International Journal of Hematology

, Volume 75, Issue 3, pp 228–236 | Cite as

Hematopoietic Stem Cell Gene Therapy

  • David W. Emery
  • Tamon Nishino
  • Ken Murata
  • Michalis Fragkos
  • George Stamatoyannopoulos
Progress in Hematology


Gene therapy applications that target hematopoietic stem cells (HSCs) offer great potential for the treatment of hematologic disease. Despite this promise, clinical success has been limited by poor rates of gene transfer, poor engraftment of modified cells, and poor levels of gene expression. We describe here the basic approach used for HSC gene therapy, briefly review some of the seminal clinical trials in the field, and describe several recent advances directed toward overcoming these limitations.

Key words

Gene therapy Hematopoiesis Stem cells Virus vectors 


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Copyright information

© The Japanese Society of Hematology 2002

Authors and Affiliations

  • David W. Emery
    • 1
  • Tamon Nishino
    • 1
  • Ken Murata
    • 1
  • Michalis Fragkos
    • 2
  • George Stamatoyannopoulos
    • 1
  1. 1.Division of Medical GeneticsUniversity of Washington Department of MedicineSeattle, WashingtonUSA
  2. 2.Department of Basic SciencesUniversity of Crete School of Medicine and Institute of Molecular Biology and BiotechnologyHeraklion, CreteGreece

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