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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。