Core D: Genetic Engineering and Gene Therapy Core

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The mission ofthe Genetic Engineering and Gene Therapy (GEGT) Core is to work cooperatively with Liver Research Center (LRC) Investigators to facilitate the development of genetic engineering reagents for in vitro and in vivo experiments. The specific aims are to (1) generate customized oncoretroviral, lentiviral, adenoviral and non-viral vectors with integrating or episomal characteristics; (2) provide state-of-the-art reagents for site-specific gene insertion for cell marking, phenotypic correction/modification or down-regulating gene expression; (3) provide viral and non-viral vectors for reprogramming somatic cells to induced pluripotent stem (IPS) cells; (4) when appropriate, train investigators and their research personnel in generating gene transfer vectors in their own laboratories; (5) provide consultation for study design and, if appropriate, data analysis to the investigators. To fulfill these needs, the GEGT is established through a cooperative cost-sharing arrangement with the Gene Therapy Core of the Albert Einstein College of Medicine. The GEGT Core will function in close collaboration with other LRC Cores, as well as other institutional Shared Facilities, but the specialized services of the GEGT do not overlap with those offered by other Cores. The Core will benefit from the 25-yr experience of the Scientific Director, Jayanta Roy- Chowdhury, Professor of Medicine and Genetics in gene transfer in vivo and ex vivo for pathobiological and translational research, as well as the significant technological expertise of the Operations Director, Dr. Xia Wang. GEGT Core will add value to LRC investigators by enabling them to generate and use vectors and reagents that would be prohibitively expensive or technically difficult for individual laboratories to develop. It will also give LRC investigators priority for service and discounted chargeback rates. Resource sharing with other LRC Cores, as well as other institution-wide shared facilities will allow the Core to provide its services in an effective and cost-efficient manner. By supporting the multifaceted pathophysiological and translational research projects of the LRC members, as well as extramural investigators, the Core will be highly relevant to healthcare-related liver research.