The Scope of Viral Vectors for the Transduction of Haemopoietic Cells.

Hematology (Amsterdam, Netherlands)

PubMedID: 27416282

Lea N, Mufti GJ. The Scope of Viral Vectors for the Transduction of Haemopoietic Cells. Hematology. 2016;3(1):37-53.
Over the last five years significant progress has been made towards the transfer of foreign genetic material to eukaryotic cells. The eventual aim to devise novel therapeutic strategies to treat human diseases, in particular solid tumours and monogenic disorders associated with various enzyme deficiency states. The easy accessibility and the ability of haemopoetic stem cells to self replicate and repopulate makes them desirable targets for gene transfer. In theory the introduction of a small number of gene modifed haemopoetic progenitor cells can allow therapy of an individual for life without any further intervention. This approach has been used for the treatment of single gene defects such as ADA deficiency. Furthermore, gene transfer technology has increasingly been exploited for bone marrow and peripheral blood stem cell marking studies, modification of cell sensitivity to cytotoxic drugs and the genetic modification of leukemic cells with the intention of inducing a leukemia specific cytotoxic T cell response. Vector development is of crucial importance for the successful delivery of genes in haemopoetic stem cells and leukemia cells. The objective of this review is to discuss in detail the properties of current vector technology that are pertinent to haemopoietic cell gene transduction.