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：
10555610
负责人：
Alice F Tarantal
金额：
$ 78.72万
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：
2022
资助国家：
美国
起止时间：
2022-12-01 至 2026-11-30
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10555610
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.

项目摘要 /摘要调查人员迫切需要准备访问专门的资源来评估新的与非人类灵长类动物的再生医学和基因治疗/体细胞基因组编辑应用人类疾病的治疗。该应用程序的重点是通过改进来满足这一需求以及对恒河猴模型以及相关工具和技术的验证，以解决新方法治疗影响一系列器官系统的遗传疾病；通过为调查人员提供机会获取新的NIH赠款申请的数据和调查新药物的进行（IND）研究。任何年龄的基因转移/基因组编辑的潜在影响都强调了严格评估恒河猴模型系统的安全性，该系统紧密概括了人类生理。拟议的资源计划将引起调查人员和一系列NIH的重大关注机构将削减各种器官系统和疾病。目标将是通过以下特定目的完成：（1）增强转化基因的工具和技术研究界的恒河猴猴子利用模型的基于方法；（2）改进研究界使用的临床前异构模型研究人类造血细胞中的人类造血细胞恒河主的主持人；（3）推出转化的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翻译研究；增强生物资源，工具，技术和研究协议；并利用已建立且已验证的基础设施，该基础架构在提供方面取得了成功的记录在全国范围内调查人员的协作研究机会。为了确保调查人员的可访问性，请致电为了验证研究，将分发以启动资源。该计划将为用于开发和测试一系列新处理的临床前和指定性研究常见和稀有疾病。