Several forms of stem cell modificafion are currently envisioned that will facilitate the development of the next generafion of cell-based therapeutics. Integrafing viral vectors are commonly used to permanently and most efficienfiy insert genes of interest, or to introduce libraries of genes to conduct screens. A current major research emphasis and area of expertise of invesfigators at CHOP is to characterize genetic disorders in stem cells. One of the next areas of high priority of research will be to develop methods to correct monogenic genefic disorders. For this purpose, ZFNs are currenfiy the only method that has sufficient efficiency to modify stem cells at levels necessary to support therapy. We will be providing these services in a fimely, cost-efficient manner that will provide high quality large-scale products that avoids the need for each investigator to develop the necessary skill sets. By providing the best state-of-the-art backbone for the vectors and the latest in ZFN technology we anticipate that we will not only allow better standardization of product on the UPENN/CHOP campus, but also enhance the quality of available products, leading to enhanced producfivity and synergy on campus in the field of benign hematopoiesis.
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