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Hematopoietic Stem Cell Gene Therapy

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Abstract

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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Authors and Affiliations

  1. Division of Medical Genetics, University of Washington Department of Medicine, Seattle, Washington, USA

    David W. Emery, Tamon Nishino, Ken Murata & George Stamatoyannopoulos

  2. Department of Basic Sciences, University of Crete School of Medicine and Institute of Molecular Biology and Biotechnology, Heraklion, Crete, Greece

    Michalis Fragkos

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  1. David W. Emery
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  2. Tamon Nishino
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  4. Michalis Fragkos
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  5. George Stamatoyannopoulos
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Correspondence to 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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