In cell-based gene transfer, a patient's autologous cells are harvested, transduced in vitro with the appropriate vector, and reintroduced. As with other gene transfer methods, this approach offers both advantages and disadvantages. The use of autologous cells reduces the likelihood of immune responses. Furthermore, the in vitro phase of the procedure allows for the generation of a homogeneous population of transduced cells that can easily be assessed for transfection and expression efficiencies. However, the in vitro techniques utilized cause an unavoidable delay resulting from the harvest of the cells, genetic modification, screening, propagation, and reintroduction. Additionally, pheno-
typical changes in cells can occur during culture, the harvest and implantation steps each require individual invasive techniques, and the implantation step may in itself result in damage to the target site. Taken together, these drawbacks may underlie the relative paucity of clinical studies based on this approach. Nonetheless, cell-based gene transfer has been successfully used in a rodent model of vascular injury (3) and a murine model of hypercholesterolemia and atherosclerosis (4). Moreover, a pilot study utilizing cell-based gene transfer of the low-density lipoprotein (LDL) receptor in humans found a significant reduction in total cholesterol levels in individuals who have familial hyper-cholesterolemia (5). Thus it may be the case that further optimization of the techniques involved in cell-based genetic modification may yet result in more widespread use of this approach as a gene therapy tool.
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