FOLR1

Chr 11AR

folate receptor alpha

Also known as: FBP, FOLR, FR-alpha, FRalpha, NCFTD

The encoded protein binds folate and reduced folic acid derivatives and mediates cellular uptake of 5-methyltetrahydrofolate through receptor-mediated endocytosis. Mutations cause neurodegeneration due to cerebral folate transport deficiency, inherited in an autosomal recessive pattern. This condition impairs folate transport across the blood-brain barrier, leading to progressive neurological deterioration despite normal systemic folate levels.

OMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismARLOEUF 0.651 OMIM phenotype
Clinical SummaryFOLR1
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Gene-Disease Validity (ClinGen)
neurodegenerative syndrome due to cerebral folate transport deficiency · ARDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.29) despite low pLI — interpret in context.
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Clinical Trials
4 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.65LOEUF
pLI 0.126
Z-score 2.48
OE 0.29 (0.140.65)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
0.67Z-score
OE missense 0.84 (0.720.98)
117 obs / 139.4 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.29 (0.140.65)
00.351.4
Missense OE0.84 (0.720.98)
00.61.4
Synonymous OE0.88
01.21.6
LoF obs/exp: 4 / 14.0Missense obs/exp: 117 / 139.4Syn Z: 0.71

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

FOLR1 · protein map & ClinVar variants

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

3D Protein StructureAlphaFold
Clinical Literature
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