Gastroenterology
Volume 130, Issue 4 , Pages 1317-1323 , April 2006

Sustained Engraftment and Tissue Enzyme Activity After Liver Cell Transplantation for Argininosuccinate Lyase Deficiency

  • Xavier Stéphenne

      Affiliations

    • Laboratoire d’hépatologie Pédiatrique et Transplantation Cellulaire, Département GYPE, Service de Pédiatrie, Université Catholique de Louvain & Cliniques St Luc, Brussels, Belgium
  • ,
  • Mustapha Najimi

      Affiliations

    • Laboratoire d’hépatologie Pédiatrique et Transplantation Cellulaire, Département GYPE, Service de Pédiatrie, Université Catholique de Louvain & Cliniques St Luc, Brussels, Belgium
  • ,
  • Catherine Sibille

      Affiliations

    • Centre de Cytogénétique, Université Catholique de Louvain & Cliniques St Luc, Brussels, Belgium
  • ,
  • Marie–Cécile Nassogne

      Affiliations

    • Service de Neurologie Pédiatrique, Université Catholique de Louvain & Cliniques St Luc, Brussels, Belgium
  • ,
  • Françoise Smets

      Affiliations

    • Laboratoire d’hépatologie Pédiatrique et Transplantation Cellulaire, Département GYPE, Service de Pédiatrie, Université Catholique de Louvain & Cliniques St Luc, Brussels, Belgium
  • ,
  • Etienne M. Sokal

      Affiliations

    • Laboratoire d’hépatologie Pédiatrique et Transplantation Cellulaire, Département GYPE, Service de Pédiatrie, Université Catholique de Louvain & Cliniques St Luc, Brussels, Belgium
    • Corresponding Author InformationAddress requests for reprints to: Etienne M. Sokal, MD, PhD, Paediatric Hepatology and Liver Transplantation, Université Catholique de Louvain, Cliniques St Luc, 10 av. Hippocrate, B-1200 Brussels, Belgium. fax: (32) 2-764-8909.

Received 7 October 2005 ,Accepted 21 December 2005.

References 

  1. Najimi M , Sokal E . Update on liver cell transplantation . J Pediatr Gastroenterol Nutr . 2004;39:311–319
  2. Fox IJ , Chowdhury JR , Kaufman SS , et al.   Treatment of the Crigler-Najjar syndrome type I with hepatocyte transplantation . N Engl J Med . 1998;338:1422–1426
  3. Sokal EM , Smets F , Bourgois A , et al.   Hepatocyte transplantation in a 4-year-old girl with peroxisomal biogenesis disease (technique, safety, and metabolic follow-up) . Transplantation . 2003;76:735–738
  4. Muraca M , Gerunda G , Neri D , et al.   Hepatocyte transplantation as a treatment for glycogen storage disease type 1a . Lancet . 2002;359:317–318
  5. Whitington PF, Alonso EM, Boyle JT, Molleston JP, Rosenthal P, Emond JC, et al. Liver transplantation for the treatment of urea cycle disorders . J Inherit Metab Dis . 1998;21(Suppl 1):112–118
  6. Leonard JV , McKiernan PJ . The role of liver transplantation in urea cycle disorders . Mol Genet Metab . 2004;81(Suppl 1):S74–S78
  7. Horslen SP, McCowan TC, Goertzen TC, Warkentin PI, Cai HB, Strom SC, et al. Isolated hepatocyte transplantation in an infant with a severe urea cycle disorder . Pediatrics . 2003;111:1262–1267
  8. Stéhenne X , Najimi M , Smets F , Reding R , de Ville de Goyet J , Sokal EM . Cryopreserved liver cell transplantation controls ornithine transcarbamylase deficient patient while waiting liver transplantation . Am J Transplant . 2005;5:2058–2061
  9. Darwish AA , Sokal E , Stephenne X , Najimi M , de Ville de Goyet J , Reding R . Permanent access to the portal system for cellular transplantation using an implantable port device . Liver Transpl . 2004;10:1213–1215
  10. Najimi M , Maloteaux JM , Hermans E . Pertussis toxin-sensitive modulation of glutamate transport by endothelin-1 type A receptors in glioma cells . Biochim Biophys Acta . 2005;1668:195–202
  11. Ponder KP , Gupta S , Leland F , et al.   Mouse hepatocytes migrate to liver parenchyma and function indefinitely after intrasplenic transplantation . Proc Natl Acad Sci U S A . 1991;88:1217–1221
  12. Wang LJ, Chen YM, George D, Smets F, Sokal EM, Bremer EG, et al. Engraftment assessment in human and mouse liver tissue after sex-mismatched liver cell transplantation by real-time quantitative PCR for Y chromosome sequences . Liver Transpl . 2002;8:822–828
  13. Mas VR , Maluf DG , Thompson M , Ferreira-Gonzalez A , Fisher RA . Engraftment measurement in human liver tissue after liver cell transplantation by short tandem repeats analysis . Cell Transplant . 2004;13:231–236
  14. Kano J , Noguchi M , Kodama M , Tokiwa T . The in vitro differentiating capacity of nonparenchymal epithelial cells derived from adult porcine livers . Am J Pathol . 2000;156:2033–2043
  15. Sudo R , Ikeda S , Sugimoto S , et al.   Bile canalicular formation in hepatic organoid reconstructed by rat small hepatocytes and nonparenchymal cells . J Cell Physiol . 2004;199:252–261
  16. Harada K , Mitaka T , Miyamoto S , et al.   Rapid formation of hepatic organoid in collagen sponge by rat small hepatocytes and hepatic nonparenchymal cells . J Hepatol . 2003;39:716–723
  17. Rosenthal N . Prometheus’s vulture and the stem-cell promise . N Engl J Med . 2003;349:267–274
  18. Wang X , Foster M , Al-Dhalimy M , Lagasse E , Finegold M , Grompe M . The origin and liver repopulating capacity of murine oval cells . Proc Natl Acad Sci U S A . 2003;100(Suppl 1):11881–11888
  19. He ZP , Tan WQ , Tang YF , Zhang HJ , Feng MF . Activation, isolation, identification and in vitro proliferation of oval cells from adult rat livers . Cell Prolif . 2004;37:177–187
  20. Gupta S , Aragona E , Vemuru RP , Bhargava KK , Burk RD . Permanent engraftment and function of hepatocytes delivered to the liver (implications for gene therapy and liver repopulation) . Hepatology . 1991;14:144–149
  21. Rhim JA , Sandgren EP , Degen JL , Palmiter RD , Brinster RL . Replacement of diseased mouse liver by hepatic cell transplantation . Science . 1994;263:1149–1152
  22. Overturf K , Al-Dhalimy M , Ou CN , Finegold M , Grompe M . Serial transplantation revals the stem-cell like regenerative potential of adult mouse hepatocytes . Am J Pathol . 1997;151:1273–1280
  23. Epstein CJ . Cell size, nuclear content and the development of polyploidy . Proc Natl Acad Sci U S A . 1967;57:327
  24. Ravid K , Lu J , Zimmet J , Jones M . Roads to polyploidy (the megakaryocyte example) . J Cell Physiol . 2002;190:7–20
  25. Guidotti JE , Bregerie O , Robert A , Debey P , Brechot C , Desdouets C . Liver cell polyploidization (a pivotal role for binuclear hepatocytes) . J Biol Chem . 2003;278:19095–19101
  26. Weglarz TC , Degen JL , Sandgren EP . Hepatocyte transplantation into diseased mouse liver. Kinetics of parenchymal repopulation and identification of the proliferative capacity of tetraploid and octaploid hepatocytes . Am J Pathol . 2000;157:1963–1974
  27. Overturf K , Al-Dhalimy M , Finegold M , Grompe M . The repopulation potential of hepatocyte populations differing in size and prior mitotic expansion . Am J Pathol . 1999;155:2135–2143
  28. Gorla GR , Malhi H , Gupta S . Polyploid associated with oxydative injury attenuates proliferative potential of cells . J Cell Sci . 2001;114:2943–2951
  29. Sigal HS , Rajvanshi P , Gorla GR , et al.   Partial hepatectomy-induced polyploidy attenuates hepatocyte replication and activates cell aging events . Am J Physiol . 1999;276:1260–1272
  30. Widhalm K, Koch S, Scheibenreiter S, Knoll E, Colombo JP, Bachmann C, et al. Long-term follow-up of 12 patients with the late-onset variant of argininosuccinic acid lyase deficiency (no impairment of intellectual and psychomotor development during therapy) . Pediatrics . 1992;89:1182–1184
  31. Walker DC , McCloskey DA , Simard LR , McInnes RR . Molecular analysis of human argininosuccinate lyase (mutant characterization and alternative splicing of the coding region) . Proc Natl Acad Sci U S A . 1990;87:9625–9629
  32. Saudubray JM , Touati G , Delonlay P , et al.   Liver transplantation in urea cycle disorders . Eur J Pediatr . 1999;158(Suppl 2):S55–S59
  33. Mori T, Nagai K, Mori M, Nagao M, Imamura M, Iijima M, et al. Progressive liver fibrosis in late-onset argininosuccinate lyase deficiency . Pediatr Dev Pathol . 2002;5:597–601
  34. Mitchell C , Mallet VO , Guidotti JE , Goulenok C , Kahn A , Gilgenkrantz H . Liver repopulation by Bcl-x(L) transgenic hepatocytes . Am J Pathol . 2002;160:31–35
  35. Ng IO, Chan KL, Shek WH, Lee JM, Fong DY, Lo CM, et al. High frequency of chimerism in transplanted livers . Hepatology . 2003;38:989–998

 The Liver Cell Transplantation Program is supported by the DGTRE, région wallonne (Grant WALEO/HEPATERA). The Cell Cryopreservation Program is supported by a Grant-Télévie-Fonds National de la Recherche Scientifique.

PII: S0016-5085(06)00009-6

doi: 10.1053/j.gastro.2006.01.008

Gastroenterology
Volume 130, Issue 4 , Pages 1317-1323 , April 2006