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

描述（由申请人提供）： 该中心的目标为胎猴基因转移心脏，肺和血液疾病是：（1）提供可访问的猴子模型的研究社区，以评估安全性和有效性的基因治疗策略，因为他们出现;和（2）提供NHLBI资助的研究人员与必要的专业知识，资源和服务，积极追求基因转移方法在猴子在他们的研究计划。此次更新的首要目标是继续履行我们中心的使命，即为研究人员提供基本服务，以探索相关临床前非人灵长类动物模型中基因递送的关键问题。我们的外展计划，研究和服务成就的成功，以及来自更大的研究界的支持表明了该中心的重要性，以及我们为推进人类疾病治疗基因治疗领域所提供的服务的必要性。我们每年都通过竞争程序资助调查人员，并提供广泛的服务和培训。我们还探讨了一系列的基本问题，在胎儿基因传递领域至关重要。我们下一个资助期的目标和具体目标包括目标1，重点为NHLBI资助的研究人员提供专业知识和专业服务，以研究基于猴子的基因方法治疗人类疾病。具体目标1解决了中心的行政结构和信息传播，具体目标2强调了我们将向NHLBI资助的研究人员提供的专业知识，服务和资源，这些研究人员希望评估他们在猴子中的病毒和非病毒基因转移策略。在目标2中，我们将评估重组病毒载体系统（慢病毒，AAV）在将基因转移到胎猴中以治疗心脏、肺和血液疾病中的效率和安全性。这些研究将扩大我们的能力，并为研究人员提供更多的研究机会。具体目标1侧重于长期监测基因表达的相关体内成像技术，具体目标2侧重于评估安全性的方法。胎猴基因转移中心在解决胎儿，婴儿和青少年非人灵长类动物基因递送中的基本问题方面具有独特的能力，并一直为研究人员提供广泛的机会，以促进有前途的新疗法的开发用于治疗人类疾病。

DESCRIPTION (provided by applicant): The goals of the Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases are: (1) to provide accessible monkey models to the research community in which to evaluate the safety and efficiency of gene therapy strategies as they emerge; and (2) to provide NHLBI-funded investigators with essential expertise, resources, and services to actively pursue gene transfer approaches in monkeys in their research programs. The overriding objective of this renewal is to continue to fulfill our Center mission of providing essential services to investigators to explore crucial issues in gene delivery in a relevant preclinical nonhuman primate model. The success of our outreach program, research and service accomplishments, and support from the greater research community demonstrates the importance of this Center and the need for the services we provide to advance the field of gene therapy for the treatment of human disease. We have funded investigators annually through a competitive process and provided extensive services and training. We have also explored a series of basic questions in fetal gene delivery essential to the field. Our Objectives and Specific Aims for the next funding period include Objective 1, to focus on providing expertise and specialized services to NHLBI-funded investigators for the study of gene-based approaches in monkeys for the treatment of human disease. Specific Aim 1 addresses the Center administrative structure and dissemination of information, and Specific Aim 2 highlights the expertise, services, and resources we will provide to NHLBI-funded investigators who wish to evaluate their viral and non-viral gene transfer strategies in monkeys. In Objective 2, we will evaluate the efficiency and safety of recombinant viral vector systems (lentivirus, AAV) in transferring genes into fetal monkeys for the treatment of heart, lung, and blood diseases. These studies will be performed to expand our capabilities and provide more research opportunities for investigators. Specific Aim 1 focuses on correlative in vivo imaging techniques for monitoring gene expression long-term, and Specific Aim 2 focuses on methods to assess safety. The Center for Fetal Monkey Gene Transfer is unique in the ability to address essential questions in gene delivery in fetal, infant, and juvenile nonhuman primates, and has consistently provided investigators with extensive opportunities to advance the development of promising new therapies for the treatment of human diseases.