Display options
Cite
Sokal EM, Stéphenne X, Ottolenghi C, et al. Liver engraftment and repopulation by in vitro expanded adult derived human liver stem cells in a child with ornithine carbamoyltransferase deficiency. JIMD Rep. 2013;13:65-72doi: 10.1007/8904_2013_257.
Sokal, E. M., Stéphenne, X., Ottolenghi, C., Jazouli, N., Clapuyt, P., Lacaille, F., Najimi, M., de Lonlay, P., & Smets, F. (2014). Liver engraftment and repopulation by in vitro expanded adult derived human liver stem cells in a child with ornithine carbamoyltransferase deficiency. JIMD reports, 1365-72. https://doi.org/10.1007/8904_2013_257
Sokal, Etienne M, et al. "Liver engraftment and repopulation by in vitro expanded adult derived human liver stem cells in a child with ornithine carbamoyltransferase deficiency." JIMD reports vol. 13 (2014): 65-72. doi: https://doi.org/10.1007/8904_2013_257
Sokal EM, Stéphenne X, Ottolenghi C, Jazouli N, Clapuyt P, Lacaille F, Najimi M, de Lonlay P, Smets F. Liver engraftment and repopulation by in vitro expanded adult derived human liver stem cells in a child with ornithine carbamoyltransferase deficiency. JIMD Rep. 2014;13:65-72. doi: 10.1007/8904_2013_257. Epub 2013 Oct 20. PMID: 24142276; PMCID: PMC4110332.
Copy Download .nbib Format: NLM
Share it on

JIMD Rep. 2014;13:65-72. doi: 10.1007/8904_2013_257. Epub 2013 Oct 20.

Liver engraftment and repopulation by in vitro expanded adult derived human liver stem cells in a child with ornithine carbamoyltransferase deficiency.

JIMD reports

Etienne M Sokal, Xavier Stéphenne, Chris Ottolenghi, Nawal Jazouli, Philippe Clapuyt, Florence Lacaille, Mustapha Najimi, Pascale de Lonlay, Françoise Smets

Affiliations

  1. Université Catholique de Louvain & Cliniques Universitaires Saint Luc, 10 av Hippocrate, B 1200, Bruxelles, Belgium, [email protected].

PMID: 24142276 PMCID: PMC4110332 DOI: 10.1007/8904_2013_257

Abstract

A 3-year-old girl suffering from ornithine carbamoyltransferase (OTC) deficiency was poorly equilibrated under conventional diet and scavenger treatment. Following unsuccessful cryopreserved hepatocyte transplantation, she received two infusions of Adult Derived Human Liver Stem/Progenitor Cells (ADHLSCs) expanded in vitro under GMP settings, the quantity being equivalent to 0.75% of her calculated liver mass. Using FISH immunostaining for the Y chromosome, the initial biopsy did not detect any male nuclei in the recipient liver. Two liver biopsies taken 100 days after ADHLSC transplantation showed 3% and 5% of male donor cells in the recipient liver, thus suggesting repopulation by donor cells. Although limited follow-up did not allow us to draw conclusions on long-term improvement, these results provide a promising proof of concept that this therapy is feasible in an OTC patient.

References

  1. Cell Transplant. 2012;21(10):2241-55 - PubMed
  2. PLoS One. 2012;7(8):e42819 - PubMed
  3. Cell Transplant. 2007;16(7):717-28 - PubMed
  4. J Inherit Metab Dis. 2005;28(3):407-14 - PubMed
  5. N Engl J Med. 1998 May 14;338(20):1422-6 - PubMed
  6. Mol Genet Metab. 2010;100 Suppl 1:S77-83 - PubMed
  7. Gut. 2009 Apr;58(4):594-603 - PubMed
  8. Am J Transplant. 2005 Aug;5(8):2058-61 - PubMed
  9. Lancet. 2008 May 10;371(9624):1579-86 - PubMed
  10. J Hepatol. 2006 Jul;45(1):144-59 - PubMed
  11. Pediatr Transplant. 2008 Feb;12(1):6-13 - PubMed
  12. Transplantation. 2003 Aug 27;76(4):735-8 - PubMed
  13. N Engl J Med. 2007 May 31;356(22):2282-92 - PubMed
  14. Cell Transplant. 2013;22(8):1369-80 - PubMed
  15. Cell Transplant. 2011;20(2):287-302 - PubMed
  16. J Intern Med. 2012 Sep;272(3):201-23 - PubMed
  17. J Inherit Metab Dis. 2012 Sep;35(5):777-85 - PubMed
  18. Mol Genet Metab. 2005 Apr;84(4):363-6 - PubMed
  19. J Pediatr. 2001 Jan;138(1 Suppl):S40-4;discussion S44-5 - PubMed
  20. Mol Genet Metab. 2010;100 Suppl 1:S37-41 - PubMed
  21. Cell Transplant. 2014;23(9):1127-42 - PubMed
  22. Gastroenterology. 2006 Apr;130(4):1317-23 - PubMed
  23. J Inherit Metab Dis. 1998;21 Suppl 1:151-9 - PubMed

Publication Types