FBN1

Chr 15AD

fibrillin 1

Also known as: ACMICD, ECTOL1, FBN, GPHYSD2, MASS, MFLS, MFS1, OCTD

The protein fibrillin-1 forms calcium-binding microfibrils that provide structural support in elastic and nonelastic connective tissue throughout the body, while the co-encoded hormone asprosin regulates glucose homeostasis. Mutations cause autosomal dominant connective tissue disorders including Marfan syndrome, MASS syndrome, ectopia lentis, Weill-Marchesani syndrome, Shprintzen-Goldberg syndrome, and several other related conditions. The pathogenic mechanism involves loss of function, leading to compromised structural integrity of connective tissues.

OMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
LOFmechanismADLOEUF 0.058 OMIM phenotypes
Clinical SummaryFBN1
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Gene-Disease Validity (ClinGen)
Marfan syndrome · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

3 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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Clinical Trials
7 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
0.05LOEUF
pLI 1.000
Z-score 11.46
OE 0.02 (0.010.05)
Highly constrained

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

Missense Constraint
5.06Z-score
OE missense 0.64 (0.610.68)
1038 obs / 1609.8 exp
Constrained

Extremely missense-constrained (top ~0.01%)

Observed / Expected Ratios
LoF OE0.02 (0.010.05)
00.351.4
Missense OE0.64 (0.610.68)
00.61.4
Synonymous OE0.99
01.21.6
LoF obs/exp: 3 / 158.8Missense obs/exp: 1038 / 1609.8Syn Z: 0.22
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
limitedFBN1-related isolated ectopia lentisOTHERAD
strongFBN1-related Weill-Marchesani syndromeOTHERAD
definitiveFBN1-related Marfan syndrome (biallelic)LOFAR
definitiveFBN1-related Marfan syndrome (monoallelic)LOFAD
DN
0.2997th %ile
GOF
0.3788th %ile
LOF
0.74top 10%

This gene has evidence for multiple mechanisms of pathogenicity (loss-of-function and dominant-negative). Both the Badonyi & Marsh prediction and the broader genomic evidence point to loss-of-function as the predominant mechanism. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

LOFprediction above median · 1 literature citation · LOEUF 0.05
DN1 literature citation

Literature Evidence

DNMFS is caused by dominant negative (DN) and haploinsufficient (HI) mutations of the FBN1 gene.PMID:33059708
LOFA pathogenic FBN1 mutation was found in 117 patients, of whom 79 patients were positive for a dominant negative mutation (67.5%) and 38 for a mutation causing haploinsufficiency (32.5%).PMID:25613431

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

FBN1 · 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

Lipodystrophy (Genetic or Acquired, Non HIV)

The LD Lync Study - Natural History Study of Lipodystrophy Syndromes

RECRUITING
NCT03087253University of MichiganStarted 2018-02-27
Marfan SyndromeMarfan's Syndrome With Cardiovascular Manifestations

Transcriptomic Study of Adult Population With Marfan Syndrome

RECRUITING
NCT05700175IRCCS Policlinico S. DonatoStarted 2021-11-02
Transcriptomic, epigenetic and proteomic analysis
Acute Aortic DissectionThoracic Aortic Aneurysm

Genetic Architecture of Acute Aortic Syndromes and Aortic Aneurysm.

RECRUITING
NCT06353607University Hospital, Basel, SwitzerlandStarted 2024-04-08
Rare DiseasesThoracic Aortic Aneurysm (TAA)Marfan Syndrome

Fibroblasts and Thoracic Aortic Aneurysms: in Vitro Characterization in With Marfan Syndrome and Genetic Aortic Diseases

ENROLLING BY INVITATION
NCT06786754IRCCS Policlinico S. DonatoStarted 2025-06-17
AortopathiesThoracic Aortic AneurysmAortic Valve Disease

Pathogenetic Basis of Aortopathy and Aortic Valve Disease

ACTIVE NOT RECRUITING
NCT03440697Yale UniversityStarted 2015-12-10
Creation of an Italian Network of Biobanks of Rare Diseases

ScATtEred Rare Disease Biobanks: a Model of Sample/Data Collection With susTainablE and Shared Criteria

RECRUITING
NCT06782230IRCCS Policlinico S. DonatoStarted 2024-08-31
Cardiomyopathy, HypertrophicCardiomyopathy, DilatedCardiomyopathy Restrictive

National Network for Cardiovascular Genomics: Advancing Cardiovascular Healthcare for Hereditary Diseases in Brazil's Unified Health System Through a Multicenter Registry

RECRUITING
NCT06546137Hospital do CoracaoStarted 2025-04-30
whole genome sequencing
Clinical Literature
Open Research Assistant →
Key Publications
Landmark & review papers · by relevance
PubMed
Marfan syndrome.
Milewicz DM et al.·Nat Rev Dis Primers
2021Review
Genetics of short stature.
Nicolae R et al.·Curr Opin Pediatr
2025Review
[Latest advances in the diagnosis and treatment of Marfan syndrome].
Yang ST et al.·Zhongguo Dang Dai Er Ke Za Zhi
2022Review
Top 5 results · since 2015Search PubMed ↗