L1CAM

Chr XXLR

L1 cell adhesion molecule

Also known as: CAML1, CD171, HSAS, HSAS1, HYCX, MASA, MIC5, N-CAM-L1

L1CAM encodes a neural cell adhesion molecule that mediates cell-cell adhesion and generates transmembrane signals, playing critical roles in neuronal migration, axonal growth and fasciculation, and synaptogenesis during brain development. Loss-of-function mutations cause X-linked recessive neurological syndromes including CRASH syndrome (corpus callosum hypoplasia, intellectual disability, aphasia, spastic paraplegia, and hydrocephalus), MASA syndrome, and X-linked congenital hydrocephalus. The pathogenic mechanism involves loss of protein function leading to disrupted neural development and connectivity.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
LOFmechanismXLRLOEUF 0.133 OMIM phenotypes
Clinical SummaryL1CAM
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Gene-Disease Validity (ClinGen)
L1 syndrome · XLDefinitive

Definitive — sufficient evidence for diagnostic panels

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.
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ClinVar Variants
74 unique Pathogenic / Likely Pathogenic· 136 VUS of 500 total submissions
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Clinical Trials
2 active or recruiting trials — potential therapeutic options may be available
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GeneReview available — L1CAM
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.13LOEUF
pLI 1.000
Z-score 6.22
OE 0.04 (0.020.13)
Highly constrained

Among the most LoF-intolerant genes (~top 3%)

Missense Constraint
2.84Z-score
OE missense 0.66 (0.610.72)
373 obs / 562.2 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.04 (0.020.13)
00.351.4
Missense OE0.66 (0.610.72)
00.61.4
Synonymous OE1.05
01.21.6
LoF obs/exp: 2 / 49.0Missense obs/exp: 373 / 562.2Syn Z: -0.66
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveL1CAM-related hydrocephalus due to stenosis of the aqueduct of sylviusLOFXLR
DN
0.4190th %ile
GOF
0.4974th %ile
LOF
0.67top 25%

The highest-scoring mechanism for this gene is loss-of-function (haploinsufficiency).

LOFprediction above median · 59% of P/LP variants are LoF · LOEUF 0.13

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

500 submitted variants in ClinVar

Classification Summary

Pathogenic50
Likely Pathogenic24
VUS136
Likely Benign179
Benign33
Conflicting2
50
Pathogenic
24
Likely Pathogenic
136
VUS
179
Likely Benign
33
Benign
2
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
26
1
23
0
50
Likely Pathogenic
18
5
1
0
24
VUS
2
118
14
2
136
Likely Benign
1
30
67
81
179
Benign
0
11
6
16
33
Conflicting
2
Total4716511199424

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

View in ClinVar →

Protein Context — Lollipop Plot

L1CAM · 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 →