Adoptive transfer of genetically modified hematopoietic stem cells

Ian D. Dubé, A. Keith Stewart

Research output: Contribution to journalArticlepeer-review

Abstract

Adoptive transfer of bone marrow derived blood forming stem cells is a well-recognized treatment option for some human genetic diseases. In this therapeutic approach a relatively small number of donor marrow cells engraft and proliferate extensively in the recipient. Arising blood cell progeny have the potential to provide the host with therapeutic levels of a desired gene product for at least two decades, quite possibly a lifetime. A long-standing challenge in human blood cell gene therapy has been progression from adoptive transfer of allogeneic stem cells which are inherently genetically 'correct', to adoptive transfer of autologous stem cells that have been specifically genetically 'corrected'. The progress in meeting this challenge will be reviewed. A promising approach for adoptive transfer of hematopoietic stem cells genetically modified in long-term marrow cultures will be presented and discussed.

Original languageEnglish (US)
Pages (from-to)137-145
Number of pages9
JournalTransfusion Science
Volume17
Issue number1
DOIs
StatePublished - Jan 1 1996

ASJC Scopus subject areas

  • Immunology
  • Hematology

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