HMGCS2 (P54868) variants and mutations

HMGCS2 (also known as P54868) is a human protein-coding gene encoding a hydroxymethylglutaryl-CoA synthase, mitochondrial protein. It catalyzes the rate-limiting mitochondrial step of ketone-body synthesis, allowing the liver to convert fatty-acid-derived acetyl-CoA into ketones during fasting. Biallelic deficiency causes impaired ketogenesis with fasting hypoglycemia and potentially severe metabolic decompensation. This analysis covers 853 HMGCS2 variants and mutations. Of these, 78% have computational variant effect predictions. Disease context includes 3-hydroxy-3-methylglutaryl-CoA synthase deficiency, hereditary disease, and hepatocellular carcinoma. Example HMGCS2 variants include R3C, R3G, and R3H.

Variant analysis overview

Variant and mutation evidence

Clinical, disease, and population context

Protein structure and variant hotspots

Data sources

Evidence in this analysis draws on EBI Proteins Variation, UniProt, gnomAD v4, EuropePMC, 3D Hotspot Analysis, Interaction Network Analysis, Protein Data Bank, AlphaFold DB, gnomAD constraint, Open Targets, MaveDB, LitVar.

Notable HMGCS2 variants

Examples include R3C, R3G, R3H, T6I, P7A, P7Q, V8A, V8M. Listed records include available protein-change notation, database identifiers, clinical classifications, computational predictions, population evidence, experimental measurements, and disease context.