RAF1

Chr 3AD

Raf-1 proto-oncogene, serine/threonine kinase

Also known as: CMD1NN, CRAF, NS5, Raf-1, c-Raf

The protein is a MAP kinase kinase kinase (MAP3K) that functions downstream of Ras GTPases and phosphorylates MEK1 and MEK2 to activate the ERK1/2 pathway, which controls gene expression involved in cell division, apoptosis, differentiation, and migration. Loss-of-function mutations cause autosomal dominant Noonan syndrome 5, LEOPARD syndrome 2, and dilated cardiomyopathy through disruption of this critical signaling cascade. The gene shows strong intolerance to loss-of-function variants, consistent with its essential role in development and cellular regulation.

OMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
GOFmechanismADLOEUF 0.353 OMIM phenotypes
VCEP Guidelines: RASopathyReleased
View SpecificationsClinGen Panel
Clinical SummaryRAF1
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Gene-Disease Validity (ClinGen)
Costello syndrome · ADDisputed

Disputed — evidence questions this relationship

4 total gene-disease associations curated

Population Constraint (gnomAD)
Moderately constrained gene (pLI 0.85) — some intolerance to loss-of-function variants.
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Clinical Trials
5 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
0.35LOEUF
pLI 0.853
Z-score 4.58
OE 0.19 (0.110.35)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
2.46Z-score
OE missense 0.64 (0.570.71)
234 obs / 366.7 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.19 (0.110.35)
00.351.4
Missense OE0.64 (0.570.71)
00.61.4
Synonymous OE0.99
01.21.6
LoF obs/exp: 7 / 37.1Missense obs/exp: 234 / 366.7Syn Z: 0.06
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveRAF1-related Noonan syndromeGOFAD
DN
0.5082th %ile
GOF
0.5170th %ile
LOF
0.72top 10%

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

LOFprediction above median · LOEUF 0.35
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 the PTPN11, KRAS, SOS1, and RAF1 genes that are components of the RAS/MEPK signaling pathway are identified in about 70-85% of individuals with Noonan syndrome.PMID:18348260

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

RAF1 · protein map & ClinVar variants

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

3D Protein StructureAlphaFold
Clinical Literature
Open Research Assistant →
Key Publications
Landmark & review papers · by relevance
PubMed
Top 5 results · since 2015Search PubMed ↗