UAB CF Research and Translation Core Center

项目摘要/摘要 UAB P30研究和翻译核心中心整合了大量外部资金的囊性 我们校园内的纤维化(CF)研究计划，构建一个创新的环境，以追求、推进和 接受过CF相关科学方面的培训。在上一个筹资期间，P30作出了有力和重要的贡献， UAB的多个实验室，我们的研究基地有70多名研究人员，从事与CFTR相关的研究 调节；CF发病机制；基因治疗时代的新结果衡量标准；感染和炎症 在高效调节剂治疗的背景下；以及对未经治疗的CFTR突变的治疗性翻译。 借助美国国立卫生研究院中心，我们研究所的翻译研究在过去五年中继续加速 几年来，调查人员的广度也是如此。加州大学伯克利分校CF基础科学的丰富性与 这种翻译扩展，在下一个资金期提供了大量的机会来利用，并且 Center的战略架构是为了利用这些机会。 P30中心允许UAB和合作站点的调查人员提高对CF的理解 疾病机制，并提供了新的机会，积极地应用这些信息来 实验疗法，包括将继续受到重视的基于遗传的疗法。这家美国国立卫生研究院 中心包括三个生物医学研究中心，帮助组织CF教职员工为 共同的和基本的目标是帮助患有CF的个人，并培养下一代CF领导者。这个 核心包括：核心A：细胞模型和分析核心(GM所罗门和J Guimbellot，Co-PI)；核心B：动物 模型核心(DM Bedwell和S Birket，Co-PI)；以及核心C：临床和翻译核心(SM Rowe和A Gaggar，Co-PI)。每个核心提供尖端的分析，专门的试剂和技术，并重视 专业知识。P30还通过试点和可行性机制聘请了新的调查人员，这是 中心充满活力，并与UAB临床和转化科学中心紧密集成，使 良好的培训环境。除了提供一个平台，初级和高级科学家 将试点项目带入实地，作为一种快速测试激动人心的进步的手段，特别是来自 临床翻译的视角。两个试验性和可行性项目正在进行中：项目1：R Patel，PI。 “慢性粒细胞白血病中的红细胞溶血和肺部疾病”；和项目2：E Plaisance，PI。“酮单酯 在合作伙伴关系中的补充：试点和可行性研究“，利用创新机会的下一个时代 改变疾病。通过这些科学计划，P30为协作 为我们研究所的CF研究创造了良好的环境，并处于有利地位，将在未来继续担任这一职务。 主要集中在治疗性翻译上。

PROJECT SUMMARY/ABSTRACT The UAB P30 Research and Translation Core Center consolidates a large number of externally funded cystic fibrosis (CF) research programs on our campus, building an innovative environment to pursue, advance, and train in CF-related science. During the last funding period, the P30 made robust and important contributions to multiple UAB laboratories of our Research Base of over 70 investigators pursuing research relevant to CFTR modulation; CF pathogenesis; novel outcome measures for the genetic therapy era; infection and inflammation in the context of highly effective modulator therapy; and therapeutic translation for untreated CFTR mutations. By virtue of the NIH Center, translational research at our Institution has continued to accelerate in the past five years, as has the breadth of investigators. The richness of CF basic science at UAB has grown in parallel with this translational expansion, providing numerous opportunities to exploit in the next funding period, and the Center is strategically structured to take advantage of these opportunities. The P30 Center has allowed investigators at UAB and collaborating sites to improve understanding of CF disease mechanism and has furnished novel opportunities to aggressively apply this information towards experimental therapeutics, including genetic-based therapies that will continue to grow in emphasis. This NIH Center includes three Biomedical Research Cores that help to organize efforts of CF faculty towards the common and essential goal of helping individuals with CF and to train the next generation of CF leaders. The Cores include: Core A: Cell Model and Assay Core (GM Solomon and J Guimbellot, Co-PIs); Core B: Animal Models Core (DM Bedwell and S Birket, Co-PIs); and Core C: Clinical and Translational Core (SM Rowe and A Gaggar, Co-PIs). Each Core provides leading-edge assays, specialized reagents and techniques, and valued expertise. The P30 has also engaged new investigators through a Pilot and Feasibility mechanism integral to Center vitality and has a tight integration with the UAB Center for Clinical and Translational Science, enabling a robust training environment. In addition to providing a platform from which junior and senior scientists are brought into the field, Pilot Projects serve as a means of rapidly testing exciting advances, particularly from the perspective of clinical translation. Two Pilot and Feasibility Projects are in progress: Project 1: R Patel, PI. “Erythrocyte Hemolysis and Lung Disease in CF”; and Project 2: E Plaisance, PI. “Ketone Monoester Supplementation in CF: A Pilot and Feasibility Study”, capitalizing on the next era of innovative opportunities to transform the disease. Through these scientific initiatives, the P30 has added value to a collaborative environment for CF research at our Institution, and is well-positioned to continue in this capacity in the future, with a major focus on therapeutic translation.