Vector Core-Core 2

抽象/摘要矢量核心（核心2） 爱荷华州大学的Vector Core服务于多个针对囊性纤维化研究的项目， 治疗发展。与来自不同学科的多名研究人员的互动允许交叉- 在载体设计方面的思想、技术进步和创新的丰富。的总体目标 Vector Core旨在支持研究人员生成和实施基因递送和基因编辑 技术.这包括与主要研究者协商，开发新型载体， 常规载体制备。核心工作人员和调查人员在矢量的所有阶段都保持密切联系 设计和生成。因此，核心既是基因转移的研究和开发设施， 研究和常规病媒制备服务设施。核心提供纯净和浓缩的 重组腺病毒、腺相关病毒（AAV）、牛痘、杆状病毒和逆转录病毒的制备 （包括慢病毒）。这些载体已被证明在递送治疗性转基因或CRISPR方面是成功的。 应用于气道细胞。其他服务包括获得标准细胞系，表达 质粒和报告病毒的储备。 Vector Core的主要职责是： ·病毒载体生产 ·研究与开发 ·教育 ·材料档案 在气道上皮中用于基因替换或基因组工程策略的递送载体可以赋予长期的 CF表型的长期校正。此外，此类载体可用于假设驱动的研究 例如标记气道内的祖细胞。在方法上取得了重要进展， 拯救CFTR蛋白功能。然而，目前还没有针对最常见突变的类似疗法。 Vector Core的目标是为致力于开发终身基因的研究人员提供支持 替代策略将是有效的，而不管引起疾病的突变。核心 专门从事基因转移和基因编辑技术，为主要研究人员提供 用于治疗基因如CFTR的载体。这个核心产生的试剂、方法和指导 提供一个重要的服务，解决CF治疗。

Abstract/Summary Vector Core (Core 2) The Vector Core at the University of Iowa serves multiple projects directed at the study of cystic fibrosis and therapy development. The interaction with multiple investigators from various disciplines allows for cross- fertilization of ideas, technical advancements, and innovations in vector designs. The overall objective of the Vector Core is to support investigators in generating and implementing gene delivery and gene editing technologies. This includes consultation with the Principle Investigator, development of novel vectors, and routine vector preparation. The Core staff and investigators are in close contact through all phases of vector design and generation. Thus, the Core serves as both a research & development facility for gene transfer studies and a service facility for routine vector preparations. The Core provides purified and concentrated preparations of recombinant adenovirus, adeno-associated virus (AAV), vaccinia, baculovirus and retrovirus (including lentivirus). These vectors have proven success at delivering therapeutic transgenes or CRISPR- based technologies to airway cells. Additional services include access to standard cell lines, expression plasmids, and stocks of reporter viruses. The main responsibilities of the Vector Core will be: · Viral Vector Production · Research and Development · Education · Materials Archive Delivery vectors for gene replacement or genome engineering strategies in airway epithelia could confer long- term correction of the CF phenotype. In addition, such vectors can be used for hypothesis driven research such as labeling progenitor cells within the airways. There have been important advancements in approaches to rescue CFTR protein function. However similar therapies for the most common mutation is not yet available. The goal of the Vector Core is to provide support for researchers working to develop a life-long gene replacement strategy that would be efficacious regardless of the disease causing mutation. The Core specializes in gene transfer and gene editing technologies that provide Principle Investigators with delivery vehicles for therapeutic genes such as CFTR. The reagents, methods, and guidance generated by this core provide an important service addressing CF therapies.