Potential of Human Induced Pluripotent Stem Cells in Studies of Liver Disease

Hepatology

Volumn 62, Issue 1, 2015, Pages 303-311

  Sampaziotis, Fotios ^a   Segeritz, Charis Patricia ^a   Vallier, Ludovic ^a,b  

^a UNIVERSITY OF CAMBRIDGE   (United Kingdom)

^b WELLCOME TRUST SANGER INSTITUTE   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

PROTEIN;

ALPHA 1 ANTITRYPSIN DEFICIENCY; AMYLOIDOSIS; ARTICLE; BILIARY TRACT DISEASE; CELLS BY BODY ANATOMY; CHOLANGIOCYTE; DISEASE MODEL; DRUG INDUCED DISEASE; ENZYME ACTIVITY; FAMILIAL HYPERCHOLESTEROLEMIA; FAMILIAL TRANSTHYRETIN AMYLOIDOSIS; GLYCOGEN STORAGE DISEASE TYPE 1; GLYCOGEN STORAGE DISEASE TYPE 1A; HEPATITIS C; HEPATITIS C VIRUS; HUMAN; IN VITRO STUDY; LIVER CELL; LIVER CIRRHOSIS; LIVER DISEASE; LIVER INJURY; LIVER TOXICITY; NONHUMAN; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; PROTEIN FOLDING; WILSON DISEASE; INDUCED PLURIPOTENT STEM CELL; PROTEOSTASIS DEFICIENCY; TOXIC HEPATITIS;

DRUG-INDUCED LIVER INJURY; HEPATOCYTES; HUMANS; INDUCED PLURIPOTENT STEM CELLS; LIVER DISEASES; PROTEOSTASIS DEFICIENCIES;

EID: 84933180646     PISSN: 02709139     EISSN: 15273350     Source Type: Journal    
DOI: 10.1002/hep.27651     Document Type: Article

References (61)

1. Horton R. GBD 2010: understanding disease, injury, and risk. Lancet 2012;380:2053-2054.
2. Murray KF, Carithers RL, Jr.; AASLD. AASLD practice guidelines: evaluation of the patient for liver transplantation. Hepatology 2005;41:1407-1432.
3. Hackam DG, Redelmeier DA. Translation of research evidence from animals to humans. JAMA 2006;296:1731-1732.
4. Handa K, Matsubara K, Fukumitsu K, Guzman-Lepe J, Watson A, Soto-Gutierrez A. Assembly of human organs from stem cells to study liver disease. Am J Pathol 2014;184:348-357.
5. Takahashi K, Tanabe K, Ohnuki M, Narita M, Ichisaka T, Tomoda K, et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007;131:861-872.
6. Kmieć Z. Cooperation of liver cells in health and disease. Adv Anat Embryol Cell Biol 2001;161:1-151.
7. Hengstler JG, Brulport M, Schormann W, Bauer A, Hermes M, Nussler AK, et al. Generation of human hepatocytes by stem cell technology: definition of the hepatocyte. Expert Opin Drug Metab Toxicol 2005;1:61-74.
8. Gerbal-Chaloin S, Funakoshi N, Caillaud A, Gondeau C, Champon B, Si-Tayeb K. Human induced pluripotent stem cells in hepatology: beyond the proof of concept. Am J Pathol 2014;184:332-347.
9. Sancho-Bru P, Najimi M, Caruso M, Pauwelyn K, Cantz T, Forbes S, et al. Stem and progenitor cells for liver repopulation: can we standardise the process from bench to bedside? Gut 2009;58:594-603.
10. Schwartz RE, Fleming HE, Khetani SR, Bhatia SN. Pluripotent stem cell-derived hepatocyte-like cells. Biotechnol Adv 2014;32:504-513.
11. Hay DC, Zhao D, Ross A, Mandalam R, Lebkowski J, Cui W. Direct differentiation of human embryonic stem cells to hepatocyte-like cells exhibiting functional activities. Cloning Stem Cells 2007;9:51-62.
12. Si-Tayeb K, Noto FK, Nagaoka M, Li J, Battle MA, Duris C, et al. Highly efficient generation of human hepatocyte-like cells from induced pluripotent stem cells. Hepatology 2010;51:297-305.
13. Touboul T, Hannan NR, Corbineau S, Martinez A, Martinet C, Branchereau S, et al. Generation of functional hepatocytes from human embryonic stem cells under chemically defined conditions that recapitulate liver development. Hepatology 2010;51:1754-1765.
14. Hannan NR, Segeritz CP, Touboul T, Vallier L. Production of hepatocyte-like cells from human pluripotent stem cells. Nat Protoc 2013;8:430-437.
15. Du Y, Wang J, Jia J, Song N, Xiang C, Xu J, et al. Human hepatocytes with drug metabolic function induced from fibroblasts by lineage reprogramming. Cell Stem Cell 2014;14:394-403.
16. Ogawa S, Surapisitchat J, Virtanen C, Ogawa M, Niapour M, Sugamori KS, et al. Three-dimensional culture and cAMP signaling promote the maturation of human pluripotent stem cell-derived hepatocytes. Development 2013;140:3285-3296.
17. Si-Tayeb K, Lemaigre FP, Duncan SA. Organogenesis and development of the liver. Dev Cell 2010;18:175-189.
18. Takayama K, Inamura M, Kawabata K, Katayama K, Higuchi M, Tashiro K, et al. Efficient generation of functional hepatocytes from human embryonic stem cells and induced pluripotent stem cells by HNF4α transduction. Mol Ther 2012;20:127-137.
19. Shan J, Schwartz RE, Ross NT, Logan DJ, Thomas D, Duncan SA, et al. Identification of small molecules for human hepatocyte expansion and iPS differentiation. Nat Chem Biol 2013;9:514-520.
20. Shan J, Schwartz RE, Ross NT, Logan DJ, Thomas D, Duncan SA, et al. Identification of small molecules for human hepatocyte expansion and iPS differentiation. Nat Chem Biol 2013;9:514-520.
21. Huang P, Zhang L, Gao Y, He Z, Yao D, Wu Z, et al. Direct reprogramming of human fibroblasts to functional and expandable hepatocytes. Cell Stem Cell 2014;14:370-384.
22. Sekiya S, Suzuki A. Direct conversion of mouse fibroblasts to hepatocyte-like cells by defined factors. Nature 2011;475:390-393.
23. Zhu S, Rezvani M, Harbell J, Mattis AN, Wolfe AR, Benet LZ, et al. Mouse liver repopulation with hepatocytes generated from human fibroblasts. Nature 2014;508:93-97.
24. Gieseck RL, Hannan NR, Bort R, Hanley NA, Drake RA, Cameron GW, et al. Maturation of induced pluripotent stem cell derived hepatocytes by 3D-culture. PLoS One 2014;9:e86372.
25. Bhatia SN, Balis UJ, Yarmush ML, Toner M. Effect of cell-cell interactions in preservation of cellular phenotype: cocultivation of hepatocytes and nonparenchymal cells. FASEB J 1999;13:1883-1900.
26. Stevens KR, Ungrin MD, Schwartz RE, Ng S, Carvalho B, Christine KS, et al. InVERT molding for scalable control of tissue microarchitecture. Nat Commun 2013;4:1847.
27. Takebe T, Sekine K, Enomura M, Koike H, Kimura M, Ogaeri T, et al. Vascularized and functional human liver from an iPSC-derived organ bud transplant. Nature 2013;499:481-484.
28. Zhao D, Chen S, Cai J, Guo Y, Song Z, Che J, et al. Derivation and characterization of hepatic progenitor cells from human embryonic stem cells. PLoS One 2009;4:e6468.
29. Tanimizu N, Miyajima A, Mostov KE. Liver progenitor cells develop cholangiocyte-type epithelial polarity in three-dimensional culture. Mol Biol Cell 2007;18:1472-1479.
30. Yu B, He ZY, You P, Han QW, Xiang D, Chen F, et al. Reprogramming fibroblasts into bipotential hepatic stem cells by defined factors. Cell Stem Cell 2013;13:328-340.
31. Nakamura N, Saeki K, Mitsumoto M, Matsuyama S, Nishio M, Saeki K, et al. Feeder-free and serum-free production of hepatocytes, cholangiocytes, and their proliferating progenitors from human pluripotent stem cells: application to liver-specific functional and cytotoxic assays. Cell Reprogram 2012;14:171-185.
32. Dianat N, Dubois-Pot-Schneider H, Steichen C, Desterke C, Leclerc P, Raveux A, et al. Generation of functional cholangiocyte-like cells from human pluripotent stem cells and HepaRG cells. Hepatology 2014;60:700-714.
33. Rashid ST, Corbineau S, Hannan N, Marciniak SJ, Miranda E, Alexander G, et al. Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells. J Clin Invest 2010;120:3127-3136.
34. Fairbanks KD, Tavill AS. Liver disease in alpha 1-antitrypsin deficiency: a review. Am J Gastroenterol 2008;103:2136-2141.
35. Yusa K, Rashid ST, Strick-Marchand H, Varela I, Liu PQ, Paschon DE, et al. Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells. Nature 2011;478:391-394.
36. Ando Y, Nakamura M, Araki S. Transthyretin-related familial amyloidotic polyneuropathy. Arch Neurol 2005;62:1057-1062.
37. Leung A, Nah SK, Reid W, Ebata A, Koch CM, Monti S, et al. Induced pluripotent stem cell modeling of multisystemic, hereditary transthyretin amyloidosis. Stem Cell Reports 2013;1:451-463.
38. Rake JP, Visser G, Labrune P, Leonard JV, Ullrich K, Smit GP. Glycogen storage disease type I: diagnosis, management, clinical course and outcome. Results of the European Study on Glycogen Storage Disease Type I. Eur J Pediatr 2002;161:S20-S34.
39. Roberts EA, Schilsky ML; AASLD. Diagnosis and treatment of Wilson disease: an update. Hepatology 2008;47:2089-2111.
40. Zhang S, Chen S, Li W, Guo X, Zhao P, Xu J, et al. Rescue of ATP7B function in hepatocyte-like cells from Wilson's disease induced pluripotent stem cells using gene therapy or the chaperone drug curcumin. Hum Mol Genet 2011;20:3176-3187.
41. Yi F, Qu J, Li M, Suzuki K, Kim NY, Liu GH, et al. Establishment of hepatic and neural differentiation platforms of Wilson's disease specific induced pluripotent stem cells. Protein Cell 2012;3:855-863.
42. Rader DJ, Cohen J, Hobbs HH. Monogenic hypercholesterolemia: new insights in pathogenesis and treatment. J Clin Invest 2003;111:1795-1803.
43. Cayo MA, Cai J, Delaforest A, Noto FK, Nagaoka M, Clark BS, et al. JD induced pluripotent stem cell-derived hepatocytes faithfully recapitulate the pathophysiology of familial hypercholesterolemia. Hepatology 2012;56:2163-2171.
44. Voora D, Shah SH, Reed CR, Zhai J, Crosslin DR, Messer C, et al. Pharmacogenetic predictors of statinmediated low-density lipoprotein cholesterol reduction and dose response. Circ Cardiovasc Genet 2008;1:100-106.
45. European Association for Study of Liver. EASL Clinical Practice Guidelines: management of hepatitis C virus infection. J Hepatol 2014;60:392-420.
46. Lohmann V, Korner F, Koch J, Herian U, Theilmann L, Bartenschlager R. Replication of subgenomic hepatitis C virus RNAs in a hepatoma cell line. Science 1999;285:110-113.
47. Wu X, Robotham JM, Lee E, Dalton S, Kneteman NM, Gilbert DM, et al. Productive hepatitis C virus infection of stem cell-derived hepatocytes reveals a critical transition to viral permissiveness during differentiation. PLoS Pathog 2012;8:e1002617.
48. Roelandt P, Obeid S, Paeshuyse J, Vanhove J, Van Lommel A, Nahmias Y, et al. Human pluripotent stem cell-derived hepatocytes support complete replication of hepatitis C virus. J Hepatol 2012;57:246-251.
49. Yoshida T, Takayama K, Kondoh M, Sakurai F, Tani H, Sakamoto N, et al. Use of human hepatocyte-like cells derived from induced pluripotent stem cells as a model for hepatocytes in hepatitis C virus infection. Biochem Biophys Res Commun 2011;416:119-124.
50. Schwartz RE, Trehan K, Andrus L, Sheahan TP, Ploss A, Duncan SA, et al. Modeling hepatitis C virus infection using human induced pluripotent stem cells. Proc Natl Acad Sci U S A 2012;109:2544-2548.
51. Zhou XL, Sullivan GJ, Sun P, Park IH. Humanized murine model for HBV and HCV using human induced pluripotent stem cells. Arch Pharm Res 2012;35:261-269.
52. Klotz C, Aebischer T, Seeber F. Stem cell-derived cell cultures and organoids for protozoan parasite propagation and studying host-parasite interaction. Int J Med Microbiol 2012;302:203-209.
53. Guyot C, Lepreux S, Combe C, Doudnikoff E, Bioulac-Sage P, Balabaud C, et al. Hepatic fibrosis and cirrhosis: the (myo)fibroblastic cell subpopulations involved. Int J Biochem Cell Biol 2006;38:135-151.
54. Van de Bovenkamp M, Groothuis GM, Meijer DK, Olinga P. Liver fibrosis in vitro: cell culture models and precision-cut liver slices. Toxicol In Vitro 2007;21:545-557.
55. Pollheimer MJ, Trauner M, Fickert P. Will we ever model PSC?-"it's hard to be a PSC model!". Clin Res Hepatol Gastroenterol 2011;35:792-804.
56. Lazaridis KN, Strazzabosco M, Larusso NF. The cholangiopathies: disorders of biliary epithelia. Gastroenterology 2004;127:1565-1577.
57. Mells GF, Floyd JA, Morley KI, Cordell HJ, Franklin CS, Shin SY, et al. Genome-wide association study identifies 12 new susceptibility loci for primary biliary cirrhosis. Nat Genet 2011;43:329-332.
58. Medine CN, Lucendo-Villarin B, Storck C, Wang F, Szkolnicka D, Khan F, et al. Developing high-fidelity hepatotoxicity models from pluripotent stem cells. Stem Cells Transl Med 2013;2:505-509.
59. Chalasani N, Bjornsson E. Risk factors for idiosyncratic drug-induced liver injury. Gastroenterology 2010;138:2246-2259.
60. Soares FA, Chandra A, Thomas RJ, Pedersen RA, Vallier L, Williams DJ. Investigating the feasibility of scale up and automation of human induced pluripotent stem cells cultured in aggregates in feeder free conditions. J Biotechnol 2014;173:53-58.
61. Choi SM, Kim Y, Shim JS, Park JT, Wang RH, Leach SD, et al. Efficient drug screening and gene correction for treating liver disease using patient-specific stem cells. Hepatology 2013;57:2458-2468.