IGF::OT::IGF - GTRP CCC - Task Area B -Clinical Trials Funding; FY14; 8470187; AMT $2,162,750

NHLBI基因治疗资源计划（GTRP）更新的目标是继续支持基因治疗的基础研究转化为心脏、肺和血液疾病领域的临床应用。GTRP的更新将继续根据良好生产规范（GMP）在临床前和临床级载体生产领域提供资源，并进行药理学/毒理学研究。此外，GTRP的更新将为希望进行基因治疗临床试验的研究人员提供资金和监管援助。GTRP更新将由以下五个部分组成：1)一个临床协调中心，负责监督和协调核心实验室的后勤工作，并为临床试验提供监管和资金援助；2)临床前级媒介生物生产核心实验室；3)腺相关病毒（AAV）临床级载体生产核心实验室；4)慢病毒临床级载体生产核心实验室；5)药理学/毒理学核心实验室。

The objective of the NHLBI Gene Therapy Resource Program (GTRP) renewal is to continue supporting the translation of basic research in gene therapy to clinical application in the areas of heart, lung, and blood diseases. The GTRP renewal will continue to provide resources in the areas of preclinical and clinical-grade vector production in accordance with Good Manufacturing Practices (GMP), and in the conduct of pharmacology/toxicology studies. In addition, the GTRP renewal will provide funds and regulatory assistance to investigators wishing to conduct gene therapy clinical trials. The GTRP renewal will consist of the following five components: 1) a clinical coordinating center that will oversee and coordinate the logistics of the core laboratories and provide regulatory and funding assistance for clinical trials; 2) a preclinical-grade vector production core laboratory; 3) a clinical-grade vector production core laboratory for adeno-associated virus (AAV); 4) a clinical-grade vector production core laboratory for lentivirus; and 5) a pharmacology/toxicology core laboratory.