Several forms of stem cell modification are currently envisioned that will facilitate the development of the next generation of cell-based therapeutics. Integrating viral vectors are commonly used to permanently and most efficiently insert genes of interest, or to introduce libraries of genes to conduct screens. A current major research emphasis and area of expertise of investigators 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 genetic disorders. For this purpose, ZFNs are currently 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 timely, 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 productivity and synergy on campus in the field of benign hematopoiesis.
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