¹Division of Hematology/Oncology, Department of Medicine, Vanderbilt University, Nashville, TN, USA

Correspondence: Professor FG Schuening, Division of Hematology/Oncology, Department of Medicine, Vanderbilt University, 777 PRB, 2220 Pierce Avenue, Nashville, TN 37232, USA. E-mail: friedrich.schuening@vanderbilt.edu

²These authors contributed equally to this review.

Received 18 May 2005; Revised 8 August 2005; Accepted 12 August 2005; Published online 7 October 2005.

Abstract

A major side effect of cancer chemotherapy is myelosuppression. Expression of drug-resistance genes in hematopoietic stem cells (HSC) using gene transfer methodologies holds the promise of overcoming marrow toxicity in cancer chemotherapy. Adequate protection of marrow cells in cancer patients from myelotoxicity in this way would permit the use of escalating doses of chemotherapy for eradicating residual disease. A second use of drug-resistance genes is for coexpression with a therapeutic gene in HSCs to provide a selection advantage to gene-modified cells. In this review, we discuss several drug resistance genes, which are well suited for in vivo selection as well as other newer candidate genes with potential for use in this manner.