Vector Core-Core 2

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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.