NHLBI GTRP Preclinical Vector Production Core Laboratory

NHLBI基因治疗资源计划(GTRP)促进了将基因治疗研究转化为心、肺和血液疾病的临床干预。 GTRP以临床前和临床级载体生产、药理学/毒理学测试、临床试验资金援助和监管支持的形式向NHLBI研究人员提供资源。调查人员提交服务申请(RSA)以访问这些资源。 GTRP由NHLBI基因治疗小组指导，由三个载体生产核心、药理学/毒理学测试核心和临床协调中心组成。科学审查委员会和指导委员会审查服务应用程序的请求，并就应用程序的科学价值、可行性和与计划使命的兼容性向NHLBI基因治疗小组提出建议。

The NHLBI Gene Therapy Resource Program (GTRP) facilitates the translation of gene therapy research into clinical interventions for heart, lung, and blood diseases. The GTRP provides resources to NHLBI investigators in the form of preclinical and clinical-grade vector production, pharmacology/toxicology testing, clinical trials funding assistance, and regulatory support. Investigators submit a Request for Service Application (RSA) to access these resources. The GTRP, directed by the NHLBI Gene Therapy Group, consists of three vector production cores, a pharmacology/toxicology testing core, and a clinical coordinating center. A Scientific Review Board and Steering Committee review Request for Service Applications and make recommendations to the NHLBI Gene Therapy Group regarding the applications' scientific merit, feasibility, and compatibility with the program's mission.