The Vector Core has been and will continue to be a critical component of the Model Systems for Gene Therapy Program, interacting with and providing reagents to all of the proposed projects. The Vector Core will function within the framework of the Program has a dynamic resource that can provide state-of-the-art viral vector technology as well as develop noel vectors. The major emphasis of the Vector core has been on the utilization of MLV-based retroviral and adenoviral vectors for gene transduction. The major emphasis of the Vector core has been on the utilization of MLV-based retroviral and adenoviral vectors for gene transduction. However, the Vector Core also is developing and providing stocks of high titer HIV-based lentiviral vectors. The role of the Vector Core in the Model Systems for Gene Therapy Program will be to construct and provide vectors expressing the appropriate genes for the proposed experiments. In addition, the core will provide cell lines, viruses, packaging lines, plasmids, and protocols as needed to each of the five projects. The Core also will work with the 5 projects and Core B to optimize viral and non-viral gene delivery and gene expression. Furthermore, the Core will provide technical assistance and training to individuals in the use of viral and non-viral vectors for gene transfer as needed.
The specific aims of the Vector Core are: 1) To provide each investigator with the appropriate viral vector or plasmid, expressing the required therapeutic or marker ge4ne, for their proposed viral vector or plasmid, expressing the required therapeutic or marked gene, for their proposed experiments; 2) To develop improved viral vectors for more efficient gene transfer to islets, neuronal cells, and inflammatory cells in the joint with higher and/or regulated gene expression; and (3) To provide technical assistance, regents, and protocols to each of the projects in the use of gene delivery systems.
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