Vector Core-Core 2

摘要/摘要向量核心(核心2) 爱荷华大学的载体核心服务于多个针对囊性纤维化和 治疗的发展。与来自不同学科的多名调查人员的互动允许交叉- 思想的结晶、技术的进步和矢量设计的创新。《公约》的总体目标 载体核心是支持研究人员生成和实施基因传递和基因编辑 技术。这包括与首席调查员协商，开发新的媒介，以及 常规的载体准备。核心工作人员和调查人员在VECTOR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 研究和例行病媒准备的服务设施。核心提供净化和浓缩 重组腺病毒、腺相关病毒、痘苗病毒、杆状病毒和逆转录病毒的制备 (包括慢病毒)。这些载体在运送治疗性转基因或CRISPR方面已被证明是成功的。 以呼吸道细胞为基础的技术。其他服务包括获得标准细胞系、表达 质粒和报告病毒的库存。 向量核心的主要职责是： ·病毒载体制作 ·研发 ·教育 ·材料档案 在呼吸道上皮细胞中，用于基因替换或基因组工程策略的递送载体可以提供长期的- Cf表型的术语校正。此外，这些向量可用于假设驱动的研究 例如标记呼吸道内的祖细胞。在方法上已经有了重要的进步 以挽救CFTR蛋白的功能。然而，对于最常见的突变，目前还没有类似的治疗方法。 载体核心的目标是为致力于开发终身基因的研究人员提供支持 无论引起突变的疾病是什么，替换策略都是有效的。《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 治疗基因的载体，如cftr。由这个核心产生的试剂、方法和指导 提供针对CF疗法的重要服务。

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.