KIT

Chr 4ADIsolated cases

KIT proto-oncogene, receptor tyrosine kinase

Also known as: C-Kit, CD117, MASTC, PBT, SCFR

This gene encodes a receptor tyrosine kinase. This gene was initially identified as a homolog of the feline sarcoma viral oncogene v-kit and is often referred to as proto-oncogene c-Kit. The canonical form of this glycosylated transmembrane protein has an N-terminal extracellular region with five immunoglobulin-like domains, a transmembrane region, and an intracellular tyrosine kinase domain at the C-terminus. Upon activation by its cytokine ligand, stem cell factor (SCF), this protein phosphorylates multiple intracellular proteins that play a role in in the proliferation, differentiation, migration and apoptosis of many cell types and thereby plays an important role in hematopoiesis, stem cell maintenance, gametogenesis, melanogenesis, and in mast cell development, migration and function. This protein can be a membrane-bound or soluble protein. Mutations in this gene are associated with gastrointestinal stromal tumors, mast cell disease, acute myelogenous leukemia, and piebaldism. Multiple transcript variants encoding different isoforms have been found for this gene. [provided by RefSeq, May 2020]

Primary Disease Associations & Inheritance

Gastrointestinal stromal tumor, familialMIM #606764
ADIsolated cases
Germ cell tumors, somaticMIM #273300
Leukemia, acute myeloid, somaticMIM #601626
Mastocytosis, cutaneousMIM #154800
AD
Mastocytosis, systemic, somaticMIM #154800
PiebaldismMIM #172800
AD
UniProtPiebald trait
UniProtTesticular germ cell tumor
12
Active trials
0
Pathogenic / LP
0
ClinVar variants
6
Pubs (1 yr)
2.6
Missense Z
0.30
LOEUF· LoF intolerant
Clinical SummaryKIT
🧬
Gene-Disease Validity (ClinGen)
gastrointestinal stromal tumor · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 0.98). One damaged copy is likely sufficient to cause disease.
💊
Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint?LOEUF (Loss-of-function Observed/Expected Upper bound Fraction) is the upper bound of the 90% CI for LoF OE — the preferred gnomAD v4 metric. Lower = more intolerant to LoF. LOEUF < 0.35 = highly constrained.
0.30LOEUF
pLI 0.981
Z-score 5.30
OE 0.17 (0.100.30)
Highly constrained

Highly LoF-intolerant (top ~10% of genes)

Missense Constraint?Missense Z-score: standard deviations fewer missense variants observed vs. expected. Z > 3.09 (p < 0.001) = gene does not tolerate missense variation. OE missense < 0.6 is also considered constrained.
2.58Z-score
OE missense 0.69 (0.630.75)
375 obs / 544.4 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios?Shaded band = 90% confidence interval. Vertical tick = point estimate. Grey threshold line = gnomAD constraint cutoff for that variant class.
LoF OE?Ratio of observed to expected LoF variants. Upper CI bound (LOEUF) ≤ 0.35 = strong LoF constraint signal.0.17 (0.100.30)
00.351.4
Missense OE?Ratio of observed to expected missense variants. OE ≤ 0.6 = fewer missense variants than expected by chance.0.69 (0.630.75)
00.61.4
Synonymous OE?Control metric — synonymous variants are largely neutral and expected near OE = 1.0. Significant deviation may indicate annotation issues.1.09
01.21.6
LoF obs/exp: 8 / 47.4Missense obs/exp: 375 / 544.4Syn Z: -1.00
DN
0.5673th %ile
GOF
0.6833th %ile
LOF
0.51top 25%

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

GOFprediction above median · 1 literature citation
LOFLOEUF 0.30
DN1 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

DNThis is consistent with a possible """"""""""""""""""""""""""""""""""""""""""""""""""""""""""""""""dominant negative"""""""""""""""""""""""""""""""""""""""""""""""""""""""""""""""" effect of missense c-kit polypeptides on the function of the dimeric receptor.PMID:1370874
GOFGain-of-function mutations of c-kit in human gastrointestinal stromal tumorsPMID:9438854

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

KIT · protein map & ClinVar variants

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

Gene2Phenotype Curations

KIT-related piebaldism

definitive
ADLoss Of FunctionAbsent Gene Product, Altered Gene Product Structure
Dev. DisordersEyeSkin
G2P ↗

KIT-related familial gastro-intestinal stromal tumours

definitive
ADUndeterminedUncertain
SkinCancer
G2P ↗

Gene2Phenotype curations · DECIPHER consortium patient cohort (public variants) · deciphergenomics.org

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Acute Myeloid Leukemia With T(8;21)(Q22;Q22)Acute Myeloid Leukemia With T(16;16)(P13;Q22)KIT Gene Mutation

A New Treatment of Newly Diagnosed KIT Mutation CBF-Acute Myeloid Leukemia

RECRUITING
NCT07028073Phase PHASE1, PHASE2The First Affiliated Hospital of Soochow UniversityStarted 2025-05-15
Group A (FIT): Avapritinib + IA regimenGroup B (UNFIT): Avapritinib + VA regimen
Cognitive DisordersMuscular Disorders, Atrophic

Dietary Strategy to Tackle Cognitive and Locomotor Abilities in Early Elderly Subjects

RECRUITING
NCT06871384Phase NAUniversity Rovira i VirgiliStarted 2025-03-26
Nonalcoholic red wine group (Intervention group)Drinking water group (Control group)
Differentiated Thyroid CarcinomaMedullary Thyroid Carcinoma

Spanish Study for Molecular Characterization of Thyroid Carcinoma

ACTIVE NOT RECRUITING
NCT04970134Grupo Español de Tratamiento de Tumores de Cabeza y CuelloStarted 2021-06-14
Immunohistochemistry (IHC)Fluorescence In-Situ Hybridization (FISH)Oncomine Focus Assay Platform with 52 genes
Primary Ciliary Dyskinesia

A Study Providing Genetic Testing to Find Those Who May Have Primary Ciliary Dyskinesia for Potential Clinical Trials

ACTIVE NOT RECRUITING
NCT06172374ReCode TherapeuticsStarted 2023-08-31
Sano Genetics Testing Kit
Spontaneous Coronary Artery DissectionSCAD

Genetic Investigations in Spontaneous Coronary Artery Dissection (SCAD)

ENROLLING BY INVITATION
NCT01427179Mayo ClinicStarted 2011-05
Genitourinary Syndrome of Menopause

Vaginal CO2 Laser Therapy for Genitourinary Syndrome in Breast Cancer Survivors

RECRUITING
NCT06007027Phase NAUniversity of AarhusStarted 2024-12-01
SmartXIDE2V2LR, Monalisa Touch, DEKA, Florence, Italy
Depression

Intern Health Study

ENROLLING BY INVITATION
NCT01809080University of MichiganStarted 2007-05
Periodontal Disease (PD)Cardio Vascular DiseaseOral-Systemic Link

PMPR and Chlorhexidine on Periodontal Disease and Vascular Function

NOT YET RECRUITING
NCT07311512Phase NAMahdi MutaharStarted 2026-01-23
Chlorhexidine (0.2%) mouthwashPlacebo mouthwash
Hematologic CancerColon CancerBreast Cancer

Precision Physical Exercise for Personalized Onco-Hematology.

NOT YET RECRUITING
NCT07286539Phase NAUniversity of SalamancaStarted 2026-04-01
Supervised InterventionHome-based Intervention
Adverse Drug Reaction

The Texas Interprofessional Pharmacogenomics (IPGx)

RECRUITING
NCT06219720Texas A&M UniversityStarted 2021-12-15
Colorectal Cancer (CRC)MicrobiomeEarly Onset Colorectal Cancer

CARE-CRC: Microbiome Insights and Correlations for Risk and Outcomes in Colorectal Cancer

NOT YET RECRUITING
NCT06734156Gulbenkian Institute for Molecular MedicineStarted 2025-12-02
No intervention: observational study
Kidney Neoplasms

MRI Functional Imaging Characteristics and Fat Quantification of CT-fat-free Renal Neoplasms: Relationships With Histological Classifications and Molecular Markers

ACTIVE NOT RECRUITING
NCT06126159Phase NAChang Gung Memorial HospitalStarted 2019-02-11
multiparametric and fat-detection magnetic resonance imaging (MRI)
Clinical Literature
Landmark / reviewRecent case evidence
Full-Text Mentions
NLP-detected gene mentions in article bodies · via PubTator3
PubTator3
Biodegradable dental kits.
Mathew MG et al.·Br Dent J
2024
KIT
Wu TM et al.·Zhonghua Xue Ye Xue Za Zhi
2021
What's In Your Kit? Mental Health Technology Kits for Depression Self-Management.
Burgess ER et al.·Proc SIGCHI Conf Hum Factor Comput Syst
2025
Top 5 full-text resultsSearch PubTator3 ↗
Key Publications
Landmark & review papers · by relevance
PubMed
Clinical exome sequencing findings in 1589 patients.
Gorukmez O et al.·Am J Med Genet A
2023Cohort
Succinate Dehydrogenase-Deficient Gastrointestinal Stromal Tumors.
Ibrahim A et al.·Arch Pathol Lab Med
2020Review
Systemic Mastocytosis: State of the Art.
Farmer I et al.·Curr Hematol Malig Rep
2024Review
Top 5 results · since 2015Search PubMed ↗