? CORE B The main function of Vector Development Core (VDC) B is to develop new lentiviral vectors (LV) and the means for their production in order to facilitate their use in clinical trials proposed in Projects 1, 2, and 3. Core B will achieve this aim by 1) producing small-scale vectors including LV, IDLV, and AAV for preclinical testing; 2) exploring new methodologies for vector manufacturing such as transient transfection in suspension cells and stable cell line production systems; 3) developing next generation producer cell lines from suspension cells growing in serum-free media; 4) developing process for scale-up manufacturing that bridges laboratory research to cGMP production; 5) optimizing protocols for LV transduction of human hematopoietic stem cells (HSCs). As such, Core B provides a critical link between the Projects and cGMP production in Core C by performing developmental studies, by providing the scientific expertise for process transfer, and by providing novel and unique reagents, vectors, and cell lines for these purposes. These research activities in Core B will ultimately integrate into all projects for improved vector manufacturing and cell transduction.
? CORE B Core B is essential to develop lentiviral vectors for clinical gene therapy of sickle cell disease and novel platform using suspension-based vector manufacturing that will increase potency of vectors and reduce manufacturing costs.
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