GDF3

Chr 12AD

growth differentiation factor 3

Also known as: KFS3, MCOP7, MCOPCB6

This gene encodes a secreted growth factor of the TGF-beta superfamily that regulates early embryonic development, particularly ocular and skeletal formation. Autosomal dominant mutations cause Klippel-Feil syndrome, isolated microphthalmia, and microphthalmia with coloboma, affecting craniofacial and skeletal development. The gene shows low constraint to loss-of-function variation, consistent with its dominant inheritance pattern.

Summary from RefSeq, OMIM, UniProt
Research Assistant →

Primary Disease Associations & Inheritance

Klippel-Feil syndrome 3, autosomal dominantMIM #613702
Microphthalmia, isolated 7MIM #613704
AD
Microphthalmia, isolated, with coloboma 6MIM #613703
AD
UniProtMicrophthalmia/Coloboma 6
0
Active trials
9
Pubs (1 yr)
47
P/LP submissions
4%
P/LP missense
1.31
LOEUF
DN
Mechanism· predicted
Clinical SummaryGDF3
Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
46 unique Pathogenic / Likely Pathogenic· 67 VUS of 144 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
1.31LOEUF
pLI 0.000
Z-score 0.84
OE 0.73 (0.431.31)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
-0.12Z-score
OE missense 1.02 (0.911.15)
202 obs / 197.4 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.73 (0.431.31)
00.351.4
Missense OE1.02 (0.911.15)
00.61.4
Synonymous OE0.99
01.21.6
LoF obs/exp: 8 / 11.0Missense obs/exp: 202 / 197.4Syn Z: 0.10
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
limitedGDF3-related microphthalmia with colobomaOTHERAD
limitedGDF3-related multiple malformationsOTHERAD
limitedGDF3-related microphthalmia, isolatedOTHERAD
DN
0.6162th %ile
GOF
0.3986th %ile
LOF
0.3162th %ile

The highest-scoring mechanism for this gene is dominant-negative.

DNprediction above median

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.

ClinVar Variant Classifications

144 submitted variants in ClinVar

Classification Summary

Pathogenic42
Likely Pathogenic4
VUS67
Likely Benign14
Benign14
Conflicting2
42
Pathogenic
4
Likely Pathogenic
67
VUS
14
Likely Benign
14
Benign
2
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
42
0
42
Likely Pathogenic
0
2
2
0
4
VUS
2
55
9
1
67
Likely Benign
0
5
1
8
14
Benign
0
3
7
4
14
Conflicting
2
Total2656113143

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

View in ClinVar →

Protein Context — Lollipop Plot

GDF3 · protein map & ClinVar variants

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

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

Search ClinicalTrials.gov →
Clinical Literature
Open Research Assistant →
Recent Gene-Specific Literature
Gene in title · MEDLINE · newest first
Europe PMC