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

项目摘要/摘要 迫切需要调查人员能够随时访问专用资源，以评估新的 再生医学和基因治疗/体细胞基因组编辑在非人灵长类动物中的应用 人类疾病的治疗。本应用程序致力于通过改进来满足这一需求 猕猴模型和相关工具和技术的验证，以解决新的方法 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 为新的NIH拨款申请和进行使能新药(IND)的研究获得数据 学习。任何年龄的基因转移/基因组编辑的潜在后果都突显了 严格评估恒河猴模型系统的安全性，该模型系统非常接近人类 生理学。拟议的资源计划将引起调查人员和一系列NIH的极大兴趣 这些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)加强基因翻译的工具和技术- 基于恒河猴模型的方法，供研究界使用；(2)改进 研究界用于研究人类造血细胞的临床前异种模型 恒河猴宿主；以及(3)推出转化型非人类灵长类再生医学和基因 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。我们的 翻译团队将通过改进和验证猴子模型系统来满足RFA的目标 跨生命周期的翻译研究；加强生物资源、工具、技术和研究 协议；以及利用已建立和经过验证的基础设施，该基础设施在提供 为全国范围内的研究人员提供合作研究机会。为了确保调查人员的可访问性，请拨打 将分发用于验证的研究报告，以启动资源。该计划将提供一条管道，用于 临床前和支持IND的调查，以开发和测试一系列新的治疗方法 常见病和罕见病。

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.