Table of Contents Author Guidelines Submit a Manuscript
Anemia
Volume 2010 (2010), Article ID 947816, 13 pages
http://dx.doi.org/10.1155/2010/947816
Research Article

Correction of Fanconi Anemia Group C Hematopoietic Stem Cells Following Intrafemoral Gene Transfer

Unité de Recherche en Pédiatrie, Department of Pediatrics, Université Laval, CHUQ-CHUL, RC-9800, 2705 Boulevard Laurier, Québec, QC, Canada G1V 4G2

Received 1 October 2009; Accepted 17 December 2009

Academic Editor: Maureen E. Hoatlin

Copyright © 2010 Ouassila Habi et al. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

Linked References

  1. N. Young, Bone Marrow Failure Syndromes, W.B. Saunders, 2000.
  2. B. P. Alter, “Cancer in Fanconi anemia, 1927–2001,” Cancer, vol. 97, no. 2, pp. 425–440, 2003. View at Publisher · View at Google Scholar · View at Scopus
  3. M. D. Tischkowitz and S. V. Hodgson, “Fanconi anaemia,” Journal of Medical Genetics, vol. 40, no. 1, pp. 1–10, 2003. View at Google Scholar · View at Scopus
  4. R. D. Kennedy and A. D. D'Andrea, “The Fanconi anemia/BRCA pathway: new faces in the crowd,” Genes and Development, vol. 19, no. 24, pp. 2925–2940, 2005. View at Publisher · View at Google Scholar · View at Scopus
  5. T. Taniguchi and A. D. D'Andrea, “Molecular pathogenesis of Fanconi anemia: recent progress,” Blood, vol. 107, no. 11, pp. 4223–4233, 2006. View at Publisher · View at Google Scholar · View at Scopus
  6. K. Sii-Felice, O. Etienne, F. Hoffschir et al., “Fanconi DNA repair pathway is required for survival and long-term maintenance of neural progenitors,” The EMBO Journal, vol. 27, no. 5, pp. 770–781, 2008. View at Publisher · View at Google Scholar · View at Scopus
  7. Ph. Guardiola, R. Pasquini, I. Dokal et al., “Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: a study on behalf of the European Group for Blood and Marrow Transplantation,” Blood, vol. 95, no. 2, pp. 422–429, 2000. View at Google Scholar · View at Scopus
  8. E. Gluckman, “Bone marrow transplantation in Fanconi's anemia,” Stem Cells, vol. 11, pp. 180–183, 1993. View at Google Scholar
  9. M. Kohli-Kumar, C. Morris, C. DeLaat et al., “Bone marrow transplantation in Fanconi anemia using matched sibling donors,” Blood, vol. 84, no. 6, pp. 2050–2054, 1994. View at Google Scholar · View at Scopus
  10. P. F. Kelly, S. Radtke, C. von Kalle et al., “Stem cell collection and gene transfer in Fanconi anemia,” Molecular Therapy, vol. 15, no. 1, pp. 211–219, 2007. View at Publisher · View at Google Scholar · View at Scopus
  11. J. M. Liu, S. Kim, E. J. Read et al., “Engraftment of hematopoietic progenitor cells transduced with the Fanconi anemia group C gene (FANCC),” Human Gene Therapy, vol. 10, no. 14, pp. 2337–2346, 1999. View at Publisher · View at Google Scholar · View at Scopus
  12. O. Habi, M.-C. Delisle, N. Messier, and M. Carreau, “Lack of self-renewal capacity in FancC/ stem cells after ex vivo expansion,” Stem Cells, vol. 23, no. 8, pp. 1135–1141, 2005. View at Publisher · View at Google Scholar · View at Scopus
  13. L. S. Haneline, X. Li, S. L. M. Ciccone et al., “Retroviral-mediated expression of recombinant Fancc enhances the repopulating ability of FancC-/- hematopoietic stem cells and decreases the risk of clonal evolution,” Blood, vol. 101, no. 4, pp. 1299–1307, 2003. View at Publisher · View at Google Scholar · View at Scopus
  14. X. Zhang, X. Shang, F. Guo et al., “Defective homing is associated with altered Cdc42 activity in cells from patients with Fanconi anemia group A,” Blood, vol. 112, no. 5, pp. 1683–1686, 2008. View at Publisher · View at Google Scholar · View at Scopus
  15. L. U. W. Müller, M. D. Milsom, M.-O. Kim, A. Schambach, T. Schuesler, and D. A. Williams, “Rapid lentiviral transduction preserves the engraftment potential of Fanca/ hematopoietic stem cells,” Molecular Therapy, vol. 16, no. 6, pp. 1154–1160, 2008. View at Publisher · View at Google Scholar · View at Scopus
  16. O. Cohen-Haguenauer, B. Peault, C. Bauche et al., “In vivo repopulation ability of genetically corrected bone marrow cells from Fanconi anemia patients,” Proceedings of the National Academy of Sciences of the United States of America, vol. 103, no. 7, pp. 2340–2345, 2006. View at Publisher · View at Google Scholar · View at Scopus
  17. M. Aube, M. Lafrance, C. Charbonneau, I. Goulet, and M. Carreau, “Hematopoietic stem cells from FancC/ mice have lower growth and differentiation potential in response to growth factors,” Stem Cells, vol. 20, no. 5, pp. 438–447, 2002. View at Google Scholar · View at Scopus
  18. C. S. McCauslin, J. Wine, L. Cheng et al., “In vivo retroviral gene transfer by direct intrafemoral injection results in correction of the SCID phenotype in Jak3 knock-out animals,” Blood, vol. 102, no. 3, pp. 843–848, 2003. View at Publisher · View at Google Scholar · View at Scopus
  19. D. N. Worsham, T. Schuesler, C. von Kalle, and D. Pan, “In vivo gene transfer into adult stem cells in unconditioned mice by in situ delivery of a lentiviral vector,” Molecular Therapy, vol. 14, no. 4, pp. 514–524, 2006. View at Publisher · View at Google Scholar · View at Scopus
  20. L. Naldini, U. Blomer, P. Gallay et al., “In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector,” Science, vol. 272, no. 5259, pp. 263–267, 1996. View at Google Scholar · View at Scopus
  21. F. Mazurier, M. Doedens, O. I. Gan, and J. E. Dick, “Rapid myeloerythroid repopulation after intrafemoral transplantation of NOD-SCID mice reveals a new class of human stem cells,” Nature Medicine, vol. 9, no. 7, pp. 959–963, 2003. View at Publisher · View at Google Scholar · View at Scopus
  22. M. Carreau, O. I. Gan, L. Liu et al., “Bone marrow failure in the Fanconi anemia group C mouse model after DNA damage,” Blood, vol. 91, no. 8, pp. 2737–2744, 1998. View at Google Scholar · View at Scopus
  23. M. Carreau, O. I. Gan, L. Liu, M. Doedens, J. E. Dick, and M. Buchwald, “Hematopoietic compartment of Fanconi anemia group C null mice contains fewer lineage-negative CD34+ primitive hematopoietic cells and shows reduced reconstitution ability,” Experimental Hematology, vol. 27, no. 11, pp. 1667–1674, 1999. View at Publisher · View at Google Scholar · View at Scopus
  24. F. Galimi, M. Noll, Y. Kanazawa et al., “Gene therapy of Fanconi anemia: preclinical efficacy using lentiviral vectors,” Blood, vol. 100, no. 8, pp. 2732–2736, 2002. View at Publisher · View at Google Scholar · View at Scopus
  25. K. A. Gush, K.-L. Fu, M. Grompe, and C. E. Walsh, “Phenotypic correction of Fanconi anemia group C knockout mice,” Blood, vol. 95, no. 2, pp. 700–704, 2000. View at Google Scholar · View at Scopus
  26. P. Rio, J. C. Segovia, H. Hanenberg et al., “In vitro phenotypic correction of hematopoietic progenitors from Fanconi anemia group A knockout mice,” Blood, vol. 100, no. 6, pp. 2032–2039, 2002. View at Google Scholar · View at Scopus
  27. K. Yamada, A. Ramezani, R. G. Hawley et al., “Phenotype correction of Fanconi anemia group A hematopoietic stem cells using lentiviral vector,” Molecular Therapy, vol. 8, no. 4, pp. 600–610, 2003. View at Publisher · View at Google Scholar · View at Scopus
  28. J. M. Croop, R. Cooper, C. Fernandez et al., “Mobilization and collection of peripheral blood CD34+ cells from patients with Fanconi anemia,” Blood, vol. 98, no. 10, pp. 2917–2921, 2001. View at Publisher · View at Google Scholar · View at Scopus
  29. X. Li, M. M. Le Beau, S. Ciccone et al., “Ex vivo culture of FancC-/- stem/progenitor cells predisposes cells to undergo apoptosis, and surviving stem/progenitor cells display cytogenetic abnormalities and an increased risk of malignancy,” Blood, vol. 105, no. 9, pp. 3465–3471, 2005. View at Publisher · View at Google Scholar · View at Scopus
  30. M. Scherr and M. Eder, “Gene transfer into hematopoietic stem cells using lentiviral vectors,” Current Gene Therapy, vol. 2, no. 1, pp. 45–55, 2002. View at Google Scholar · View at Scopus
  31. J. D'Costa, S. G. Mansfield, and L. M. Humeau, “Lentiviral vectors in clinical trials: current status,” Current Opinion in Molecular Therapeutics, vol. 11, no. 5, pp. 554–564, 2009. View at Google Scholar · View at Scopus
  32. N. Cartier, S. Hacein-Bey-Abina, C. C. Bartholomae et al., “Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy,” Science, vol. 326, no. 5954, pp. 818–823, 2009. View at Publisher · View at Google Scholar · View at Scopus