Reference Animal model Type and number of
transplanted cells
Percentage of repopulation Improved liver functions Mouse survival after
transplantation
Teratani et al. [118] 129X1/SvJ and BALB/c
nude mice
(DMN, cirrhosis)
mESC-Hep
5 e6/mouse (IV)
ND Suppression of onset of fibrosis/cirrhosis
plasma fibrinogen
Alb
~40% after 12 weeks
Heo et al. [116] MUP-uPA/SCID + CCL4 mESC-Hep
1 e6/mouse (IS)
isolated Alb+ cells
1.94% +/- 5.81 after 82 days ND ND
Gouon-Evans et al. [50] Dpp4-/-, Rag2-/- (CCl4
and retrorsine) and Fah-/-
mESC-Hep
0.25 - 1.5 e6/mouse (IS)
Scattered endothelial and
hepatocytic clusters
ND ND
Agarwal et al. [121] NOD/SCID
(CCl4 and retrorsine,
acute liver failure)
hESC-Hep
1 e6/mouse
(PV)
sporadic hepatocytic clusters ND ND
Hay et al. [26] NOD/SCID hESC-Hep
1 e6/mouse
(IS)
Clusters of CK18/CK19 and
Alb cells after 3 days in the
spleen
Alb  
Cai et al. [25] SCID (CCl4, acute liver
failure)
hESC-Hep
1 e6/mouse
(IS)
scattered AAT human cells in
livers
ND ND
Duan et al. [122] NOD/SCID hESC-Hep
0.5 e6/site (IH)
ND Alb ND
Haridass et al. [114] Alb-uPA, Rag2-/-, IL2Rγ-/- ES-Hep
0.5-1 e6/mouse (IS)
Few cells scattered and
teratoma formation
ND ND
Basma et al. [115] NOD/SCID (retrorsine and
50% hepatectomy)
Alb-uPA SCID
hES-Hep
0.1-0.2 e6/mouse (IS)
Isolated ASGPR+ cells
Few clusters of albumin+ cells
after 28 days
Alb, AAT ND
Li et al. [78] Fah-/- mES-Hep
1 e6/mouse (IS)
Isolated
ckit-Epcam+
24%+/-15% ND at least 10 weeks
Touboul et al. [27] Alb-uPA, Rag2-/-, IL2Rγ-/- hES-Hep
0.5 e6/mouse (IH)
Small and large clusters
throughout the liver
after 8 weeks
AAT
Alb
ND
Si-Tayeb et al. [17]   hiPSC-Hep
0.3 e6/mouse (IH)
ND ND ND
Huang et al. [102] Fah-/-, Rag2-/- m iHep from fibroblasts
0.833 e6/mouse (IS)
5%-80% ALT, AST, Tyrosine, Phenylalanine,
Bilirubin
5/12 mice survived
after 8 weeks
Sekiya and Suzuki [103] Fah-/- m iHep from fibroblasts
(IS)
Large clusters of Bilirubin, ALT, ALP, Alb 40% at 10 weeks
Li et al. [119] BALB/c nude
(TAA, liver fibrosis)
m iPSC-Hep
~0.25 e6/mouse (IV)
Scattered cells in the liver
after 24h00
ALT, AST, Bilirubin, Ammonia,
decreased ROS levels and necrosis
~80% after 14 days
Chang et al. [117] BALBc nude
(CCl4, acute liver failure)
m iPSC-Hep
0.2-5 e6/mouse (IP)
Necrosis : 70% control, 20%
transplant
ALT, AST, Bilirubin 90% at 14 days
Woo et al. [39] BALBc nude
(CCl4, acute liver failure)
hES-Hep / hiPSC-Hep
2 e6/mouse (IS)
Isolated ICG+ cells
~20% at day 3 versus ~10%
after 35 days
Alb ND
Chen et al. [38] NOD/SCID
(CCl4, acute liver failure)
hiPSC -Hep
(IS)
ND rescue of hepatic necrosis
glutamyl oxaloacetic aminotransferase
glutamyl pyruvic aminotransferase
Bilirubin
lactate dehydrogenase
HepPar1
Alb
5/7 (71%) after 21 days
Liu et al. [41] NSG (DMN, cirrhosis) hiPSC-Hep
0.1-2 e6/mouse (IV)
2 to 17% Alb, CYP2E1 90% after 8 weeks
He et al. [113] Fah-/- mESC-Hep
(IS)
0.001%-12.5% after 8-10
weeks
ALT, AST, Bilirubin, Alb, succinylacetone,
tyrosine and phenylalanine secretion
30% of wt FAH activity in recipients
8/20 survived after 2nd
transplantation
Bandi et al. [120] CD17.NOD/SCID
(Rif + Phen + MCT, acute
liver failure)
hESC-MEC
4-6 e6/mouse
(IP with microcarriers)
Detection of transplanted cells
in the peritoneal cavity
Endogeneous hepatocytes proliferation
Phenobarbital metabolism
Ammonium chloride
11/11 after 14 days
(MCT 125, mild injury)
38% after 2 weeks
(MCT 160, strong
injury)
PSC: pluripotent stem cell, IV: intra venous injection; IS: intra splenic injection; IH: intra hepatic injection; IP: intra peritoneal injection; hESC-MEC: hESC meso-endodermal cells. Detection in serum of Alb (Albumin), AAT (alpha 1 anti-trypsin), ALT (alanine aminotransferase), AST (aspartate aminotransferase), bilirubin, AP (Alkaline phosphatase) and CYP2E1 activity.
Table 2: Summary of in vivo functions of PSC-Hep in mice.