Gene Therapy for Nonneoplastic Hematologic and Histiocytic Disorders

  • Kareem N. Washington
  • John F. Tisdale
  • Matthew M. Hsieh
Part of the Molecular Pathology Library book series (MPLB, volume 4)


Over the past decades, gene therapy as a tool to cure disease has blossomed into an exciting clinical possibility. Gene transfer technologies have progressed immensely since the field’s inception (Figure 45.1). The first replication-defective retroviral vector was described in 1983, and by 1990 clinical studies using retroviral vectors began. Although initial efforts were robust, by 1996, none of the clinical gene “therapy” trials had shown clinical efficacy. After a change in policy and focus instituted by a panel convened by the National Institutes of Health, efforts to develop gene transfer technologies shifted toward targeting diseases that were early and obvious targets for therapeutic intervention. The simultaneous experimental advances in our understanding of hematopoiesis and the significant advances in gene transfer technology have led to success in humans, at least for disorders requiring modest transfer rates of genes not requiring complex regulation to hematopoietic stem cell (HSC) targets.


Chronic Granulomatous Disease Fanconi Anemia Insertional Mutagenesis Sleep Beauty Gene Therapy Trial 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.


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Copyright information

© Springer Science+Business Media, LLC 2010

Authors and Affiliations

  • Kareem N. Washington
    • 1
  • John F. Tisdale
    • 1
  • Matthew M. Hsieh
    • 1
  1. 1.NHLBI-NIDDK-MCHBNational Institutes of HealthBethesdaUSA

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