HCFC1

Chr XXLR

host cell factor C1

Also known as: CFF, HCF, HCF-1, HCF1, HFC1, MAHCX, MRX3, PPP1R89

The protein functions as a nuclear coactivator that regulates cell cycle control and transcriptional regulation, containing Kelch repeats and HCF repeats that undergo proteolytic cleavage to form functional N- and C-terminal chains. Loss-of-function mutations cause methylmalonic aciduria and homocysteinemia (cblX type), an X-linked recessive disorder affecting vitamin B12 metabolism. The gene is highly intolerant to loss-of-function variation, indicating that HCFC1 haploinsufficiency disrupts normal cellular metabolism.

Summary from RefSeq, OMIM, UniProt, Mechanism

Primary Disease Associations & Inheritance

Methylmalonic aciduria and homocysteinemia, cblX typeMIM #309541
XLR
0
Active trials
13
Pubs (1 yr)
0
P/LP submissions
P/LP missense
0.06
LOEUF· LoF intol.
LOF
Mechanism· G2P
Clinical SummaryHCFC1
🧬
Gene-Disease Validity (ClinGen)
X-linked intellectual disability · XLDefinitive

Definitive — sufficient evidence for diagnostic panels

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.

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Dual constrained — LoF & missense intolerant
LoF Constraint
0.06LOEUF
pLI 1.000
Z-score 6.50
OE 0.00 (0.000.06)
Highly constrained

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

Missense Constraint
5.63Z-score
OE missense 0.47 (0.430.51)
419 obs / 892.7 exp
Constrained

Extremely missense-constrained (top ~0.01%)

Observed / Expected Ratios
LoF OE0.00 (0.000.06)
00.351.4
Missense OE0.47 (0.430.51)
00.61.4
Synonymous OE1.13
01.21.6
LoF obs/exp: 0 / 49.2Missense obs/exp: 419 / 892.7Syn Z: -2.08
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveHCFC1-related cobalamin disorderLOFXLR
definitiveHCFC1-related intellectual developmental disorderOTHERXLR
DN
0.18100th %ile
GOF
0.1799th %ile
LOF
0.87top 5%

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

LOFprediction above median · LOEUF 0.06

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

HCFC1 · 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.

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Clinical Literature
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