HMGCR

Chr 5AR

3-hydroxy-3-methylglutaryl-CoA reductase

Also known as: LDLCQ3, LGMDR28, MYPLG

HMG-CoA reductase catalyzes the rate-limiting step in cholesterol synthesis by converting HMG-CoA to mevalonic acid, playing a critical role in cellular cholesterol homeostasis. Mutations cause cerebrotendinous xanthomatosis and other sterol biosynthesis disorders with autosomal recessive inheritance, affecting the nervous system, tendons, and cardiovascular system. This gene is highly constrained against loss-of-function variants, indicating that complete loss of function is likely incompatible with normal development.

OMIMResearchSummary from RefSeq, UniProt
LOFmechanismARLOEUF 0.263 OMIM phenotypes
Clinical SummaryHMGCR
🧬
Gene-Disease Validity (ClinGen)
autosomal recessive limb-girdle muscular dystrophy · ARModerate

Moderate evidence — consider for supplementary testing

Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 1.00). One damaged copy is likely sufficient to cause disease.
💊
Clinical Trials
4 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Dual constrained — LoF & missense intolerant
LoF Constraint
0.26LOEUF
pLI 0.999
Z-score 5.43
OE 0.13 (0.070.26)
Highly constrained

Highly LoF-intolerant (top ~10% of genes)

Missense Constraint
3.76Z-score
OE missense 0.53 (0.470.58)
262 obs / 498.1 exp
Constrained

Highly missense-constrained (top ~0.1%)

Observed / Expected Ratios
LoF OE0.13 (0.070.26)
00.351.4
Missense OE0.53 (0.470.58)
00.61.4
Synonymous OE0.90
01.21.6
LoF obs/exp: 6 / 45.6Missense obs/exp: 262 / 498.1Syn Z: 1.01
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
moderateHMGCR-related limb-girdle muscular dystrophyLOFAR

Predictions shown for reference only — model trained on dominant genes, not applicable to AR conditions.

DN
0.4190th %ile
GOF
0.4678th %ile
LOF
0.67top 25%

The Badonyi & Marsh prediction model was trained exclusively on dominant disease genes. Predictions are not reliable for genes with autosomal recessive inheritance and are shown at reduced opacity for reference only.

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

HMGCR · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold
Clinical Literature
Open Research Assistant →