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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

基本信息

批准号：
8403719
负责人：
Alice F Tarantal
金额：
$ 97.99万
依托单位：
UNIVERSITY OF CALIFORNIA AT DAVIS
依托单位国家：
美国
项目类别：
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/8403719
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 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

项目摘要

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.

胚胎猴心脏基因转移中心的目标。肺、血疾病都是要进行的