行政部门, 财政和组织方面的问题核心活动将与科学和 所有机构的行政人员提供一个桥梁,确保顺利运作。
英文摘要
Development of an integrated FV-based gene therapy for SCID-Xl requires efficient integration of our proposed basic and translafional research efforts and supporting core services across five institufions (Seattle Children's Research Institute (SCRl); Fred Hutchinson Cancer Research Institute (FHCRC); Washington State University (WSU); University of Washington (UW) and University of Pennsylvania (U of Penn)). The overall goal of the Administrative Core is to facilitate success by coordinafion of the administrative, fiscal and organizafional aspects of our PPG program. Specifically, the Core will: provide administrative support for management of financial resources, expenditure and budgetary tracking; coordinate meefings and communicafions and disseminate key informafion among invesfigators and with the larger scientific community; orchestrate an annual External Advisory Committee site visit; and ensure that the highest ethical standards are met in conducting our studies. Core activities will interface with scientific and administrafive staff at all collaborafing institutions and provide a bridge between these institutions to ensure smooth operafion of the program. Our administrative staff is already adept at handling the complexities created by such a combined program. Dr. Rawlings and Dr. Kiem have been collaborating for more than 5 years on gene therapy related studies. They will work together to lead Core A and thereby effectively direct and coordinate overall project work flow. To accomplish this, key Core A personnel will be located at each of their respective institutions, SCRl and FHCRC. RELEVANCE: Successful development of FV vector-based gene therapy for SCID-Xl requires integration of our studies across five insfitutions. The Administrafive Core will facilitate success by coordination of the administrafive, fiscal and organizafional aspects of our program. Core acfivifies will interface with scientific and administrative staff at all institufions to provide a bridge ensuring smooth operation.
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会议论文
An integrated strategy to define the functional and synergistic impact of T1D causal variants
  • 批准号:
    9227381
  • 项目类别:
  • 资助金额:
    $441.14万
  • 财政年份:
    2016
  • 负责人:
    David J Rawlings
  • 依托单位:
Lentiviral Gene Therapy of X-Linked Agammaglobulinemia
  • 批准号:
    8825754
  • 项目类别:
  • 资助金额:
    $8.78万
  • 财政年份:
    2014
  • 负责人:
    David J Rawlings
  • 依托单位:
Pre-clinical Modeling of Foamy Viral gene Therapy for Murine and Human SCID-X1
  • 批准号:
    8278864
  • 项目类别:
  • 资助金额:
    $25.01万
  • 财政年份:
    2012
  • 负责人:
    David J Rawlings
  • 依托单位:
B cell function and phenotype as predictors of therapeutic response to rituximab
  • 批准号:
    8044994
  • 项目类别:
  • 资助金额:
    $39.36万
  • 财政年份:
    2010
  • 负责人:
    David J Rawlings
  • 依托单位:
海外基金