Translational Nonhuman Primate Regenerative Medicine and Gene Therapy/Genome Editing Resource Program

项目总结/摘要 迫切需要调查人员能够随时获得专门资源，以评价新的 再生医学和基因治疗/体细胞基因组编辑应用于非人灵长类动物， 人类疾病的治疗。该应用程序的重点是通过改进来满足这一需求 和验证恒河猴模型及相关工具和技术， 治疗影响一系列器官系统的遗传性疾病;并为研究人员提供机会 获得新的NIH拨款申请和研究性新药（IND）的数据-使 问题研究基因转移/基因组编辑在任何年龄的潜在后果强调了以下方面的重要性： 在恒河猴模型系统中严格评估安全性， physiology.拟议的资源计划将对研究人员和一系列NIH 研究所作为机会将跨越一系列器官系统和疾病。目标将是 通过以下具体目标实现：（1）加强翻译基因的工具和技术， （2）在恒河猴模型中使用基于方法的方法，供研究界使用; 临床前异种模型，供研究界用于研究人类造血细胞， 恒河猴宿主;（3）启动翻译非人类灵长类动物再生医学和基因 用于模型验证和治疗测试的治疗/体细胞基因组编辑资源计划。我们 翻译团队将通过改进和验证猴子模型系统来实现RFA的目标， 整个生命周期的转化研究;增强生物资源，工具，技术和研究 协议;以及利用已建立并经过验证的基础设施，该基础设施在提供 为全国各地的研究人员提供合作研究的机会。为了确保调查人员的可访问性， 将分发验证研究报告，以启动资源。该计划将提供一个管道， 临床前和IND研究，用于开发和测试一系列新的治疗方法， 常见和罕见疾病。

PROJECT SUMMARY / ABSTRACT There is a critical need for investigators to have ready access to a dedicated resource to evaluate new regenerative medicine and gene therapy/somatic cell genome editing applications with nonhuman primates for the treatment of human diseases. This application is focused on meeting this need through the improvement and validation of the rhesus monkey model and related tools and technologies to address new approaches to treat inherited disorders that impact a range of organ systems; and by providing investigators with opportunities to obtain data for new NIH grant applications and for the conduct of investigational new drug (IND)-enabling studies. The potential ramifications of gene transfer/genome editing at any age underscores the importance of rigorous assessments of safety in the rhesus monkey model system which closely recapitulates human physiology. The proposed resource program will be of significant interest to investigators and a range of NIH Institutes as the opportunities will cut across a spectrum of organ systems and diseases. The goals will be accomplished through the following Specific Aims: (1) Enhance tools and technologies for translational gene- based approaches in the rhesus monkey model for utilization by the research community; (2) Improve preclinical xenogeneic models for use by the research community to study human hematopoietic cells in the rhesus host; and (3) Launch the Translational Nonhuman Primate Regenerative Medicine and Gene Therapy/Somatic Cell Genome Editing Resource Program for model validation and therapeutic testing. Our translational team will meet the goals of the RFA by improving and validating the monkey model system for translational research across the lifespan; enhancing biological resources, tools, technologies, and research protocols; and utilizing an established and proven infrastructure that has a successful track record in providing collaborative research opportunities to investigators nationwide. To ensure accessibility for investigators, a call for validation studies will be circulated to launch the resource. The program will provide a pipeline for preclinical and IND-enabling investigations for the development and testing of new treatments for a range of common and rare diseases.