Evaluation of an anti-tumor necrosis factor therapeutic in a mouse model of Niemann-Pick C liver disease

PLoS ONE

Volumn 5, Issue 9, 2010, Pages

  Vincent, Melanie ^a   Sayre, Naomi L ^a   Graham, Mark J ^b   Crooke, Rosanne M ^b   Shealy, David J ^c   Liscum, Laura ^a  

^a TUFTS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b ISIS PHARMACEUTICALS   (United States)

^c CENTOCOR INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE AMINOTRANSFERASE; ANTISENSE OLIGONUCLEOTIDE; CELL PROTEIN; CHOLESTEROL; CNTO 5048; MESSENGER RNA; MONOCLONAL ANTIBODY; PROTEIN NPC1; TUMOR NECROSIS FACTOR INHIBITOR; UNCLASSIFIED DRUG; NPC1 PROTEIN, MOUSE; PROTEIN; TUMOR NECROSIS FACTOR ALPHA;

ALANINE AMINOTRANSFERASE BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; APOPTOSIS; ARTICLE; CELL ACTIVATION; CELL PROLIFERATION; CONTROLLED STUDY; DISEASE COURSE; DRUG EFFICACY; ENZYME BLOOD LEVEL; FEMALE; GENE SILENCING; HEPATOMEGALY; LIPID STORAGE; LIVER PARENCHYMA; MACROPHAGE; MOUSE; NIEMANN PICK DISEASE; NONHUMAN; PROTEIN EXPRESSION; STELLATE CELL; WILD TYPE; ANIMAL; C57BL MOUSE; CYTOLOGY; DISEASE MODEL; DRUG EFFECT; DRUG SCREENING; GENETICS; HUMAN; IMMUNOLOGY; LIPID METABOLISM; LIVER; METABOLISM;

MUS;

ANIMALS; ANTIBODIES, MONOCLONAL; APOPTOSIS; CHOLESTEROL; DISEASE MODELS, ANIMAL; DRUG EVALUATION, PRECLINICAL; FEMALE; HUMANS; LIPID METABOLISM; LIVER; MICE; MICE, INBRED C57BL; NIEMANN-PICK DISEASE, TYPE C; PROTEINS; TUMOR NECROSIS FACTOR-ALPHA;

EID: 77958551161     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0012941     Document Type: Article

References (23)

1. Vanier MT, Millat G (2003) Niemann-Pick disease type C. Clin Genet 64: 269-281.
2. Patterson MC, Vanier MT, Suzuki K, Morris JA, Carstea E, et al. (2001) Niemann-Pick Disease Type C: A Lipid Trafficking Disorder. In: Scriver CR, Beaudet AL, Sly WS, Valle D, eds. The Metabolic and Molecular Bases of Inherited Disease. 8th ed. New York: McGraw-Hill. pp 3611-3633.
3. Garver WS, Francis GA, Jelinek D, Shepherd G, Flynn J, et al. (2007) The National Niemann-Pick C1 disease database: report of clinical features and health problems. Am J Med Genet A 143: 1204-1211.
4. Kelly DA, Portmann B, Mowat AP, Sherlock S, Lake BD (1993) Niemann-Pick disease type C: diagnosis and outcome in children, with particular reference to liver disease. J Pediatr 123: 242-247.
5. Yerushalmi B, Sokol RJ, Narkewicz MR, Smith D, Ashmead JW, et al. (2002) Niemann-pick disease type C in neonatal cholestasis at a North American Center. J Pediatr Gastroenterol Nutr 35: 44-50.
6. Beltroy EP, Richardson JA, Horton JD, Turley SD, Dietschy JM (2005) Cholesterol accumulation and liver cell death in mice with Niemann-Pick type C disease. Hepatology 42: 886-893.
7. Erickson RP, Bhattacharyya A, Hunter RJ, Heidenreich RA, Cherrington NJ (2005) Liver disease with altered bile acid transport in Niemann-Pick C mice on a high-fat, 1% cholesterol diet. Am J Physiol Gastrointest Liver Physiol 289: G300-307.
8. Beltroy EP, Liu B, Dietschy JM, Turley SD (2007) Lysosomal unesterified cholesterol content correlates with liver cell death in murine Niemann-Pick type C disease. J Lipid Res 48: 869-881.
9. Garver WS, Jelinek D, Oyarzo JN, Flynn J, Zuckerman M, et al. (2007) Characterization of liver disease and lipid metabolism in the Niemann-Pick C1 mouse. J Cell Biochem 101: 498-516.
10. Kulinski A, Vance JE (2007) Lipid homeostasis and lipoprotein secretion in Niemann-Pick C1-deficient hepatocytes. J Biol Chem 282: 1627-1637.
11. Rimkunas V, Graham M, Crooke R, Liscum L (2008) In vivo antisense oligonucleotide reduction of NPC1 expression as a novel mouse model for Niemann Pick type C- associated liver disease. Hepatology 47: 1504-1512.
12. Rimkunas VM, Graham MJ, Crooke RM, Liscum L (2009) TNF-{alpha} plays a role in hepatocyte apoptosis in Niemann-Pick type C liver disease. J Lipid Res 50: 327-333.
13. Beutler BA, Milsark IW, Cerami A (1985) Cachectin/tumor necrosis factor: production, distribution, and metabolic fate in vivo. J Immunol 135: 3972-3977.
14. Sayre NL, Rimkunas VM, Graham MJ, Crooke RM, Liscum L. Recovery from liver disease in a Niemann-Pick type C mouse model. J Lipid Res: In press.
15. Karten B, Peake KB, Vance JE (2009) Mechanisms and consequences of impaired lipid trafficking in Niemann-Pick type C1-deficient mammalian cells. Biochim Biophys Acta 1791: 659-670.
16. Wraith JE, Baumgartner MR, Bembi B, Covanis A, Levade T, et al. (2009) Recommendations on the diagnosis and management of Niemann-Pick disease type C. Mol Genet Metab 98: 152-165.
17. Li H, Repa JJ, Valasek MA, Beltroy EP, Turley SD, et al. (2005) Molecular, anatomical, and biochemical events associated with neurodegeneration in mice with Niemann-Pick type C disease. J Neuropathol Exp Neurol 64: 323-333.
18. Wu YP, Mizukami H, Matsuda J, Saito Y, Proia RL, et al. (2005) Apoptosis accompanied by up-regulation of TNF-alpha death pathway genes in the brain of Niemann-Pick type C disease. Mol Genet Metab 84: 9-17.
19. Repa JJ, Li H, Frank-Cannon TC, Valasek MA, Turley SD, et al. (2007) Liver X receptor activation enhances cholesterol loss from the brain, decreases neuroinflammation, and increases survival of the NPC1 mouse. J Neurosci 27: 14470-14480.
20. Shealy DJ, Visvanathan S (2008) Anti-TNF antibodies: lessons from the past, roadmap for the future. Handb Exp Pharmacol. pp 101-129.
21. Crooke RM, Graham MJ, Lemonidis KM, Whipple CP, Koo S, et al. (2005) An apolipoprotein B antisense oligonucleotide lowers LDL cholesterol in hyperlipidemic mice without causing hepatic steatosis. J Lipid Res 46: 872-884.
22. Geary RS, Watanabe TA, Truong L, Freier S, Lesnik EA, et al. (2001) Pharmacokinetic properties of 29-O-(2-methoxyethyl)-modified oligonucleotide analogs in rats. J Pharmacol Exp Ther 296: 890-897.
23. Folch J, Lees M, Sloane Stanley GH (1957) A simple method for the isolation and purification of total lipides from animal tissues. J Biol Chem 226: 497-509.