RPA1

Chr 17AD

replication protein A1

This gene encodes the largest subunit of replication protein A (RPA), which binds and stabilizes single-stranded DNA during replication and DNA repair, recruits DNA damage response proteins, and activates the ATR kinase pathway. Mutations cause pulmonary fibrosis and/or bone marrow failure syndrome with telomere dysfunction, inherited in an autosomal dominant pattern. The gene shows significant constraint against loss-of-function variants (LOEUF 0.43), reflecting its essential role in DNA metabolism.

OMIMResearchSummary from RefSeq, OMIM, UniProt
MultiplemechanismADLOEUF 0.431 OMIM phenotype
Clinical SummaryRPA1
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Gene-Disease Validity (ClinGen)
dyskeratosis congenita and related telomere biology disorder · ADLimited

Limited evidence — not for standalone diagnostic reporting

Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.25) despite low pLI — interpret in context.
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Clinical Trials
2 active or recruiting trials — potential therapeutic options may be available
Some data sources returned errors (1)

ncbi: Error: NCBI fetch failed: 429 https://eutils.ncbi.nlm.nih.gov/entrez/eutils/esearch.fcgi

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.43LOEUF
pLI 0.088
Z-score 4.34
OE 0.25 (0.160.43)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
1.22Z-score
OE missense 0.82 (0.740.90)
292 obs / 356.7 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.25 (0.160.43)
00.351.4
Missense OE0.82 (0.740.90)
00.61.4
Synonymous OE1.01
01.21.6
LoF obs/exp: 10 / 39.4Missense obs/exp: 292 / 356.7Syn Z: -0.12
DN
0.6842th %ile
GOF
0.4874th %ile
LOF
0.3745th %ile

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

DNprediction above median
GOF1 literature citation

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.

Literature Evidence

GOFGain-of-function mutations in RPA1 cause a syndrome with short telomeres and somatic genetic rescue.PMID:34767620

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

RPA1 · 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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