UAB CF Research and Translation Core Center

项目总结/摘要 UAB P30研究和翻译核心中心整合了大量外部资助的囊性 纤维化（CF）研究计划在我们的校园，建立一个创新的环境，追求，推进， 在CF相关的科学培训。在上一个供资期间，P30为以下方面作出了强有力的重要贡献： 我们研究基地的多个UAB实验室，超过70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局指标;感染和炎症 在高度有效的调节剂治疗的背景下;以及用于未治疗的CFTR突变的治疗性翻译。 凭借NIH中心，我们机构的转化研究在过去五年中继续加速 多年来，随着调查人员的广泛性。UAB的CF基础科学的丰富性与 这一平移式扩张，为下一个融资期提供了众多机会， 中心的战略结构是利用这些机会。 P30中心允许UAB和合作研究中心的研究人员提高对CF的理解 疾病机制，并提供了新的机会，积极应用这些信息， 实验疗法，包括将继续受到重视的基于基因的疗法。这个NIH 中心包括三个生物医学研究核心，有助于组织CF教师的努力， 共同的和基本的目标，帮助个人与CF和培训下一代CF领导人。的 核心包括：核心A：细胞模型和测定核心（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）;和核心C：临床和转化核心（SM Rowe和A Gaggar，Co-PI）。每个核心都提供领先的检测、专业试剂和技术， 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， 中心活力，并与UAB临床和转化科学中心紧密结合， 强大的训练环境。除了为初级和高级科学家提供一个平台外， 进入该领域，试点项目作为一种手段，迅速测试令人兴奋的进展，特别是从 临床翻译的角度。两个试点和可行性项目正在进行中：项目1：R Patel，PI。 “CF中的红细胞溶血和肺部疾病”;以及项目2：血小板生成素，PI。“酮单酯 补充CF：试点和可行性研究”，利用下一个时代的创新机会， 转化疾病。通过这些科学举措，P30为合作伙伴关系增加了价值。 环境CF研究在我们的机构，并处于有利地位，继续在这方面的能力，在未来， 主要专注于治疗翻译。

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.