CPT1B

Chr 22

carnitine palmitoyltransferase 1B

Also known as: CPT1-M, CPT1M, CPTI, CPTI-M, M-CPT1, MCCPT1, MCPT1

CPT1B encodes carnitine palmitoyltransferase IB, the rate-controlling enzyme that catalyzes the transfer of long-chain fatty acids onto carnitine for mitochondrial uptake and subsequent beta-oxidation in muscle. Mutations cause CPT1B deficiency, an autosomal recessive disorder presenting with exercise intolerance, muscle weakness, and rhabdomyolysis triggered by prolonged exercise or fasting. The gene shows low constraint against loss-of-function variants, consistent with the recessive inheritance pattern of this fatty acid oxidation disorder.

GeneReviewsOMIMResearchSummary from RefSeq, UniProt
DNmechanismLOEUF 0.83
Clinical SummaryCPT1B
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Gene-Disease Validity (ClinGen)
inherited fatty acid metabolism disorder · UDNo Known Disease Relationship

No known disease relationship

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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ClinVar Variants
128 unique Pathogenic / Likely Pathogenic· 58 VUS of 200 total submissions
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GeneReview available — CPT1B
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.83LOEUF
pLI 0.000
Z-score 2.44
OE 0.59 (0.420.83)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
0.49Z-score
OE missense 0.94 (0.871.01)
449 obs / 479.5 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.59 (0.420.83)
00.351.4
Missense OE0.94 (0.871.01)
00.61.4
Synonymous OE0.98
01.21.6
LoF obs/exp: 24 / 40.8Missense obs/exp: 449 / 479.5Syn Z: 0.25
DN
0.7229th %ile
GOF
0.5367th %ile
LOF
0.2582th %ile

The highest-scoring mechanism for this gene is dominant-negative.

DNprediction above median

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

200 submitted variants in ClinVar

Classification Summary

Pathogenic127
Likely Pathogenic1
VUS58
Likely Benign6
Benign8
127
Pathogenic
1
Likely Pathogenic
58
VUS
6
Likely Benign
8
Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
127
0
127
Likely Pathogenic
0
0
1
0
1
VUS
2
44
12
0
58
Likely Benign
0
3
3
0
6
Benign
0
0
5
3
8
Total2471483200

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

Protein Context — Lollipop Plot

CPT1B · protein map & ClinVar variants

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

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

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Clinical Literature
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