Center for Fetal Gene Transfer for Heart, Lung, and Blood Diseases

心脏、肺和血液疾病胎儿基因转移中心

• 批准号: 8235272
• 负责人: Alice F Tarantal
• 金额: $ 51.12万
• 依托单位: UNIVERSITY OF CALIFORNIA AT DAVIS
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-09-01 至 2016-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8235272
• 关键词: Address; Adverse effects; Advisory Committees; Allogenic; Animals; Autologous; Biological Assay; Bioluminescence; Calendar; Cell Separation; Cells; Childhood; Clinical; Clinical Trials; Communities; Core Facility; Data; Development; Disease; Ensure; Fetus; Flow Cytometry; Funding; Future; Gene Delivery; Gene Expression Profiling; Gene Transfer; Genes; Germ Lines; Goals; Health Services Research; Heart Diseases; Hematological Disease; Housing; Human; Image; Infant; Institution; Instruction; Investigation; Laboratories; Letters; Longevity; Lung diseases; Methods; Mission; Modeling; Molecular; Monkeys; National Heart, Lung, and Blood Institute; Organ; Outcome; Outreach Research; Positron-Emission Tomography; Primates; Process; Research; Research Personnel; Resources; Risk; Rodent; Role; Safety; Series; Services; Site; Structure; Techniques; Technology; Testing; Time; Tissues; Translating; Ultrasonography; adeno-associated viral vector; base; design; experience; fetal; follow-up; gene therapy; human disease; in vivo; interest; nonhuman primate; novel; novel strategies; outreach; outreach program; postnatal; pre-clinical; prenatal; programs; success; symposium; technology/technique; therapeutic gene; transgene expression; translational study; transmission process; web site

DESCRIPTION (provided by applicant): The goals of the Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases are to conduct gene transfer studies in monkeys to evaluate the safety and efficiency of gene therapy strategies as they emerge; and to provide NHLBI-supported investigators with essential expertise, resources, and services to actively pursue gene transfer approaches in monkeys in their research programs. The overriding objective is to fulfill the mission of service to investigators and to explore crucial questions in gene delivery in a relevant translational nonhuman primate model. The success of our outreach program, service and research accomplishments, and ongoing interest from the greater research community to pursue gene transfer studies in monkeys demonstrates the importance of this Center and role in advancing the field of gene therapy for the treatment of human disease. The Center has supported and conducted projects for NHLBI-funded investigators spanning institutions across the U.S. These projects have addressed novel approaches to gene delivery that cut across the lifespan with a special focus on the fetus and infant, "proof-of-principle," evaluated if findings in rodents could be shown in nonhuman primates, or were conducted as a preclinical step in advance of a new clinical trial. The Specific Aims for this renewal application include: (1) Provide expertise, specialized services, and resources to NHLBI-supported investigators in which to explore gene transfer strategies in monkeys for the treatment of human disease, and (2) Develop technologies to enhance the resource and continue to explore long-term safety post-gene delivery in monkeys. Our established administrative structure will provide these services in a proven collaborative framework. Investigators will have access to these opportunities through a competitive process; projects are selected through an established Scientific Advisory Committee. We will also further develop new techniques specific to translational studies with monkeys for the greater research community, continue to rigorously assess safety, and communicate research opportunities through websites, announcements, and our Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases. RELEVANCE: Gene transfer could replace defective or missing genes for therapeutic benefit provided that such methods are carefully tested and found to be safe. The focus of this application is to continue to ensure the resources, expertise, and services necessary for NHLBI-supported investigators to assess new gene transfer strategies in a primate model are available to provide the preclinical data necessary for future human clinical trials.

描述（由申请人提供）：胎猴心脏、肺和血液疾病基因转移中心的目标是在猴子中进行基因转移研究，以评估出现的基因治疗策略的安全性和效率；为 NHLBI 支持的研究人员提供必要的专业知识、资源和服务，以便在其研究项目中积极探索猴子基因转移方法。最重要的目标是履行为调查人员服务的使命并探索调查中的关键问题 相关翻译非人类灵长类动物模型中的基因传递。我们的外展计划、服务和研究成果的成功，以及更大的研究界对猴子基因转移研究的持续兴趣，证明了该中心的重要性以及在推进人类疾病治疗基因治疗领域的作用。该中心为 NHLBI 资助的美国各地机构的研究人员提供支持和开展项目。这些项目研究了跨越生命周期的基因传递新方法，特别关注胎儿和婴儿，“原理验证”，评估啮齿类动物的研究结果是否可以在非人类灵长类动物中显示，或者作为新临床试验之前的临床前步骤进行。此次更新申请的具体目标包括：(1) 向 NHLBI 支持的研究人员提供专业知识、专业服务和资源，以探索猴子中治疗人类疾病的基因转移策略，以及 (2) 开发技术以增强资源并继续探索猴子基因传递后的长期安全性。我们建立的管理结构将在经过验证的协作框架中提供这些服务。研究人员将通过竞争过程获得这些机会；项目是通过已成立的科学咨询委员会选出的。我们还将进一步为更大的研究界开发针对猴子转化研究的新技术，继续严格评估安全性，并通过网站、公告和年度心脏、肺和血液疾病基因治疗研讨会传达研究机会。 相关性：基因转移可以替代有缺陷或缺失的基因以获得治疗益处，前提是这些方法经过仔细测试并发现是安全的。该申请的重点是继续确保 NHLBI 支持的研究人员评估灵长类动物模型中的新基因转移策略所需的资源、专业知识和服务，从而为未来的人类临床试验提供所需的临床前数据。