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

胎猴心脏、肺和血液疾病基因转移中心

• 批准号: 7137758
• 负责人: Alice F Tarantal
• 金额: $ 95.41万
• 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 2011-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7137758
• 关键词: Lentivirus; Macaca mulatta; adeno associated virus group; blood disorder; embryo /fetus therapy; enzyme linked immunosorbent assay; flow cytometry; gene expression; gene therapy; genetic promoter element; genetic transduction; gestational age; heart disorder; immune tolerance /unresponsiveness; immunocytochemistry; longitudinal animal study; lung disorder; neutralizing antibody; nonhuman therapy evaluation; polymerase chain reaction; transfection /expression vector; western blottings

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.

描述（由申请人提供）： 胎儿猴子基因转移中心的心脏，肺和血液疾病的目标是：（1）为研究社区提供可访问的猴子模型，以评估基因治疗策略出现的安全性和效率； （2）为NHLBI资助的研究人员提供基本的专业知识，资源和服务，以在其研究计划中积极采用猴子的基因转移方法。这种更新的重要目标是继续履行我们的中心使命，即在相关的临床前非人类灵长类动物模型中向研究人员提供基本服务，以探索基因传递中的关键问题。我们的外展计划，研究和服务成就以及更大的研究社区的支持的成功表明了该中心的重要性以及我们提供的服务需要推进基因治疗领域以治疗人类疾病。我们每年通过竞争过程为调查人员提供资金，并提供了广泛的服务和培训。我们还探索了对该领域必不可少的胎儿基因传递中的一系列基本问题。我们的下一个融资期的目标和具体目标包括目标1，专注于为NHLBI资助的研究人员提供专业知识和专业服务，以研究猴子治疗人类疾病的基于基因的方法。具体目的1解决了中心的行政结构和信息的传播，具体目的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，他们希望评估其猴子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）将基因转移到胎儿猴子中以治疗心脏，肺和血液疾病的效率和安全性。将进行这些研究以扩大我们的能力并为研究人员提供更多的研究机会。特定目标1的重点是长期监测基因表达的体内成像技术，而特定的目标2专注于评估安全性的方法。胎儿猴子基因转移中心具有独特的能力，能够解决胎儿，婴儿和少年非人类灵长类动物的基因递送中的基本问题，并始终为研究人员提供了广泛的机会，以促进发展有希望的新疗法以治疗人类疾病。