Hematopoietic stem cell gene therapy with drug resistance genes: An update

Tulin Budak-Alpdogan, Debabrata Banerjee, Joseph R. Bertino

Research output: Contribution to journalArticlepeer-review

25 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)849-863
Number of pages15
JournalCancer gene therapy
Issue number11
StatePublished - Nov 2005

All Science Journal Classification (ASJC) codes

  • Molecular Medicine
  • Molecular Biology
  • Cancer Research


  • Drug resistance genes
  • Gene transfer
  • Hematopoietic stem cells
  • Oncoretrovirus


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