FLNA

Chr XX-linkedXLRXLD

filamin A

Also known as: ABP-280, ABPX, CSBS, CVD1, FGS2, FLN, FLN-A, FLN1

The encoded protein is an actin-binding protein that crosslinks actin filaments and links them to membrane glycoproteins, functioning in cytoskeletal remodeling to control cell shape and migration. Loss-of-function mutations cause X-linked periventricular nodular heterotopia, otopalatodigital syndromes, frontometaphyseal dysplasia, Melnick-Needles syndrome, and congenital intestinal pseudoobstruction, with inheritance patterns varying from X-linked recessive (typically for periventricular heterotopia in females) to X-linked dominant (for the skeletal dysplasias). The protein interacts with integrins and transmembrane complexes, and its disruption leads to abnormal neuronal migration and skeletal development.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
MultiplemechanismX-linked/XLR/XLDLOEUF 0.0810 OMIM phenotypes
Clinical SummaryFLNA
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Gene-Disease Validity (ClinGen)
periventricular nodular heterotopia · XLDefinitive

Definitive — sufficient evidence for diagnostic panels

2 total gene-disease associations curated

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
25 unique Pathogenic / Likely Pathogenic· 143 VUS of 405 total submissions
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Clinical Trials
1 active or recruiting trial — potential therapeutic options may be available
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GeneReview available — FLNA
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Dual constrained — LoF & missense intolerant
LoF Constraint
0.08LOEUF
pLI 1.000
Z-score 7.89
OE 0.03 (0.010.08)
Highly constrained

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

Missense Constraint
3.78Z-score
OE missense 0.70 (0.660.74)
867 obs / 1241.9 exp
Constrained

Highly missense-constrained (top ~0.1%)

Observed / Expected Ratios
LoF OE0.03 (0.010.08)
00.351.4
Missense OE0.70 (0.660.74)
00.61.4
Synonymous OE1.16
01.21.6
LoF obs/exp: 2 / 76.5Missense obs/exp: 867 / 1241.9Syn Z: -2.98
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveFLNA-related epileptic encephalopathyOTHERmonoallelic_X_heterozygous
definitiveFLNA-related otopalatodigital syndromeOTHERXLR
definitiveFLNA-related frontometaphyseal dysplasiaGOFXLR
definitiveFLNA-related Melnick-Needles syndromeOTHERmonoallelic_X_heterozygous
definitiveFLNA-related terminal osseous dysplasiaLOFXLR
definitiveFLNA-related periventricular nodular heterotopiaLOFmonoallelic_X_heterozygous
definitiveFLNA-related congenital idiopathic intestinal pseudoobstructionOTHERXLR
DN
0.3296th %ile
GOF
0.5856th %ile
LOF
0.73top 10%

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

LOFprediction above median · 84% of P/LP variants are LoF · LOEUF 0.08

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. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

405 submitted variants in ClinVar

Classification Summary

Pathogenic20
Likely Pathogenic5
VUS143
Likely Benign115
Benign9
Conflicting1
20
Pathogenic
5
Likely Pathogenic
143
VUS
115
Likely Benign
9
Benign
1
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
17
0
3
0
20
Likely Pathogenic
4
1
0
0
5
VUS
2
127
10
4
143
Likely Benign
0
10
53
52
115
Benign
0
5
1
3
9
Conflicting
1
Total231436759293

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

View in ClinVar →

Protein Context — Lollipop Plot

FLNA · 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 →
Full-Text Mentions
NLP-detected gene mentions in article bodies · via PubTator3
PubTator3
Top 5 full-text resultsSearch PubTator3 ↗
Key Publications
Landmark & review papers · by relevance
PubMed
Top 5 results · since 2015Search PubMed ↗