Table 1.

Physiological functions of UPR components in mouse models and their genetic association with human disease

Gene Factors that regulate expression Phenotypes of knockout mouse model Genetic association with human diseases References 
IRE1α N.A. (1) Embryonic lethality at E12.5 due to liver hypoplasia; (2) Liver deletion: hypolipidemia (1) Human somatic cancers Zhang et al., 2005, 2011; Greenman et al., 2007  
XBP1s XBP1s and ATF6α (1) Embryonic lethality at E13.5 due to liver hypoplasia; (2) Liver deletion: hypolipidemia; (3) Intestinal epithelial cell deletion: enteritis; (4) Pancreatic acinar cell deletion: extensive pancreas regeneration; (5) Pancreatic β cell deletion: hyperglycemia; (6) Neuron deletion: leptin resistance (1) Inflammatory bowel disease; (2) Schizophrenia in the Japanese population; (3) Bipolar disorder; (4) Ischemic stroke Kakiuchi et al., 2003b, 2004; Kaser et al., 2008; Yilmaz et al., 2010  
ATF6α N.A. (1) Susceptible to pharmacologically induced ER stress (1) Type 2 diabetes and pre-diabetic traits; (2) Increased plasma cholesterol levels Chu et al., 2007; Wu et al., 2007; Meex et al., 2009  
CREBH PPARα, HNF4α, and ATF6α (1) Hypoferremia and spleen iron sequestration; (2) Hyperlipidemia; (3) Liver knockdown: fasting hyperglycemia (1) Extreme hypertriglyceridemia Zhang et al., 2006; Vecchi et al., 2009; J.H. Lee et al., 2011  
PERK N.A. (1) Neonatal hyperglycemia (1) Wolcott-Rallison syndrome; (2) Supranuclear palsy Delépine et al., 2000; Höglinger et al., 2011  
ATF4 CHOP (1) Delayed bone formation; (2) Severe fetal anemia; (3) Increased insulin sensitivity; (4) Defects in long-term memory N.A. Elefteriou et al., 2006; Costa-Mattioli et al., 2007; Yamaguchi et al., 2008  
CHOP ATF4 and ATF6α (1) Protected from pharmacologically induced ER stress; (2) Protected from type 2 diabetes; (3) Protected from atherosclerosis; (4) Protected from leukodystrophy Pelizaeus-Merzbacher disease (1) Early-onset type 2 diabetes in Italians Oyadomari et al., 2002; Marciniak et al., 2004; Silva et al., 2005; Gragnoli, 2008; Song et al., 2008  
WFS1 XBP1s (1) Diabetes due to insufficient insulin secretion; (2) Growth retardation (1) Wolfram syndrome; (2) Risk of type 2 diabetes in Japanese and European populations Karasik et al., 1989; Inoue et al., 1998; Ishihara et al., 2004; Mita et al., 2008  
ORMDL3 N.A. N.A. (1) Ulcerative colitis; (2) Risk of childhood asthma Hjelmqvist et al., 2002; Breslow et al., 2010; McGovern et al., 2010  
Grp78 (BiP) ATF6α and ATF4 (1) Embryonic lethality at E3.5 due to impaired embryo peri-implantation; (2) Liver deletion: simultaneous liver damage and hepatic steatosis (1) Bipolar disorder Kakiuchi et al., 2005; Luo et al., 2006; R. Ye et al., 2010  
SIL1 XBP1s (1) Adult-onset ataxia with cerebellar Purkinje cell loss (1) Marinesco-Sjogren syndrome; (2) Alzheimer’s disease Tyson and Stirling, 2000; Anttonen et al., 2005; Zhao et al., 2005, 2010  
Grp94 XBP1s, ATF6α, and ATF4 (1) Embryonic lethality at E7; (2) B cell deletion: reduced antibody production; (3) Bone marrow deletion: hematopoietic stem cell expansion (1) Bipolar disorder Kakiuchi et al., 2007; Mao et al., 2010  
P58 IPK XBP1s and ATF6α (1) Diabetes N.A. Ladiges et al., 2005  
Calnexin XBP1s and ATF6α (1) Postnatal death; (2) Motor disorder N.A. Denzel et al., 2002  
Calreticulin XBP1s and ATF6α (1) Embryonic lethality at E14.5 (1) A case of schizophrenia Aghajani et al., 2006  
Seleno-protein S N.A. (1) Disturbed redox homeostasis in the liver and cataract development in eyes (1) Inflammatory response; (2) Non-small cell lung cancer Hart et al., 2011; Kasaikina et al., 2011  
Gene Factors that regulate expression Phenotypes of knockout mouse model Genetic association with human diseases References 
IRE1α N.A. (1) Embryonic lethality at E12.5 due to liver hypoplasia; (2) Liver deletion: hypolipidemia (1) Human somatic cancers Zhang et al., 2005, 2011; Greenman et al., 2007  
XBP1s XBP1s and ATF6α (1) Embryonic lethality at E13.5 due to liver hypoplasia; (2) Liver deletion: hypolipidemia; (3) Intestinal epithelial cell deletion: enteritis; (4) Pancreatic acinar cell deletion: extensive pancreas regeneration; (5) Pancreatic β cell deletion: hyperglycemia; (6) Neuron deletion: leptin resistance (1) Inflammatory bowel disease; (2) Schizophrenia in the Japanese population; (3) Bipolar disorder; (4) Ischemic stroke Kakiuchi et al., 2003b, 2004; Kaser et al., 2008; Yilmaz et al., 2010  
ATF6α N.A. (1) Susceptible to pharmacologically induced ER stress (1) Type 2 diabetes and pre-diabetic traits; (2) Increased plasma cholesterol levels Chu et al., 2007; Wu et al., 2007; Meex et al., 2009  
CREBH PPARα, HNF4α, and ATF6α (1) Hypoferremia and spleen iron sequestration; (2) Hyperlipidemia; (3) Liver knockdown: fasting hyperglycemia (1) Extreme hypertriglyceridemia Zhang et al., 2006; Vecchi et al., 2009; J.H. Lee et al., 2011  
PERK N.A. (1) Neonatal hyperglycemia (1) Wolcott-Rallison syndrome; (2) Supranuclear palsy Delépine et al., 2000; Höglinger et al., 2011  
ATF4 CHOP (1) Delayed bone formation; (2) Severe fetal anemia; (3) Increased insulin sensitivity; (4) Defects in long-term memory N.A. Elefteriou et al., 2006; Costa-Mattioli et al., 2007; Yamaguchi et al., 2008  
CHOP ATF4 and ATF6α (1) Protected from pharmacologically induced ER stress; (2) Protected from type 2 diabetes; (3) Protected from atherosclerosis; (4) Protected from leukodystrophy Pelizaeus-Merzbacher disease (1) Early-onset type 2 diabetes in Italians Oyadomari et al., 2002; Marciniak et al., 2004; Silva et al., 2005; Gragnoli, 2008; Song et al., 2008  
WFS1 XBP1s (1) Diabetes due to insufficient insulin secretion; (2) Growth retardation (1) Wolfram syndrome; (2) Risk of type 2 diabetes in Japanese and European populations Karasik et al., 1989; Inoue et al., 1998; Ishihara et al., 2004; Mita et al., 2008  
ORMDL3 N.A. N.A. (1) Ulcerative colitis; (2) Risk of childhood asthma Hjelmqvist et al., 2002; Breslow et al., 2010; McGovern et al., 2010  
Grp78 (BiP) ATF6α and ATF4 (1) Embryonic lethality at E3.5 due to impaired embryo peri-implantation; (2) Liver deletion: simultaneous liver damage and hepatic steatosis (1) Bipolar disorder Kakiuchi et al., 2005; Luo et al., 2006; R. Ye et al., 2010  
SIL1 XBP1s (1) Adult-onset ataxia with cerebellar Purkinje cell loss (1) Marinesco-Sjogren syndrome; (2) Alzheimer’s disease Tyson and Stirling, 2000; Anttonen et al., 2005; Zhao et al., 2005, 2010  
Grp94 XBP1s, ATF6α, and ATF4 (1) Embryonic lethality at E7; (2) B cell deletion: reduced antibody production; (3) Bone marrow deletion: hematopoietic stem cell expansion (1) Bipolar disorder Kakiuchi et al., 2007; Mao et al., 2010  
P58 IPK XBP1s and ATF6α (1) Diabetes N.A. Ladiges et al., 2005  
Calnexin XBP1s and ATF6α (1) Postnatal death; (2) Motor disorder N.A. Denzel et al., 2002  
Calreticulin XBP1s and ATF6α (1) Embryonic lethality at E14.5 (1) A case of schizophrenia Aghajani et al., 2006  
Seleno-protein S N.A. (1) Disturbed redox homeostasis in the liver and cataract development in eyes (1) Inflammatory response; (2) Non-small cell lung cancer Hart et al., 2011; Kasaikina et al., 2011  

N.A., not applicable.

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