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.

Public Health Relevance

Gene transfer could replace defective or missing genes for therapeutic benefit provided that such methods are carefully tested and found to be safe. The focus of this application is to continue to ensure the resources, expertise, and services necessary for NHLBI-supported investigators to assess new gene transfer strategies in a primate model are available to provide the preclinical data necessary for future human clinical trials.

National Institute of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
Resource-Related Research Projects (R24)
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Special Emphasis Panel (ZHL1-CSR-S (O2))
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Mcdonald, Cheryl
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University of California Davis
Anatomy/Cell Biology
Schools of Medicine
United States
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Corti, Manuela; Cleaver, Brian; Clément, Nathalie et al. (2015) Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning. Hum Gene Ther Clin Dev 26:185-93
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