FLI1

Chr 11ADAR

Fli-1 proto-oncogene, ETS transcription factor

Also known as: BDPLT21, EWSR2, FLI-1, SIC-1

FLI1 encodes a transcription factor with an ETS DNA-binding domain that functions as a sequence-specific transcriptional activator. Mutations cause bleeding disorder, platelet-type, 21, inherited in both autosomal dominant and autosomal recessive patterns. The gene is highly constrained against loss-of-function variants (pLI 0.99, LOEUF 0.28), indicating intolerance to functional disruption.

Summary from RefSeq, OMIM, UniProt
Research Assistant →

Primary Disease Associations & Inheritance

Bleeding disorder, platelet-type, 21MIM #617443
ADAR
UniProtEwing sarcoma
2
Active trials
171
Pubs (1 yr)
18
P/LP submissions
6%
P/LP missense
0.28
LOEUF· LoF intol.
LOF
Mechanism· predicted
Clinical SummaryFLI1
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Gene-Disease Validity (ClinGen)
bleeding disorder, platelet-type, 21 · ADModerate

Moderate evidence — consider for supplementary testing

Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 0.99). One damaged copy is likely sufficient to cause disease.
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ClinVar Variants
18 unique Pathogenic / Likely Pathogenic· 80 VUS of 200 total submissions
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Clinical Trials
2 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.28LOEUF
pLI 0.989
Z-score 4.00
OE 0.09 (0.040.28)
Highly constrained

Highly LoF-intolerant (top ~10% of genes)

Missense Constraint
2.40Z-score
OE missense 0.59 (0.520.68)
164 obs / 276.4 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.09 (0.040.28)
00.351.4
Missense OE0.59 (0.520.68)
00.61.4
Synonymous OE1.09
01.21.6
LoF obs/exp: 2 / 22.5Missense obs/exp: 164 / 276.4Syn Z: -0.79
DN
0.3892th %ile
GOF
0.3094th %ile
LOF
0.82top 5%

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

LOFprediction above median · 1 literature citation · LOEUF 0.28

Literature Evidence

LOFFli1 haploinsufficiency underlies Paris-Trousseau thrombopeniaPMID:15525489

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

200 submitted variants in ClinVar

Classification Summary

Pathogenic13
Likely Pathogenic5
VUS80
Likely Benign37
Benign43
Conflicting1
13
Pathogenic
5
Likely Pathogenic
80
VUS
37
Likely Benign
43
Benign
1
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
1
0
12
0
13
Likely Pathogenic
1
1
3
0
5
VUS
3
69
8
0
80
Likely Benign
0
0
14
23
37
Benign
0
0
39
4
43
Conflicting
1
Total5707627179

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

View in ClinVar →

Protein Context — Lollipop Plot

FLI1 · protein map & ClinVar variants

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

Clinical Literature
Open Research Assistant →
Key Publications
Landmark & review papers · by relevance
PubMed
The Molecular Taxonomy of Primary Prostate Cancer.
Cancer Genome Atlas Research Network·Cell
2015
Emerging therapies in Ewing sarcoma.
Strauss SJ et al.·Curr Opin Oncol
2024Review
Pseudomyogenic Hemangioendothelioma.
Al-Qaderi A et al.·Arch Pathol Lab Med
2019Review
Pseudomyogenic Hemangioendothelioma.
Ansai SI et al.·J Nippon Med Sch
2019Case report
Top 5 results · since 2015Search PubMed ↗