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

转化非人类灵长类再生医学和基因治疗/基因组编辑资源计划

基本信息

批准号：
10889393
负责人：
Alice F Tarantal
金额：
$ 24.59万
依托单位：
UNIVERSITY OF CALIFORNIA AT DAVIS
依托单位国家：
美国
项目类别：
财政年份：
2023
资助国家：
美国
起止时间：
2023-08-01 至 2026-11-30
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10889393
关键词：
Address Adverse event Advisory Committees Age Animal Model Animals Applications Grants Biological Biological Models Bioluminescence Brain CD34 gene Cells Clinical Trials Communities Data Dedications Dependovirus Development Disease Engraftment Ensure Female Gene Expression Gene Transfer Genes Genetic Diseases Goals Heart Hematopoietic Hereditary Disease Human Imaging technology Immune response Immune system Infrastructure Investigation Investigational Drugs Kidney Liver Longevity Lung Macaca mulatta Methods Modeling Monitor Monkeys Musculoskeletal National Heart, Lung, and Blood Institute National Institute of Allergy and Infectious Disease National Institute of Arthritis, and Musculoskeletal, and Skin Diseases National Institute of Child Health and Human Development National Institute of Diabetes and Digestive and Kidney Diseases National Institute of Neurological Disorders and Stroke Organ Outcome Patients Physiology Positron-Emission Tomography Primates Process Protocols documentation Rare Diseases Regenerative Medicine Research Research Personnel Resources Rhesus Safety Serotyping Specificity Technology Testing Tissues Translational Research Umbilical Cord Blood United States National Institutes of Health Validation Xenograft Model adaptive immune response age group body system conditioning effective therapy gene therapy genome editing human disease imaging modality immunogenicity improved in utero transplantation in vivo evaluation in vivo imaging individualized medicine innovation insight interest male meetings nonhuman primate novel strategies novel therapeutics postnatal pre-clinical prime editor programs prototype real time monitoring response safety assessment somatic cell gene editing technology/technique therapeutic evaluation tool translational approach translational pipeline validation studies

项目摘要

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.

项目概要/摘要研究人员迫切需要能够随时访问专用资源来评估新的再生医学和基因治疗/体细胞基因组编辑在非人类灵长类动物中的应用人类疾病的治疗。该应用程序致力于通过改进来满足这一需求恒河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；并为调查人员提供机会获取新的 NIH 拨款申请和新药研究 (IND) 支持的数据研究。基因转移/基因组编辑在任何年龄段的潜在影响强调了对密切再现人类的恒河猴模型系统的安全性进行严格评估生理。拟议的资源计划将引起研究人员和一系列 NIH 的极大兴趣研究所所带来的机遇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来实现：（1）增强转化基因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； (3) 启动转化性非人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实现 RFA 的目标整个生命周期的转化研究；加强生物资源、工具、技术和研究协议；并利用成熟且经过验证的基础设施，该基础设施在提供服务方面拥有成功的记录为全国研究人员提供合作研究机会。为了确保调查人员能够访问，请致电将分发以进行验证研究以启动该资源。该计划将提供一个管道针对一系列新疗法的开发和测试的临床前和 IND 支持研究常见和罕见疾病。