PTEN

Chr 10AD

phosphatase and tensin homolog

Also known as: 10q23del, BZS, CWS1, DEC, GLM2, MHAM, MMAC1, PTEN1

This gene encodes a dual-specificity phosphatase that functions primarily as a lipid phosphatase, dephosphorylating PIP3 to negatively regulate the PI3K-AKT signaling pathway critical for cell growth, survival, and neuronal development. Germline mutations cause autosomal dominant conditions including Cowden syndrome, macrocephaly/autism syndrome, and Lhermitte-Duclos disease, predominantly through loss-of-function resulting in excessive PI3K-AKT pathway activation. The protein plays essential roles in neuronal migration, dendritic development, synaptic plasticity, and regulation of cell size and proliferation.

OMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
LOFmechanismADLOEUF 0.516 OMIM phenotypes
VCEP Guidelines: PTENReleased
View SpecificationsClinGen Panel
Clinical SummaryPTEN
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Gene-Disease Validity (ClinGen)
PTEN hamartoma tumor syndrome · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.24) despite low pLI — interpret in context.
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Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Missense constrained — critical functional residues
LoF Constraint
0.51LOEUF
pLI 0.257
Z-score 3.20
OE 0.24 (0.130.51)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
3.49Z-score
OE missense 0.33 (0.270.41)
72 obs / 216.4 exp
Constrained

Highly missense-constrained (top ~0.1%)

Observed / Expected Ratios
LoF OE0.24 (0.130.51)
00.351.4
Missense OE0.33 (0.270.41)
00.61.4
Synonymous OE1.02
01.21.6
LoF obs/exp: 5 / 20.8Missense obs/exp: 72 / 216.4Syn Z: -0.12
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitivePTEN-related hamartoma tumour syndrome (Cowden syndrome)LOFAD
definitivePTEN-related Proteus syndromeLOFAD
definitivePTEN-related Lhermitte-Duclos diseaseLOFAD
Mechanism Note (expert annotation)
LOF

Tumor suppressor phosphatase. LOF (haploinsufficiency) causes PHTS (Cowden, Bannayan-Riley-Ruvalcaba). This is a classic tumor suppressor with LOF mechanism; the DN prediction does not reflect the established biology.

References:PMID:9140397
DN
0.6938th %ile
GOF
0.6542th %ile
LOF
0.3744th %ile

This gene has evidence for multiple mechanisms of pathogenicity (dominant-negative, gain-of-function and loss-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 · 1 literature citation
GOFprediction above median
LOF1 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

DNCancer-associated PTEN mutants act in a dominant-negative manner to suppress PTEN protein function.PMID:24766807
LOFA clinical scoring system for selection of patients for PTEN mutation testing is proposed on the basis of a prospective study of 3042 probandsPMID:21194675

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

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

PTEN · protein map & ClinVar variants

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

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Breast CancerGenetic Susceptibility

ScreenIng of Genetic Susceptibility Genes for Breast Cancer Patients in CHinese communiTies

RECRUITING
NCT04265937Fudan UniversityStarted 2019-07-01
Prostate Cancer

Hypofractionated, Dose Escalation Radiotherapy for High Risk Adenocarcinoma of the Prostate

ACTIVE NOT RECRUITING
NCT01444820Phase PHASE3Sir Mortimer B. Davis - Jewish General HospitalStarted 2012-01
hypofractionationconventional
Lymphoma, Non-HodgkinMultiple MyelomaAdvanced Solid Tumors

Canadian Profiling and Targeted Agent Utilization Trial (CAPTUR)

RECRUITING
NCT03297606Phase PHASE2Canadian Cancer Trials GroupStarted 2018-03-23
OlaparibDasatinibNivolumab plus Ipilimumab
Parkinson DiseaseNervous System DisorderNeurodegenerative Diseases

Molecular and Functional Imaging in Monogenic PD.

RECRUITING
NCT05518617University of ExeterStarted 2022-07-01
Positron Emission Tomography (PET) scan using DASB tracer
Cowden's DiseaseCowden's SyndromeLhermitte-Duclos Disease

Double-Blind Trial of Everolimus for Improving Social Abilities in PTEN Germline Mutations

NOT YET RECRUITING
NCT07218575Phase PHASE2, PHASE3Boston Children's HospitalStarted 2026-04
Everolimus blinded oral capsulesPlacebo
Polycythemia VeraEssential ThrombocythaemiaMyelofibrosis

Prevalence Of Germline Gene Mutations In Patients With Myeloproliferative Neoplasms With Family History

NOT YET RECRUITING
NCT06923670Phase NAFondazione Policlinico Universitario Agostino Gemelli IRCCSStarted 2025-05-21
NGS testingNGS analysis for mutations in genes involved in familial predisposition to hematological malignancies
Prostate Cancer

Detection of Minimal Residual Disease Post-prostatectomy

RECRUITING
NCT07334275Radboud University Medical CenterStarted 2025-09-16
Prostate Cancer

Comparative Study of Radiotherapy Treatments to Treat High Risk Prostate Cancer Patients

ACTIVE NOT RECRUITING
NCT02303327Phase PHASE3Sir Mortimer B. Davis - Jewish General HospitalStarted 2015-01
EBRT + HDR brachytherapy boostHypofractionated Dose Escalation RadiotherapyAndrogen deprivation therapy
Stage IB Lung Non-Small Cell Carcinoma AJCC v7Stage II Lung Non-Small Cell Cancer AJCC v7Stage IIA Lung Cancer AJCC v8

Genetic Testing in Screening Patients With Stage IB-IIIA Non-small Cell Lung Cancer That Has Been or Will Be Removed by Surgery (The ALCHEMIST Screening Trial)

ACTIVE NOT RECRUITING
NCT02194738Phase NANational Cancer Institute (NCI)Started 2014-09-26
Biospecimen CollectionCarboplatinCisplatin
BRCA1 MutationPOLD1 Gene MutationCDKN2A Mutation

An Intervention to Increase Genetic Testing in Families Who May Share a Gene Mutation Related to Cancer Risk and An Intervention to Help Patients and Their Primary Care Providers Stay Up-to-date About Uncertain Genetic Test Results

RECRUITING
NCT05420064Phase NAMemorial Sloan Kettering Cancer CenterStarted 2022-12-01
Intervention Arm At-risk Relative/ARR ContactsMyGene PortalStandard of Care
Lynch SyndromeLi Fraumeni SyndromePTEN Hamartoma Syndrome

Video Capsule Examination in Patients With Lynch Syndrome

RECRUITING
NCT06712095Phase NARoyal Marsden NHS Foundation TrustStarted 2024-03-04
Video capsule investigation
Acute LeukemiaAdenomatous PolyposisAdrenocortical Carcinoma

Familial Investigations of Childhood Cancer Predisposition

RECRUITING
NCT03050268St. Jude Children's Research HospitalStarted 2017-04-06
Clinical Literature
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