9 - Exogenous Gene Transfer into Lymphoid and Hematopoietic Progenitor Cells

Abstract

Hematopoiesis is the tightly regulated process of cell proliferation and maturation that results in the production of terminally differentiated cell types, which are derived from hematopoietic stem cells. These have the potential to give rise to progeny cell differentiation along any of the cell-lineage pathways that is summarized in this chapter. The chapter also discusses the retroviral vectors that have been developed for gene transfer into human hematopoietic cells and are based on the Moloney murine leukemia virus (MoMLV) backbone. Moreover, the chapter outlines the use of lymphoid cells as targets for gene transfer for hereditary diseases. This helps in the treatment of leukocyte adhesion or acquired diseases such as acquired immunodeficiency syndrome (AIDS). Finally, the chapter concludes by presenting a few future challenges for gene therapy. These include the identification of the optimal cell type, growth and transduction conditions, and gene transfer vectors. It is not inconceivable that medical treatment of the future will have cell therapy as part of its armament and though it is not clear how soon that will be, the pace of advance indicates that it will indeed come.