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中文摘要
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项目摘要/摘要--行政核心 CRISPR愿景计划是一项真正的团队科学独特努力,旨在促进基因编辑治疗 几种视网膜通道性疾病的候选人前往诊所。该提案利用了以下方面的协同效应 工程学和生物学方面的发现,需要学术界和学术界之间的密切合作和联系 实验室、行业合作伙伴、监管专业人员、制造设施、国家卫生研究院 (NIH)和美国食品和药物管理局(FDA)。因此,这一巨大的努力需要强有力的 行政(行政)核心的行政支助,其首要目标是确保效率, 团队合作,并通过提供基础设施跨软件在项目和核心之间实现协同, 会议、财务、人员配置、后勤、通信和出版物。所有这些活动对于 CRISPR远景规划所采用的创新团队科学方法。管理核心构建在 在常设咨询小组第一阶段和几个外部会议期间被证明成功的组织原则 自2018年以来的项目。这些原则包括几个支持功能,包括通信、记录 保存、处理报告、出版物、基础设施、与大学管理人员的工作关系、软件 工具,还有更多。在实践中,将执行四项主要任务。首先,管理核心将分配和 管理人员,确保所有项目和核心的人员配备适当,并组成委员会进行监督 所有职责。管理核心将根据他们收集的信息执行项目管理任务 定期会议、进度与阶段时间表的比较以及其他因素。第二,管理核心 将通过组织定期会议支持团队成员之间的沟通和协同, 采用数据和协议共享的标准实践,并提供软件工具来协助项目 管理和团队沟通。第三,管理核心将协调以下方面的外部沟通 研究、数据集、协议和过程,包括与NIH、SCGE 联合体翻译协调和传播中心,以及其他中心,以及 传播和发布成果和资源。最后,管理核心将提供资金 管理,与机构行政部门对接,并确保以符合成本效益的方式使用各种资源 项目和核心。总体而言,管理核心将帮助整合项目,以便真正的团队科学 可以在每个项目内实现,也可以在项目之间实现。协同效应的要点预计将提供 将前景看好的基因组编辑技术快速转化为临床。管理核心将由Dr。 Krishanu Saha,他在许多大型团队科学计划中带来了领导经验,包括 SCGE的第一阶段。核心将受益于一名项目经理,他将通过以下方式执行上述任务 萨哈博士和威斯康星大学麦迪逊分校一批经验丰富的拨款管理人员。
英文摘要
PROJECT SUMMARY/ABSTRACT – ADMINISTRATIVE CORE The CRISPR Vision Program is a truly team science unique effort to advance gene editing therapeutic candidates for several retinal channelopathies towards the clinic. This proposal leverages synergy between discoveries in engineering and biology and requires close collaborations and contacts between academic labs, industry partners, regulatory professionals, manufacturing facilities, the National Institutes of Health (NIH), and the U.S. Food and Drug Administration (FDA). This large effort therefore requires strong administrative support from the Administrative (Admin) Core, whose overarching goal is to ensure efficiency, teamwork, and synergy among the Projects and Cores by providing infrastructure spanning software, meetings, finances, staffing, logistics, communications, and publications. All these activities are critical to an innovative team science approach taken by the CRISPR Vision Program. The Admin Core builds on organizational principles that have proven successful during Phase I of the SCGE and several extramural projects since 2018. These principles include several supporting functions including communication, record keeping, process reports, publications, infrastructure, working relationships with university admin, software tools, and many more. In practice, four primary tasks will be executed. First, the Admin Core will assign and manage personnel to ensure all Projects and Cores are appropriately staffed and form committees to oversee all duties. The Admin Core will perform project management tasks based on the information they collect from regular meetings, comparison of progress to the phase timelines, and other factors. Second, the Admin Core will support communication and synergy among team members through organizing regular meetings, adopting standard practices for data and protocol sharing, and providing software tools to assist with project management and team communication. Third, the Admin Core will coordinate external communications of research, datasets, protocols, and processes including information sharing with the NIH, the SCGE Consortium Translational Coordination and Dissemination Center, and across other Centers, and dissemination and publication of results and resources. Finally, the Admin Core will provide financial management, interface with institutional administration, and ensure the cost-effective use of resources across the Projects and Cores. Overall, the Admin Core will help integrate the Projects so that true Team Science can be achieved within each Project and also between Projects. The points of synergy are expected to provide a rapid translation of promising genome editing technology into the clinic. The Admin Core will be led by Dr. Krishanu Saha, who brings leadership experience with many large team science initiatives including the Phase I of the SCGE. The Core will benefit from a Program Manager who will execute the tasks above with Dr. Saha and an experienced set of grants administrators at the University of Wisconsin-Madison.
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The CRISPR Vision Program: Nonviral Genome Editing Platforms to Treat Inherited Retinal Channelopathies
  • 批准号:
    10668161
  • 项目类别:
  • 资助金额:
    $615.91万
  • 财政年份:
    2023
  • 负责人:
    Krishanu Saha
  • 依托单位:
Streamlined development of an IND with the silica nanocapsule loaded with Cas9 genome editors to disrupt the dominant BEST1 mutant allele
  • 批准号:
    10668168
  • 项目类别:
  • 资助金额:
    $69.69万
  • 财政年份:
    2023
  • 负责人:
    Krishanu Saha
  • 依托单位:
Assembly of Novel Gene Editing Particles to Understand Genome Surgery in Patient-Derived Cells
  • 批准号:
    10618322
  • 项目类别:
  • 资助金额:
    $41.57万
  • 财政年份:
    2016
  • 负责人:
    Krishanu Saha
  • 依托单位:
Assembly of Novel Gene Editing Particles to Understand Genome Surgery in Patient-Derived Cells
  • 批准号:
    9142548
  • 项目类别:
  • 资助金额:
    $36.98万
  • 财政年份:
    2016
  • 负责人:
    Krishanu Saha
  • 依托单位:
海外基金