Abstract
Whole body cytosolic phosphoenolpyruvate carboxykinase knockout (PEPCK-C KO) mice die early after birth with profound hypoglycemia therefore masking the role of PEPCK-C in adult, non-gluconeogenic tissues where it is expressed. To investigate whether PEPCK-C deletion in the liver was critically responsible for the hypoglycemic phenotype, we reexpress this enzyme in the liver of PEPCK-C KO pups by early postnatal administration of PEPCK-C-expressing adenovirus. This maneuver was sufficient to partially rescue hypoglycemia and allow the pups to survive and identifies the liver as a critical organ, and hypoglycemia as the critical pathomechanism, leading to early postnatal death in the whole-body PEPCK-C knockout mice. Pathology assessment of survivors also suggest a possible role for PEPCK-C in lung maturation and muscle metabolism.
Keywords:
Gluconeogenesis; Hepatic lipidosis; KO; Liver; Neonatal hypoglycemia; PEPCK.
MeSH terms
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Animals
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Animals, Newborn
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Brain / enzymology
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Brain / metabolism
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Brain / pathology
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Carbohydrate Metabolism, Inborn Errors / enzymology
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Carbohydrate Metabolism, Inborn Errors / physiopathology
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Carbohydrate Metabolism, Inborn Errors / therapy
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Carbohydrate Metabolism, Inborn Errors / veterinary*
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Crosses, Genetic
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Gene Transfer Techniques
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Gluconeogenesis
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Heterozygote
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Hypoglycemia / etiology
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Hypoglycemia / metabolism
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Hypoglycemia / pathology
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Hypoglycemia / prevention & control*
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Lipid Droplets / metabolism
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Lipid Droplets / pathology
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Lipid Metabolism
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Lipidoses / etiology
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Liver / enzymology*
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Liver / metabolism
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Liver / pathology
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Liver Diseases / enzymology
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Liver Diseases / physiopathology
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Liver Diseases / therapy
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Liver Diseases / veterinary*
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Lung / enzymology
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Lung / metabolism*
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Lung / pathology
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Mice, Inbred C57BL
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Mice, Knockout
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Muscle, Skeletal / enzymology
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Muscle, Skeletal / metabolism*
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Muscle, Skeletal / pathology
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Neurons / enzymology
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Neurons / metabolism
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Neurons / pathology
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Phosphoenolpyruvate Carboxykinase (GTP) / deficiency*
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Phosphoenolpyruvate Carboxykinase (GTP) / genetics
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Phosphoenolpyruvate Carboxykinase (GTP) / metabolism*
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Phosphoenolpyruvate Carboxykinase (GTP) / therapeutic use
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Recombinant Proteins / metabolism
Substances
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Recombinant Proteins
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Phosphoenolpyruvate Carboxykinase (GTP)
Supplementary concepts
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Phosphoenolpyruvate carboxykinase deficiency