Cell Manipulation Tools

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PROJECT SUMMARY/ABSTRACT - Cell Manipulation Tools Core (Tools) The recent characterization of the human genome combined with powerful new sequence-directed gene manipulation technologies have revolutionized our ability to ask biological questions, identify and manipulate disease causing alterations, and create targeted therapeutics. However, serious barriers still exist for individual researchers attempting to perform and assess genome manipulations. This is particularly true for applications involving human hematopoietic cell products, which require specialized knowledge and functional assays. The mission of the Tools Core is to act as an interface between cutting edge genome manipulation techniques and researchers and clinicians to provide access to protocols, reagents, and consultants for genetic alteration of human and canine hematopoietic cell products. The ultimate goal of the Tools Core is to break down access barriers and provide appropriate infrastructure and support for functional genetic and viral vector technology platforms in hematopoietic cell products. The Tools Core will be directed by Drs. Kiem and Paddison who have outstanding track records in the fields of viral vector-mediated gene therapy, functional genomics, gene editing, vector production, and stem and progenitor cell manipulation. Functions of the Tools Core include provision of reagents for user-performed genome manipulations, best practices protocols, consultations for manipulation of hematopoietic cell products, gene edited cell products, and development of new technology to improve gene editing and viral vector performance.