RAG1

Chr 11AR

recombination activating 1

Also known as: RAG-1, RNF74

RAG1 encodes the catalytic component of the RAG complex that mediates DNA cleavage during V(D)J recombination, the process that assembles diverse immunoglobulin and T-cell receptor genes in developing B and T lymphocytes. Autosomal recessive mutations cause severe combined immunodeficiency with absent B cells, Omenn syndrome, combined immune defects with granulomas, and T-cell lymphopenia with autoimmunity, affecting both cellular and humoral immunity. The gene is highly intolerant to loss-of-function variants (pLI near 1.0), reflecting its essential role in adaptive immune system development.

Summary from RefSeq, OMIM, UniProt
Research Assistant →

Primary Disease Associations & Inheritance

Alpha/beta T-cell lymphopenia with gamma/delta T-cell expansion, severe cytomegalovirus infection, and autoimmunityMIM #609889
Combined cellular and humoral immune defects with granulomasMIM #233650
AR
Omenn syndromeMIM #603554
AR
Severe combined immunodeficiency, B cell-negativeMIM #601457
AR
1
Active trials
133
Pubs (1 yr)
149
P/LP submissions
19%
P/LP missense
0.74
LOEUF
LOF
Mechanism· G2P
Clinical SummaryRAG1
🧬
Gene-Disease Validity (ClinGen)
recombinase activating gene 1 deficiency · ARDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
97 unique Pathogenic / Likely Pathogenic· 157 VUS of 497 total submissions
💊
Clinical Trials
1 active or recruiting trial — potential therapeutic options may be available
📖
GeneReview available — RAG1
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.74LOEUF
pLI 0.000
Z-score 2.60
OE 0.45 (0.290.74)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
0.58Z-score
OE missense 0.93 (0.861.00)
504 obs / 542.1 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.45 (0.290.74)
00.351.4
Missense OE0.93 (0.861.00)
00.61.4
Synonymous OE0.99
01.21.6
LoF obs/exp: 12 / 26.4Missense obs/exp: 504 / 542.1Syn Z: 0.08
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveRAG1-related Omenn syndromeLOFAR

Predictions shown for reference only — model trained on dominant genes, not applicable to AR conditions.

DN
0.6745th %ile
GOF
0.3986th %ile
LOF
0.52top 25%

The Badonyi & Marsh prediction model was trained exclusively on dominant disease genes. Predictions are not reliable for genes with autosomal recessive inheritance and are shown at reduced opacity for reference only.

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

497 submitted variants in ClinVar

Classification Summary

Pathogenic51
Likely Pathogenic46
VUS157
Likely Benign217
Benign11
Conflicting11
51
Pathogenic
46
Likely Pathogenic
157
VUS
217
Likely Benign
11
Benign
11
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
27
6
18
0
51
Likely Pathogenic
22
12
12
0
46
VUS
0
133
23
1
157
Likely Benign
0
7
1
209
217
Benign
0
3
8
0
11
Conflicting
11
Total4916162210493

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

View in ClinVar →

Protein Context — Lollipop Plot

RAG1 · protein map & ClinVar variants

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

Clinical Literature
Open Research Assistant →