Hematopoietic stem cell gene therapy with drug resistance genes: an update.
Transfer of drug resistance genes into hematopoietic stem cells (HSCs) has promise for the treatment of a variety of inherited, that is, X-linked severe combined immune deficiency, adenosine deaminase deficiency, thalassemia, and acquired disorders, that is, breast cancer, lymphomas, brain tumors, and testicular cancer. Drug resistance genes are transferred into HSCs either for providing myeloprotection against chemotherapy-induced myelosuppression or for selecting HSCs that are concomitantly transduced with another gene for correction of an inherited disorder. In this review, we describe ongoing experimental approaches, observations from clinical trials, and safety concerns related to the drug resistance gene transfer.
Budak-Alpdogan
Tulin
T
Department of Medicine, The Cancer Institute of New Jersey, Robert Wood Johnson Medical School, University of Medicine and Dentistry of New Jersey, 195 Little Albany Street, New Brunswick, New Jersey 08903, USA.
Banerjee
Debabrata
D
Bertino
Joseph R
JR
D002986:Clinical Trials as Topic; D004351:Drug Resistance / Q000235:genetics*; D018014:Gene Transfer Techniques; D040181:Genetic Diseases, X-Linked / Q000235:genetics / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D009369:Neoplasms / Q000235:genetics / Q000628:therapy