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Hematopoietic Stem Cell Gene Therapy

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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.

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Correspondence to David W. Emery or Tamon Nishino or Ken Murata or Michalis Fragkos or George Stamatoyannopoulos.

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Emery, D.W., Nishino, T., Murata, K. et al. Hematopoietic Stem Cell Gene Therapy. Int J Hematol 75, 228–236 (2002). https://doi.org/10.1007/BF02982035

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Key words

  • Gene therapy
  • Hematopoiesis
  • Stem cells
  • Virus vectors