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GeneE
50 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
β“˜GeneE is for informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment.
SF3B1
splicing factor 3b subunit 1
Chromosome 2 Β· 2q33.1
NCBI Gene: 23451Ensembl: ENSG00000115524.17HGNC: HGNC:10768UniProt: B4DGZ4
532PubMed Papers
20Diseases
0Drugs
4Pathogenic Variants
FUNCTIONAL ROLE
Highly ConstrainedHub Gene
RESEARCH IMPACT
Highly StudiedTrending
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
nuclear specknucleusRNA bindingpositive regulation of transcription by RNA polymerase IIIchronic lymphocytic leukemiamyelodysplastic syndromelymphoid leukemiabreast adenocarcinoma
✦AI Summary

SF3B1 is a core component of the U2 small nuclear ribonucleoprotein (snRNP) complex within the spliceosome, where it plays a critical role in pre-mRNA splicing by mediating recognition of the intron branch point sequence 1. The protein functions in both major U2-type and minor U12-type spliceosomes, facilitating the removal of introns from pre-mRNA transcripts. Mechanistically, SF3B1 participates in early spliceosome assembly and promotes selection of the branch-site adenosine required for splicing catalysis 1. Cancer-associated SF3B1 mutations, particularly hotspot mutations, cause aberrant 3' splice site selection by utilizing alternative branch point sequences, leading to cryptic splicing events 1. These mutations also impair RNA polymerase II elongation and reduce chr2 accessibility at gene promoters 2. Clinically, SF3B1 mutations are highly significant in hematological malignancies, occurring in approximately 80% of patients with myelodysplastic syndrome with ring sideroblasts (MDS-RS) 3. SF3B1-mutant MDS represents a distinct disease entity characterized by ring sideroblasts, ineffective erythropoiesis, and relatively favorable prognosis 4. The mutations serve as biomarkers for patient stratification and therapeutic decision-making, with SF3B1-mutant patients showing potential responsiveness to targeted therapies like luspatercept 43.

Sources cited
1
SF3B1 mediates branch point sequence recognition and cancer mutations cause aberrant splicing through alternative branch points
PMID: 26565915
2
SF3B1 mutations occur in β‰₯80% of MDS-RS patients and correlate with ring sideroblasts
PMID: 33428785
3
SF3B1-mutant MDS represents a distinct entity with specific diagnostic criteria and favorable prognosis
PMID: 32347921
4
SF3B1 mutations reduce RNA polymerase II elongation and alter chromatin accessibility at promoters
PMID: 38521065
⚠Limited data available β€” This gene has 4 indexed publications. Summary and analysis may be incomplete.
Disease Associationsβ“˜20
chronic lymphocytic leukemiaOpen Targets
0.73Strong
myelodysplastic syndromeOpen Targets
0.64Moderate
lymphoid leukemiaOpen Targets
0.56Moderate
breast adenocarcinomaOpen Targets
0.54Moderate
myeloproliferative disorderOpen Targets
0.53Moderate
neurodegenerative diseaseOpen Targets
0.50Moderate
leukemiaOpen Targets
0.50Moderate
Uveal MelanomaOpen Targets
0.47Moderate
Alzheimer diseaseOpen Targets
0.46Moderate
lysosomal storage diseaseOpen Targets
0.46Moderate
multiple sclerosisOpen Targets
0.46Moderate
Parkinson diseaseOpen Targets
0.46Moderate
acute myeloid leukemiaOpen Targets
0.44Moderate
anemia (phenotype)Open Targets
0.42Moderate
chronic myelogenous leukemiaOpen Targets
0.41Moderate
neoplasmOpen Targets
0.41Moderate
hematologic diseaseOpen Targets
0.39Weak
Richter syndromeOpen Targets
0.37Weak
rectum malignant melanomaOpen Targets
0.37Weak
vulvar melanomaOpen Targets
0.37Weak
Pathogenic Variants4
NM_012433.4(SF3B1):c.1998G>T (p.Lys666Asn)Pathogenic
Myelodysplastic syndrome progressed to acute myeloid leukemia|Myelodysplastic syndrome
β˜…β˜…β˜†β˜†2021β†’ Residue 666
NM_012433.4(SF3B1):c.1866G>C (p.Glu622Asp)Likely pathogenic
Myelodysplastic syndrome
β˜…β˜†β˜†β˜†2025β†’ Residue 622
NM_012433.4(SF3B1):c.1998G>C (p.Lys666Asn)Likely pathogenic
Acute myeloid leukemia|Myelodysplastic syndrome
β˜…β˜†β˜†β˜†2023β†’ Residue 666
NM_012433.4(SF3B1):c.1874G>A (p.Arg625His)Likely pathogenic
See cases
β˜…β˜†β˜†β˜†2021β†’ Residue 625
View on ClinVar β†—
Related Genes
DHX15Protein interaction100%IKProtein interaction100%MFAP1Protein interaction100%SRSF1Protein interaction100%SRSF5Protein interaction100%SNRPAProtein interaction100%
Tissue Expression6 tissues
Bone Marrow
100%
Ovary
61%
Lung
48%
Heart
43%
Liver
38%
Brain
27%
Gene Interaction Network
Click a node to explore
SF3B1DHX15IKMFAP1SRSF1SRSF5SNRPA
PROTEIN STRUCTURE
Preparing viewer…
PDB4OZ1 Β· 1.74 Γ… Β· X-ray
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
0.10Highly Constrained
pLIβ“˜
1.00Intolerant
Observed/Expected LoF0.05 [0.03–0.10]
RankingsWhere SF3B1 stands among ~20K protein-coding genes
  • #476of 20,598
    Most Researched532 Β· top 5%
  • #3,664of 5,498
    Most Pathogenic Variants4
  • #58of 17,882
    Most Constrained (LOEUF)0.10 Β· top 1%
Genes detectedSF3B1
Sources retrieved50 papers
Response timeβ€”
πŸ“„ Sources
50β–Ό
1
Myelodysplastic Syndrome Updated.
PMID: 30041243
Pathobiology Β· 2019
1.00
2
Myelodysplastic syndromes with ring sideroblasts (MDS-RS) and MDS/myeloproliferative neoplasm with RS and thrombocytosis (MDS/MPN-RS-T) - "2021 update on diagnosis, risk-stratification, and management".
PMID: 33428785
Am J Hematol Β· 2021
0.90
3
GPATCH8 modulates mutant SF3B1 mis-splicing and pathogenicity in hematologic malignancies.
PMID: 38688280
Mol Cell Β· 2024
0.88
4
SF3B1 mutation and ATM deletion codrive leukemogenesis via centromeric R-loop dysregulation.
PMID: 37463047
J Clin Invest Β· 2023
0.86
5
Distinct and convergent effects of
PMID: 41055979
Proc Natl Acad Sci U S A Β· 2025
0.84