long chain acyl-CoA dehydrogenase deficiency

disease
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Also known as ACADL deficiencyacyl-CoA dehydrogenase, long-chain deficiencyinborn error of long-chain-acyl-CoA dehydrogenase activityinborn long-chain-acyl-CoA dehydrogenase activity disorderLCADLCAD deficiencylong-chain acyl-CoA dehydrogenase deficiencylong-chain acyl-Coenzyme A dehydrogenase deficiencyrare inborn error of long-chain-acyl-CoA dehydrogenase activity

Summary

long chain acyl-CoA dehydrogenase deficiency (MONDO:0020531) is a disease with 1 cohort gene.

At a glance

  • Cohort genes: 1
  • ClinVar variants: 4

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

FieldValue
Canonical namelong chain acyl-CoA dehydrogenase deficiency
Mondo IDMONDO:0020531
MeSHC535690
Orphanet99900
ICD-11692829041
NCITC84537
SNOMED CT237996001
UMLSC0220711
MedGen65087
GARD0009700
Is cancer (heuristic)no

Also known as: ACADL deficiency · acyl-CoA dehydrogenase, long-chain deficiency · inborn error of long-chain-acyl-CoA dehydrogenase activity · inborn long-chain-acyl-CoA dehydrogenase activity disorder · LCAD · LCAD deficiency · long chain acyl-CoA dehydrogenase deficiency · long-chain acyl-CoA dehydrogenase deficiency · long-chain acyl-Coenzyme A dehydrogenase deficiency · rare inborn error of long-chain-acyl-CoA dehydrogenase activity

Data availability: 4 ClinVar variants.

Disease family

Classification path: disease › human disease › disease by etiologic mechanism › disease of genetic or genomic mechanism › hereditary diseaseinborn errors of metabolisminborn disorder of energy metabolismdisorder of fatty acid and ketone body metabolism › disorder of fatty acid oxidation and ketogenesis › long chain acyl-CoA dehydrogenase deficiency

Related subtypes (9): very long chain acyl-CoA dehydrogenase deficiency, carnitine-acylcarnitine translocase deficiency, systemic primary carnitine deficiency disease, 3-hydroxy-3-methylglutaric aciduria, 3-hydroxy-3-methylglutaryl-CoA synthase deficiency, long chain 3-hydroxyacyl-CoA dehydrogenase deficiency, acyl-CoA dehydrogenase 9 deficiency, acyl-CoA dehydrogenase deficiency, 3-hydroxyacyl-CoA dehydrogenase deficiency

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

ClinVar germline variants

4 retrieved; paginated sample, class counts are floors:

3 conflicting classifications of pathogenicity, 1 uncertain significance

ClinVarVariant (HGVS)GeneClassificationReview
439352NM_001608.4(ACADL):c.932G>T (p.Arg311Met)ACADLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
593544NM_001608.4(ACADL):c.722G>C (p.Gly241Ala)ACADLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
973447NM_001608.4(ACADL):c.799C>T (p.Arg267Trp)ACADLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
3778929NM_001608.4(ACADL):c.935_936insTGTTATGTTTAA (p.Asn312_Tyr313insValMetPheAsn)ACADLUncertain significancecriteria provided, single submitter

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 0 · Orphanet: 0 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Cohort genes → proteins

1 cohort genes, 1 distinct canonical proteins.

Evidence partition

SubsetGenes
multi_evidence1

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
ACADLHGNC:88ENSG00000115361P28330Long-chain specific acyl-CoA dehydrogenase, mitochondrialclinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
ACADLLong-chain specific acyl-CoA dehydrogenase, mitochondrialLong-chain specific acyl-CoA dehydrogenase is one of the acyl-CoA dehydrogenases that catalyze the first step of mitochondrial fatty acid beta-oxidation (FAO), breaking down fatty acids into acetyl-CoA and allowing the production of energy…

Protein-family classification

Druggable: 0 · Difficult: 0 · Unknown: 1 · Druggable fraction: 0.0

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

FamilyGenesFoldFDR
Other/Unknown11.8×0.558

Per-gene assignment

SymbolFamilyDruggable?ECInterPro (top 3)
ACADLOther/UnknownnoAcyl-CoA_DH_CS, AcylCoA_DH/ox_M, AcylCo_DH/oxidase_C

Expression context

Cohort genes with no expression data: 0.

1 cohort gene are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

BucketGenes
narrow (1-5 tissues)0
moderate (6-20)0
broad (>20)1
unknown0

Top tissues across cohort

TissueCohort genes
body of pancreas1
popliteal artery1
tibial artery1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
ACADL208broadmarkerbody of pancreas, popliteal artery, tibial artery

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
ACADL2,251

Structural data

PDB: 1 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

SymbolUniProtPDB entries
ACADLP283303

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 8. Enrichment computed across 1 evidence-associated genes (1 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

PathwayCohort genesFoldFDRSample cohort genes
Beta oxidation of myristoyl-CoA to lauroyl-CoA13806.7×0.001ACADL
Beta oxidation of lauroyl-CoA to decanoyl-CoA-CoA12284.0×0.001ACADL
mitochondrial fatty acid beta-oxidation of unsaturated fatty acids11903.3×0.001ACADL
mitochondrial fatty acid beta-oxidation of saturated fatty acids11631.4×0.001ACADL
Mitochondrial Fatty Acid Beta-Oxidation1380.7×0.004ACADL
Fatty acid metabolism1131.3×0.010ACADL
Metabolism of lipids131.6×0.036ACADL
Metabolism111.6×0.086ACADL

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO termCohort genesFoldFDRSample cohort genes
carnitine catabolic process116852.0×6e-04ACADL
carnitine metabolic process, CoA-linked15617.3×9e-04ACADL
long-chain fatty acid catabolic process12808.7×0.001ACADL
negative regulation of fatty acid oxidation11685.2×0.001ACADL
fatty acid beta-oxidation using acyl-CoA dehydrogenase11404.3×0.001ACADL
regulation of cholesterol metabolic process11123.5×0.001ACADL
negative regulation of fatty acid biosynthetic process1887.0×0.002ACADL
temperature homeostasis1648.1×0.002ACADL
lipid catabolic process1244.2×0.005ACADL
positive regulation of cold-induced thermogenesis1163.6×0.006ACADL

Therapeutics

Drug target analysis

Approved (phase 4): 0 · Phase ≥3: 0 · Phased (≥1): 0 · Undrugged: 1

Druggability breadth: 0 of 1 evidence-associated genes (0%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

SymbolMoleculesMax phase
ACADL00

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 0.

Pharmacogenomics

Cohort genes with a PharmGKB record: 1; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Chemical tractability of cohort targets

0 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

TierDefinitionGenesSymbols
AApproved (phase 4 drug)0
BPhased (≥1) drug, not yet approved0
CDruggable family + PDB, no drug0
DDruggable family + AlphaFold only, no drug0
EDifficult family or no structure, no drug1ACADL

Undrugged target profiles

1 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

SymbolChEMBL assaysDrugged partners (top 3)
ACADL0

Clinical trials & evidence

Clinical trials

Clinical trials: 0.