Lentiviral gene transfer ameliorates disease progression in Long-Evans cinnamon rats: An animal model for Wilson disease

Scandinavian Journal of Gastroenterology

Volumn 41, Issue 8, 2006, Pages 974-982

  Merle, Uta ^a   Enckea, Jens ^a   Tuma, Sabine ^a   Volkmann, Martin ^a   Naldini, Luigi ^b   Stremmel, Wolfgang ^a  

^a UNIVERSITY HOSPITAL HEIDELBERG   (Germany)

^b SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

Author keywords

Copper; LEC rat; Lentiviral gene therapy; Wilson disease

Indexed keywords

CERULOPLASMIN; CERULOPLASMIN OXIDASE; COPPER; LENTIVIRUS VECTOR; UNCLASSIFIED DRUG; WILSON DISEASE PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ATP7B GENE; CONTROLLED STUDY; DISEASE COURSE; ENZYME ACTIVITY; ENZYME BLOOD LEVEL; GENE EXPRESSION; GENE MUTATION; IMMUNOFLUORESCENCE; LIVER CELL; LIVER CIRRHOSIS; LIVER FIBROSIS; LIVER HISTOLOGY; MALE; NONHUMAN; PILOT STUDY; PRIORITY JOURNAL; RAT; RAT STRAIN; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SYSTEMIC THERAPY; TISSUE SECTION; TRANSGENE; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM; WILSON DISEASE;

ADENOSINE TRIPHOSPHATASES; ANIMALS; CATION TRANSPORT PROTEINS; CERULOPLASMIN; COPPER; DISEASE MODELS, ANIMAL; DISEASE PROGRESSION; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEPATOCYTES; HEPATOLENTICULAR DEGENERATION; LENTIVIRUS; LIVER; RATS; RATS, INBRED LEC; RATS, LONG-EVANS; TRANSDUCTION, GENETIC;

EID: 33745712832     PISSN: 00365521     EISSN: 15027708     Source Type: Journal    
DOI: 10.1080/00365520600554790     Document Type: Article

References (23)

1. Bull PC, Thomas GR, Rommens JM, Forbes JR, Cox DW. The Wilson disease gene is a putative copper transporting P-type ATPase similar to the Menkes gene. Nat Genet 1993;5:327-37.
2. Ha-Hao D, Merle U, Hofmann C, Wesch H, Doll J, Auburger G, et al. Chances and shortcomings of adenovirus-mediated ATP7B gene transfer in Wilson disease: proof of principle demonstrated in a pilot study with LEC rats. Z Gastroenterol 2002;40:209-16.
3. Terada K, Nakako T, Yang XL, Iida M, Aiba N, Minamiya Y, et al. Restoration of holoceruloplasmin synthesis in LEC rat after infusion of recombinant adenovirus bearing WND cDNA. J Biol Chem 1998;273:1815-20.
4. Naldini L, Blomer U, Gallay P, Ory D, Mulligan R, Gage F H, et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996;272:263-7.
5. Follenzi A, Sabatino G, Lombardo A, Boccaccio C, Naldini L. Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors. Hum Gene Ther 2002;13:243-60.
6. Irani AN, Malhi H, Slehria S, Gorla G R, Volenberg I, Schilsky M L, et al. Correction of liver disease following transplantation of normal rat hepatocytes into Long-Evans Cinnamon rats modeling Wilson's disease. Mol Ther 2001;3:302-9.
7. Malhi H, Irani AN, Volenberg I, Schilsky ML, Gupta S. Early cell transplantation in LEC rats modeling Wilson's disease eliminates hepatic copper with reversal of liver disease. Gastroenterology 2002;122:438-47.
8. Yoshida Y, Tokusashi Y, Lee GH, Ogawa K. Intrahepatic transplantation of normal hepatocytes prevents Wilson's disease in Long-Evans Cinnamon rats. Gastroenterology 1996;111:1654-60.
9. Schilsky ML, Stockert RJ, Sternlieb I. Pleiotropic effect of LEC mutation: a rodent model of Wilson's disease. Am J Physiol 1994;266:G907-13.
10. Wu J, Forbes JR, Chen HS, Cox DW. The LEC rat has a deletion in the copper transporting ATPase gene homologous to the Wilson disease gene. Nat Genet 1994;7:541-5.
11. Schulze-Bergkamen H, Untergasser A, Dax A, Vogel H, Buchler P, Klar E, et al. Primary human hepatocytes: a valuable tool for investigation of apoptosis and hepatitis B virus infection. J Hepatol 2003;38:736-44.
12. Berry MN, Friend DS. High-yield preparation of isolated rat liver parenchymal cells: a biochemical and fine structural study. J Cell Biol 1969;43:506-20.
13. Follenzi A, Ailles LE, Bakovic S, Geuna M, Naldini L. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat Genet 2000;25:217-22.
14. Reiser J. Production and concentration of pseudotyped HIV-1-based gene transfer vectors. Gene Ther 2000;7:910-3.
15. Nguyen TH, Oberholzer J, Birraux J, Majno P, Morel P, Trono D. Highly efficient lentiviral vector-mediated transduction of nondividing, fully reimplantable primary hepatocytes. Mol Ther 2002;6:199-209.
16. Higgins G, Anderson R. Experimental pathology of the liver. Restoration of the liver of the white rat following partial surgical removal. Arch Pathol 1931;12:186-202.
17. Schosinsky KH, Lehmann HP, Beeler MF. Measurement of ceruloplasmin from its oxidase activity in serum by use of o-dianisidine dihydrochloride. Clin Chem 1974;20:1556-63.
18. Li Q, Kay MA, Finegold M, Stratford-Perricaudet LD, Woo SL. Assessment of recombinant adenoviral vectors for hepatic gene therapy. Hum Gene Ther 1993;4:403-9.
19. Ghosh A, Allamarvdasht M, Pan CJ, Sun M S, Mansfield BC, Byrne B J, et al. Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer. Gene Ther 2005;13:321-9.
20. Harding CO, Gillingham MB, Hamman K, Clark H, Goebel-Daghighi E, Bird A, et al. Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria. Gene Ther;Dec 1; [Epub ahead of print].
21. Nathwani AC, Davidoff AM, Hanawa H, Hu Y, Hoffer F A, Nilkanorov A, et al. Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques. Blood 2002;100:1662-9.
22. McDonald JA, Snitch P, Painter D, Hensley W, Gallagher ND, McCaughan GW. Striking variability of hepatic copper levels in fulminant hepatic failure. J Gastroenterol Hepatol 1992;7:396-8.
23. Yamada T, Agui T, Suzuki Y, Sato M, Matsumoto K. Inhibition of the copper incorporation into ceruloplasmin leads to the deficiency in serum ceruloplasmin activity in Long-Evans cinnamon mutant rat. J Biol Chem 1993;268:8965-71.