Administrative Core

行政核心

• 批准号: 10669044
• 负责人: David Loren Nelson
• 金额: $ 13.86万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-09-25 至 2025-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10669044
• 关键词: Address; Basic Science; Beavers; CGG repeat; Committee Members; Communication; Communities; Development; Discipline of obstetrics; Disease; Ensure; FMR1; FMR1 Premutation; FMR1 repeat; FXTAS; Fostering; Fragile X Premutation; Fragile X Syndrome; Functional disorder; General Population; Generations; Genetic; Goals; Hares; Institution; Journals; Leadership; Literature; Medicine; Michigan; Molecular; Mutation; Neurology; Neurosciences; Participant; Pediatrics; Publishing; Recording of previous events; Research; Research Activity; Research Personnel; Resource Allocation; Resources; Role; Schedule; Scientist; Structure; Talents; Therapeutic; Training; Training and Education; United States National Institutes of Health; Universities; Work; college; interest; medical schools; meetings; member; novel; novel therapeutics; operation; pre-clinical; primary ovarian insufficiency; success; symposium; videoconference

As described in the Overall component of this proposal, the overarching goal of the proposed Center is to understand and address the molecular and genetic mechanisms underlying the FMR1 premutation disorders, focusing on Fragile X tremor ataxia syndrome (FXTAS) and Fragile X associated primary ovarian insufficiency (FXPOI), and to answer fundamental questions regarding the roles of the CGG repeats in FMR1 function and dysfunction. The proposed Center integrates the substantial talents of research groups at Baylor College of Medicine (BCM), University of Michigan School of Medicine (UM) and Emory University School of Medicine (Emory). These groups have a significant history of collaborative interaction, and their common interests will lead to robust findings that will suggest therapeutic approaches to FXTAS, FXPOI and possibly fragile X syndrome (FXS). A well-defined leadership and management plan overseeing all research endeavors is critical for the success of the proposed work and will ensure that goals set by the Center Investigators are met in a timely and efficient manner. To accomplish the goals of our Center, the Administrative Core will support the Center's Investigators. Led by co-Directors Drs. David Nelson and Peter Todd (leaders of Projects 2 and 1, respectively), and assisted by Ms. LaTonya Beavers, the Administrative Core will be responsible for the management and administration of the overall Center. The planning and coordination of the synergistic research activities will be integrated through this central hub. Efforts will be conducted by an Executive Team, including Drs. Emily Allen and Stephanie Sherman, Project 3 Co-PIs, with the guidance of an Executive Advisory Board, composed of four external committee members who will address all Center operations and help to ensure the timely completion of each proposed project. In addition, the Administrative Core will be responsible for fiscal and resource management, as well as maintaining open lines of communication between Center Investigators, Project Staff, NIH officials, and FX researchers. A significant emphasis will also be placed on communication and engagement with the broader community of scientists and clinicians in neuroscience, genetics, neurology, obstetrics and pediatrics, and with the public. Training and educational opportunities are provided through regularly scheduled journal clubs, research presentations, and an annual Fragile X-Associated Disorders (FXaDs) Symposium beginning in year 2. These activities will foster a deeper interest in FX research in the new generation of young scientists, as well as encourage and stimulate investigators at all levels who may not be in our field to pursue studies related to FXaDs. We envision that the BCM-Michigan-Emory Fragile Research X Center will be a nexus of exciting scientific research, training and preclinical endeavors focused on development of novel therapeutics for FXaDs.

正如本提案总体部分所述，拟议中心的总体目标是 了解并解决 FMR1 预突变背后的分子和遗传机制 疾病，重点关注脆性 X 震颤共济失调综合征 (FXTAS) 和脆性 X 相关原发性 卵巢功能不全 (FXPOI)，并回答有关 CGG 作用的基本问题 FMR1 功能和功能障碍中的重复。拟议的中心整合了大量的研究人才 贝勒医学院 (BCM)、密歇根大学医学院 (UM) 和埃默里大学的研究小组 大学医学院（埃默里大学）。这些团体有着重要的协作互动历史，并且 他们的共同兴趣将带来强有力的发现，为 FXTAS、FXPOI 的治疗方法提出建议 可能还有脆性 X 综合征 (FXS)。监督所有研究的明确的领导和管理计划 努力对于拟议工作的成功至关重要，并将确保中心设定的目标 及时有效地会见调查人员。为了实现我们中心的目标，行政 核心将支持该中心的调查人员。由联合董事博士领导。大卫·尼尔森 (David Nelson) 和彼得·托德 (Peter Todd)（ 项目 2 和 1），在 LaTonya Beavers 女士的协助下，行政核心将 负责中心整体管理和行政工作。的规划和协调 协同研究活动将通过这个中心枢纽整合起来。相关工作将由一个 执行团队，包括博士。 Emily Allen 和 Stephanie Sherman，项目 3 联合 PI，在一位 执行顾问委员会，由四名外部委员会成员组成，负责解决中心的所有运营问题 并帮助确保每个拟议项目的按时完成。此外，行政核心将是 负责财政和资源管理，并保持双方之间开放的沟通渠道 中心研究人员、项目工作人员、NIH 官员和外汇研究人员。也将重点强调 与更广泛的神经科学领域的科学家和临床医生进行沟通和接触， 遗传学、神经病学、产科和儿科以及公众。培训和教育机会是 通过定期安排的期刊俱乐部、研究报告和年度脆弱 X 协会提供 疾病 (FXaDs) 研讨会从第二年开始。这些活动将培养人们对外汇研究更深入的兴趣 培养新一代年轻科学家，并鼓励和激励各级研究人员 不在我们的领域从事与 FXaD 相关的研究。我们预计 BCM-密歇根-埃默里脆弱 Research X 中心将成为令人兴奋的科学研究、培训和临床前工作的纽带，重点关注 开发 FXaD 的新疗法。