CORE--VECTOR PRODUCTION

说明：（直接取自应用程序） 载体生产核心旨在提供腺病毒载体的批量合成。 和质粒DNA载体用于基因转移的临床前研究。我们有 确定了大规模制备这些载体的实质性需求， 我们的校园我们还计划开发改进的技术， 复制型腺病毒（RCA）从批量制备，并提高 质粒产量。 病媒生产核心设施的主要作用是协助 随着早期实验室概念验证类型研究和 为了支持临床前研究， （主要是动物）基因治疗试验。的数量和质量 猪或非人灵长类动物研究所需的临床前级载体，或 即使是在小鼠、大鼠或兔子身上进行详细的研究， 单个实验室尝试验证特定 构建体因此，我们中心的矢量生产核心要求在 以下基础。首先，一些UAB教师感兴趣的囊性 纤维化或其他基因转移实验表明， 腺病毒或质粒批量制剂的大规模生产。二是 准备和大规模生产的标准化格式将有助于 制剂的重现性和一致性。第三，问题不 通常由一个调查员来管理的实验室可以最好地解决在 一个集中的设施，如存在复制主管 腺病毒（RCA）或质粒DNA产率的优化。最后，虽然不是 在正式的良好生产规范（GMP）条件下生产， 设施中的文档和记录保存可能有助于跟踪任何变化 在不同的载体制剂中观察到的反应中， 优化未来的媒介修复。此外，良好实验室规范 (GLP)设施中使用的条件将使核心B中产生的载体 对于人类临床试验的开发有用且相关， 基因治疗

Description: (Taken directly from the application) The Vector Production Core is intended to provide batch synthesis of adenoviral and plasmid DNA vectors for preclinical studies of gene transfer. We have identified a substantial need for large scale preparation of these vectors on our campus. We also plan development of improved techniques to minimize replication competent adenovirus (RCA) from batch preparations, and to improve plasmid yield. The principal role of the Vector Production Core Facility will be to assist with the transition between early laboratory proof-of-concept type studies and the scale-up production that becomes crucial in order to support preclinical (predominantly animal) gene therapy trials. The amounts and quality of preclinical grade vector needed for studies of pigs or non-human primates, or even for detailed studies in mice, rats, or rabbits are often well beyond the capabilities of an individual laboratory attempting to validate a particular construct. A Vector Production Core at our center is, therefore, requested on the following basis. First, a number of UAB faculty interested in cystic fibrosis or other gene-transfer experiments have indicated a need for the large-scale production of adenovirus or plasmid batch preparations. Second, a standardized format of preparative and large-scale production will facilitate the reproducibility and consistency of the preparations. Third, issues not normally managed in a single investigator?s laboratory can be best addressed in a centralized facility, such as the presence of replication competent adenovirus (RCA), or optimization of plasmid DNA yields. Finally, although not produced under formal, good manufacturing practice (GMP) conditions, the documentation and record keeping in the facility may help track any variability in the responses observed with different vector preparations and may also help optimize future vector reparations. Moreover, the good laboratory practice (GLP) conditions used in the facility will make vectors produced in Core B useful and relevant with regards to development of human clinical trials of gene therapy.