CORE--GENE & VIRAL THERAPY

这一共享资源满足了促进从我们的基础工作台研究中涌现出来的有希望的基因和病毒疗法进入临床前和临床试验的迫切需求。它的设计符合食品和药物管理局的所有严格要求，包括能够提供足够的临床级材料来确定候选试剂的有效性、毒理学和药理学特性，以及监管过程的所有步骤的专业知识。实现这一目标的专业知识和设施通常在学术环境中找不到，导致在引入新的创造性和有前途的疗法的关键过程中出现延误或实际障碍，而不是得到制药业良好支持的小分子药物。基因和病毒治疗的支持 RESOURCE有三个组成部分，即病毒媒介生产实验室、毒理药理实验室和质量保证，它们以无缝的方式与每个研究人员合作，以满足每个项目的独特要求。病毒载体生产实验室拥有专门的设施和人员，用于大规模病毒生产、工艺开发和使用良好制造规范(GMP)生产临床级产品。毒理药理实验室拥有专门的设备和训练有素的人员，在AAALAC批准的梅奥诊所动物护理设施中使用良好实验室规范(GLP)在动物模型中设计和进行新生物产品的毒理学和药理学表征。已经成立了一个质量保证小组，以确保基因和病毒治疗的程序和结果的质量 支持资源。

This shared resource fills a critical need to facilitate the translation of promising gene and viral therapies emerging from our basic bench investigations to enter into preclinical and clinical trials. It has been designed to meet all the rigorous requirements of the Food and Drug Administration, including the ability to provide adequate clinical grade material to determine the efficacy, toxicological, and pharmacological characterization of the candidate agent, as well as expertise with all steps of the regulatory process. The expertise and facilities to achieve this are not often found in an academic setting, resulting in delays or actual blocks in the critical process of introducing new creative and promising therapies, other than small molecule drugs that are well supported by the pharmaceutical industry. The Gene and Virus Therapy Support Resource has three components, the Viral Vector Production Laboratory, the Toxicology Pharmacology Laboratory and Quality Assurance, which work together in a seamless manner in collaboration with each investigator to fulfill the unique requirements of each project. The Viral Vector Production Laboratory has the dedicated facilities and personnel for large-scale virus production, process development, and the manufacture of clinical grade product using Good Manufacturing Practices (GMP). The Toxicology Pharmacology Laboratory has specialized equipment and trained personnel to design and conduct toxicological and pharmacological characterization of novel biological products in animal models using Good Laboratory Practices (GLP) in the AAALAC-approved Mayo Clinic animal care facilities. A Quality Assurance unit has been formed to assure the quality of the procedures and results of the Gene and Virus Therapy Support Resource.