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

胚胎猴心脏基因转移中心的目标。肺病、血病都要进行 对猴子进行基因转移研究，以评估基因治疗策略的安全性和有效性 并向NHLBI支持的调查人员提供必要的专业知识、资源和服务，以 在研究项目中积极探索猴子的基因转移方法。压倒一切的目标是 履行为调查人员服务的使命，探索相关的基因传递中的关键问题 翻译非人灵长类动物模型。我们外展计划、服务和研究的成功 成就，以及更大的研究界对进行基因转移研究的持续兴趣 证明了该中心的重要性和在推进癌症基因治疗领域中的作用 人类疾病的治疗。该中心支持并实施了NHLBI资助的项目 横跨美国各地机构的研究人员。这些项目解决了基因研究的新方法 缩短生命周期的分娩，特别关注胎儿和婴儿，这是一种“原则证明”。 评估啮齿动物的发现是否可以在非人类灵长类动物身上显示，或者作为临床前研究进行 为一项新的临床试验提前一步。是次续期申请的具体目的包括：(1)提供 为NHLBI支持的研究人员探索基因提供专业知识、专门服务和资源 用于治疗人类疾病的猴子移植战略，以及(2)开发技术，以增强 并继续探索在猴子体内长期安全的基因后传递。我们老牌的 行政结构将在经过验证的协作框架中提供这些服务。调查人员将 通过竞争过程获得这些机会；项目是通过 成立了科学咨询委员会。我们还将进一步开发专门针对 为更大的研究社区对猴子进行的翻译研究，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交流研究机会 心脏研讨会。肺病和血液病。

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.