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

胎猴心脏基因转移中心的目标。肺和血液疾病是进行 在猴子中进行基因转移研究，以评估基因治疗策略的安全性和有效性， 并为NHLBI支持的调查人员提供必要的专业知识，资源和服务， 在他们的研究项目中积极寻求猴子的基因转移方法。首要目标是 完成为研究人员服务的使命，并在相关领域探索基因递送的关键问题。 翻译的非人灵长类动物模型。我们的推广计划、服务和研究的成功 取得的成就，以及更大的研究社区对进行基因转移研究的持续兴趣 在猴子中的实验证明了该中心的重要性以及在推进基因治疗领域中的作用， 人类疾病的治疗。该中心支持并开展了NHLBI资助的项目。 这些项目涉及基因治疗的新方法， 特别关注胎儿和婴儿的整个生命周期的分娩，“原则证明”。 评价啮齿类动物中的发现是否可以在非人灵长类动物中显示，或作为临床前研究进行 在一项新的临床试验之前本续期申请的具体目的包括：（1）提供 专业知识，专业服务和资源，以NHLBI支持的研究人员，以探索基因 在猴子中转移策略，用于治疗人类疾病，以及（2）开发技术， 资源，并继续探索长期安全的猴子基因后交付。我们既定 行政结构将在一个行之有效的合作框架内提供这些服务。调查人员将 通过竞争过程获得这些机会;项目通过 成立科学咨询委员会。我们还将进一步开发新技术， 为更大的研究社区进行猴子转化研究，继续严格评估安全性， 并通过网站，公告和我们的年度基因治疗交流研究机会 心的研讨会。肺和血液疾病。

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.