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Genetic modification of hematopoietic stem cells with nonviral systems: past progress and future prospects

Gene Therapy volume 12, pages S118–S130 (2005)Cite this article

Abstract

Serious unwanted complications provoked by retroviral gene transfer into hematopoietic stem cells (HSCs) have recently raised the need for the development and assessment of alternative gene transfer vectors. Within this context, nonviral gene transfer systems are attracting increasing interest. Their main advantages include low cost, ease of handling and large-scale production, large packaging capacity and, most importantly, biosafety. While nonviral gene transfer into HSCs has been restricted in the past by poor transfection efficiency and transient maintenance, in recent years, biotechnological developments are converting nonviral transfer into a realistic approach for genetic modification of cells of hematopoietic origin. Herein we provide an overview of past accomplishments in the field of nonviral gene transfer into hematopoietic progenitor/stem cells and we point at future challenges. We argue that episomally maintained self-replicating vectors combined with physical methods of delivery show the greatest promise among nonviral gene transfer strategies for the treatment of disorders of the hematopoietic system.

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Acknowledgements

This work was supported by research grants "Karatheodori 2003" (University of Patras, B112) and EPAN 2003 (EU via GSRT, SP-YB90) to AA.

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  1. Department of Biology, Faculty of Medicine, University of Patras, Patras, Greece

    E P Papapetrou & A Athanassiadou

  2. Hematology Division, Department of Internal Medicine, University Hospital of Patras, Patras, Greece

    E P Papapetrou & N C Zoumbos

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Papapetrou, E., Zoumbos, N. & Athanassiadou, A. Genetic modification of hematopoietic stem cells with nonviral systems: past progress and future prospects. Gene Ther 12 (Suppl 1), S118–S130 (2005). https://doi.org/10.1038/sj.gt.3302626

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