Not Just an Alternative Energy Source: Diverse Biological Functions of Ketone Bodies and Relevance of HMGCS2 to Health and Disease

Ketogenesis, a mitochondrial metabolic pathway, occurs primarily in liver, but kidney, colon and retina are also capable of this pathway. It is activated during fasting and exercise, by “keto” diets, and in diabetes as well as during therapy with SGLT2 inhibitors. The principal ketone body is β-hydr...

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Main Authors: Varshini V. Suresh, Sathish Sivaprakasam, Yangzom D. Bhutia, Puttur D. Prasad, Muthusamy Thangaraju, Vadivel Ganapathy
Format: Article
Language:English
Published: MDPI AG 2025-04-01
Series:Biomolecules
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Online Access:https://www.mdpi.com/2218-273X/15/4/580
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author Varshini V. Suresh
Sathish Sivaprakasam
Yangzom D. Bhutia
Puttur D. Prasad
Muthusamy Thangaraju
Vadivel Ganapathy
author_facet Varshini V. Suresh
Sathish Sivaprakasam
Yangzom D. Bhutia
Puttur D. Prasad
Muthusamy Thangaraju
Vadivel Ganapathy
author_sort Varshini V. Suresh
collection DOAJ
description Ketogenesis, a mitochondrial metabolic pathway, occurs primarily in liver, but kidney, colon and retina are also capable of this pathway. It is activated during fasting and exercise, by “keto” diets, and in diabetes as well as during therapy with SGLT2 inhibitors. The principal ketone body is β-hydroxybutyrate, a widely recognized alternative energy source for extrahepatic tissues (brain, heart, muscle, and kidney) when blood glucose is sparse or when glucose transport/metabolism is impaired. Recent studies have identified new functions for β-hydroxybutyrate: it serves as an agonist for the G-protein-coupled receptor GPR109A and also works as an epigenetic modifier. Ketone bodies protect against inflammation, cancer, and neurodegeneration. HMGCS2, as the rate-limiting enzyme, controls ketogenesis. Its expression and activity are regulated by transcriptional and post-translational mechanisms with glucagon, insulin, and glucocorticoids as the principal participants. Loss-of-function mutations occur in HMGCS2 in humans, resulting in a severe metabolic disease. These patients typically present within a year after birth with metabolic acidosis, hypoketotic hypoglycemia, hepatomegaly, steatotic liver damage, hyperammonemia, and neurological complications. Nothing is known about the long-term consequences of this disease. This review provides an up-to-date summary of the biological functions of ketone bodies with a special focus on HMGCS2 in health and disease.
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spelling doaj-art-daeb45cab2e5481a87348a0234061c1a2025-08-20T03:14:20ZengMDPI AGBiomolecules2218-273X2025-04-0115458010.3390/biom15040580Not Just an Alternative Energy Source: Diverse Biological Functions of Ketone Bodies and Relevance of HMGCS2 to Health and DiseaseVarshini V. Suresh0Sathish Sivaprakasam1Yangzom D. Bhutia2Puttur D. Prasad3Muthusamy Thangaraju4Vadivel Ganapathy5Department of Cell Biology and Biochemistry, Texas Tech University Health Sciences Center, Lubbock, TX 79430, USADepartment of Cell Biology and Biochemistry, Texas Tech University Health Sciences Center, Lubbock, TX 79430, USADepartment of Cell Biology and Biochemistry, Texas Tech University Health Sciences Center, Lubbock, TX 79430, USADepartment of Biochemistry and Molecular Biology, Augusta University, Augusta, GA 30912, USADepartment of Biochemistry and Molecular Biology, Augusta University, Augusta, GA 30912, USADepartment of Cell Biology and Biochemistry, Texas Tech University Health Sciences Center, Lubbock, TX 79430, USAKetogenesis, a mitochondrial metabolic pathway, occurs primarily in liver, but kidney, colon and retina are also capable of this pathway. It is activated during fasting and exercise, by “keto” diets, and in diabetes as well as during therapy with SGLT2 inhibitors. The principal ketone body is β-hydroxybutyrate, a widely recognized alternative energy source for extrahepatic tissues (brain, heart, muscle, and kidney) when blood glucose is sparse or when glucose transport/metabolism is impaired. Recent studies have identified new functions for β-hydroxybutyrate: it serves as an agonist for the G-protein-coupled receptor GPR109A and also works as an epigenetic modifier. Ketone bodies protect against inflammation, cancer, and neurodegeneration. HMGCS2, as the rate-limiting enzyme, controls ketogenesis. Its expression and activity are regulated by transcriptional and post-translational mechanisms with glucagon, insulin, and glucocorticoids as the principal participants. Loss-of-function mutations occur in HMGCS2 in humans, resulting in a severe metabolic disease. These patients typically present within a year after birth with metabolic acidosis, hypoketotic hypoglycemia, hepatomegaly, steatotic liver damage, hyperammonemia, and neurological complications. Nothing is known about the long-term consequences of this disease. This review provides an up-to-date summary of the biological functions of ketone bodies with a special focus on HMGCS2 in health and disease.https://www.mdpi.com/2218-273X/15/4/580β-hydroxybutyrateketoacidosisketone body transportersGPR109Aepigenetic modificationHMGCS2
spellingShingle Varshini V. Suresh
Sathish Sivaprakasam
Yangzom D. Bhutia
Puttur D. Prasad
Muthusamy Thangaraju
Vadivel Ganapathy
Not Just an Alternative Energy Source: Diverse Biological Functions of Ketone Bodies and Relevance of HMGCS2 to Health and Disease
Biomolecules
β-hydroxybutyrate
ketoacidosis
ketone body transporters
GPR109A
epigenetic modification
HMGCS2
title Not Just an Alternative Energy Source: Diverse Biological Functions of Ketone Bodies and Relevance of HMGCS2 to Health and Disease
title_full Not Just an Alternative Energy Source: Diverse Biological Functions of Ketone Bodies and Relevance of HMGCS2 to Health and Disease
title_fullStr Not Just an Alternative Energy Source: Diverse Biological Functions of Ketone Bodies and Relevance of HMGCS2 to Health and Disease
title_full_unstemmed Not Just an Alternative Energy Source: Diverse Biological Functions of Ketone Bodies and Relevance of HMGCS2 to Health and Disease
title_short Not Just an Alternative Energy Source: Diverse Biological Functions of Ketone Bodies and Relevance of HMGCS2 to Health and Disease
title_sort not just an alternative energy source diverse biological functions of ketone bodies and relevance of hmgcs2 to health and disease
topic β-hydroxybutyrate
ketoacidosis
ketone body transporters
GPR109A
epigenetic modification
HMGCS2
url https://www.mdpi.com/2218-273X/15/4/580
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