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: 8588959
• 负责人: Alice F Tarantal
• 金额: $ 101.05万
• 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/8588959
• 关键词: Address; Adverse effects; Advisory Committees; Allogenic; Animals; Autologous; Biological Assay; 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; Heart Diseases; Hematological Disease; Housing; Human; Image; Infant; Institution; Instruction; Investigation; Laboratories; Letters; Longevity; Lung; 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; bioluminescence imaging; design; experience; fetal; follow-up; gene therapy; human disease; in vivo; in vivo imaging; 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

The goals of ttie Center for Fetal Monkey Gene Transfer for Heart. Lung, and Blood Diseases are to conduct gene transfer studies in monkeys to evaluate tlie 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.

TTIE胎儿猴子基因转移的目标。肺和血液疾病要进行 猴子中的基因转移研究，以评估基因治疗策略的安全性和效率 出现;并向NHLBI支持的调查人员提供基本的专业知识，资源和服务 积极采用其研究计划中猴子中的基因转移方法。压倒性目标是 履行对调查人员的服务的使命，并探索相关的基因传递中的关键问题 翻译非人类灵长类动物模型。我们的外展计划，服务和研究的成功 成就以及更大的研究社区的持续兴趣从事基因转移研究 在猴子中，展示了该中心的重要性以及在推进基因治疗领域的作用 人类疾病的治疗。该中心支持并为NHLBI资助的项目进行了项目 跨越美国机构的调查人员这些项目已经解决了基因的新方法 分娩在整个生命周期中，特别关注胎儿和婴儿，“原理证明”。 评估啮齿动物中的发现是否可以在非人类灵长类动物中显示，还是作为临床前进行 在一项新的临床试验之前逐步。此续签应用的具体目的包括：（1）提供 向NHLBI支持的研究人员提供专业知识，专业服务和资源，以探索基因 在猴子治疗人类疾病的猴子中转移策略，（2）开发技术以增强 资源并继续探索猴子的长期安全后交付后的长期安全。我们建立的 行政结构将在经过验证的协作框架中提供这些服务。调查人员会 可以通过竞争过程获得这些机会；通过 成立的科学咨询委员会。我们还将进一步开发特定于 与猴子的翻译研究为更大的研究社区，继续严格评估安全性， 并通过网站，公告和我们的年度基因疗法传达研究机会 心脏研讨会。肺和血液疾病。