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Therapeutic applications for hematopoietic stem cell gene transfer

Nature Immunology volume 3, pages 318–321 (2002)Cite this article

Transfer of genes into HSCs could be used to treat a variety of diseases from AIDS to cancer. Here Bordignon and Roncarolo discuss the logistics and progress of HSC gene transfer technology for the treatment of different diseases.

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Figure 1: The advantages, limitations and risks associated with stem cell versus lymphocyte gene therapy in the treatment of ADA-deficient SCID.
Figure 2: The gene transfer protocol used to genetically engineer DCs to achieve permanent tumor antigen presentation.

References

  1. Kay, M. A., Glorioso, J. C. & Naldini, L. Nature Med. 7, 33–40 (2001).

    Article  CAS  Google Scholar 

  2. Takatoku, M. et al. J. Clin. Invest. 108, 447–455 (2001).

    Article  CAS  Google Scholar 

  3. Naldini, L. et al. Science 272, 263–267 (1996).

    Article  CAS  Google Scholar 

  4. Miller, N. & Whelan, J. Hum. Gene Ther. 8, 803–815 (1997).

    Article  CAS  Google Scholar 

  5. May, C. et al. Nature 406, 82–86 (2000).

    Article  CAS  Google Scholar 

  6. Bordignon, C. et al. Science 270, 470–475 (1995).

    Article  CAS  Google Scholar 

  7. Kohn, D. B. et al. Nature Med. 1, 1017–1023 (1995).

    Article  CAS  Google Scholar 

  8. Kohn, D. B. et al. Nature Med. 4, 775–780 (1998).

    Article  CAS  Google Scholar 

  9. Bordignon, C. Nature Med. 4, 19–20 (1998).

    Article  CAS  Google Scholar 

  10. Malech, H. L. et al. Proc. Natl Acad. Sci. USA 94, 12133–12138 (1997).

    Article  CAS  Google Scholar 

  11. Cavazzana-Calvo, M. et al. Science 288, 669–672 (2000).

    Article  CAS  Google Scholar 

  12. Buchschacher, G. L. Jr & Wong-Staal, F. Hum. Gene Ther. 12, 1013–1019 (2001).

    Article  CAS  Google Scholar 

  13. Rizzardi. G. P. & Pantaleo, G. in HIV Human Virus Guide (ed. Richman, D. D.) 300–310 (International Medical Press, London, 2002).

    Google Scholar 

  14. Mautino, M. R., Keiser, N. & Morgan, R. A. J. Virol. 75, 3590–3599 (2001).

    Article  CAS  Google Scholar 

  15. Brenner, M. K. et al. Lancet 341, 85–86 (1993).

    Article  CAS  Google Scholar 

  16. Pardoll, D. M. Clin. Immunol. 95, 44–62 (2000).

    Article  Google Scholar 

  17. Banchereau, J. et al. Annu. Rev. Immunol. 18, 767–811 (2000).

    Article  CAS  Google Scholar 

  18. Dallal, R. M. & Lotze, M. T. Curr. Opin. Immunol. 12, 583–588 (2000).

    Article  CAS  Google Scholar 

  19. Falcone, M. & Sarvetnick, N. Curr. Opin. Immunol. 11, 670–676 (1999).

    Article  CAS  Google Scholar 

  20. Tarner, I. H. & Fathman, C. G. Curr. Opin. Immunol. 13, 676–682 (2001).

    Article  CAS  Google Scholar 

  21. Roncarolo, M. G. & Levings, M. K. Curr. Opin. Immunol 12, 676–683 (2000).

    Article  CAS  Google Scholar 

  22. Zhang, J., Vandevyver, C., Stinissen, P. & Raus, J. J. Immunol. 155, 5868–5877 (1995).

    CAS  PubMed  Google Scholar 

  23. Vandenbark, A. A. et al. Nature Med. 2, 1109–1115 (1996).

    Article  CAS  Google Scholar 

  24. Yao, Q. et al. J. Gene Med. 2, 210–219 (2000).

    Article  CAS  Google Scholar 

  25. Hoekzema, R., Hannema, A. J., Swaak, T. J., Paardekooper, J. & Hack, C. E. J. Immunol. 135, 265–271 (1985).

    CAS  PubMed  Google Scholar 

  26. Groux, H. et al. Nature 389, 737–742 (1997).

    Article  CAS  Google Scholar 

  27. Tomita, Y., Khan, A. & Sykes, M. J. Immunol. 153, 1087–1098 (1994).

    CAS  PubMed  Google Scholar 

  28. Lee, H. C., Kim, S. J., Kim, K. S., Shin, H. C. & Yoon, J. W. Nature 408, 483–488 (2000).

    Article  CAS  Google Scholar 

  29. Lumelsky, N. et al. Science 292, 1389–1394 (2001).

    Article  CAS  Google Scholar 

  30. Ardouin, L. et al. J. Exp. Med. 187, 105–116 (1998).

    Article  CAS  Google Scholar 

Download references

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Authors and Affiliations

  1. HSR–Telethon Institute for Gene Therapy (TIGET) of Genetic Disease, Cancer Immunotherapy and Gene Therapy Program, Istituto Scientifico H.S. Raffaele, Universitá Vita-Salute San Raffaele, via Olgettina 58, Milan, 20132, Italy

    Claudio Bordignon & Maria Grazia Roncarolo

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  1. Claudio Bordignon
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  2. Maria Grazia Roncarolo
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Bordignon, C., Roncarolo, M. Therapeutic applications for hematopoietic stem cell gene transfer. Nat Immunol 3, 318–321 (2002). https://doi.org/10.1038/ni0402-318

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