FKBP4

Chr 12

FKBP prolyl isomerase 4

Also known as: FKBP51, FKBP52, FKBP59, HBI, Hsp56, PPIase, p52

The protein is a cis-trans prolyl isomerase and co-chaperone that associates with heat shock proteins to facilitate intracellular trafficking of steroid hormone receptors, controls neuronal growth cones through TRPC1 channel regulation, and inhibits tau-mediated microtubule assembly. Mutations cause autosomal recessive intellectual disability and developmental delay. This gene is highly constrained against loss-of-function variants, suggesting that complete protein loss is not well tolerated.

Summary from RefSeq, UniProt
Research Assistant →
3
Active trials
23
Pubs (1 yr)
63
P/LP submissions
0%
P/LP missense
0.50
LOEUF
Multiple*
Mechanism· predicted
Clinical SummaryFKBP4
Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.26) despite low pLI — interpret in context.
📋
ClinVar Variants
63 unique Pathogenic / Likely Pathogenic· 46 VUS of 135 total submissions
💊
Clinical Trials
3 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.50LOEUF
pLI 0.155
Z-score 3.34
OE 0.26 (0.140.50)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
1.21Z-score
OE missense 0.79 (0.700.88)
197 obs / 250.8 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.26 (0.140.50)
00.351.4
Missense OE0.79 (0.700.88)
00.61.4
Synonymous OE1.14
01.21.6
LoF obs/exp: 6 / 23.5Missense obs/exp: 197 / 250.8Syn Z: -1.06
DN
0.6743th %ile
GOF
0.7027th %ile
LOF
0.2777th %ile

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

GOFprediction above median
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

135 submitted variants in ClinVar

Classification Summary

Pathogenic60
Likely Pathogenic3
VUS46
Likely Benign4
Benign4
60
Pathogenic
3
Likely Pathogenic
46
VUS
4
Likely Benign
4
Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
60
0
60
Likely Pathogenic
0
0
3
0
3
VUS
0
41
5
0
46
Likely Benign
0
3
1
0
4
Benign
0
2
1
1
4
Total046701117

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

View in ClinVar →

Protein Context — Lollipop Plot

FKBP4 · protein map & ClinVar variants

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

Clinical Literature
Open Research Assistant →