Background and update to a phase I trial of stem cell gene marking in multiple myeloma

Abstract

Attractive target cells in gene transfer approaches to cancer treatment are pluripotent hematopoietic stem cells (HSCs) because of their high proliferative potential, self renewal and the wide systemic distribution of their progeny. A retroviral-mediated approach to gene transfer into HSCs is currently favored because the transduced gene is permanently integrated into the genome of the target cell resulting in the long-term stable expression of the gene product. Unfortunately, this approach requires that the target cell be in cycle and thus retroviral transduction of normally quiescent HSCs generally occurs at low efficiency. In previous work we have demonstrated that reinfused genetically m..