Genetic Information

Gene & Transcript Details

Gene
PTEN
Transcript
NM_000314.8 MANE Select
Total Exons
Reference Sequence
NC_000010.10
Alternative Transcripts
IDStatusDetails
NM_000314.8 MANE Select 8515 nt | 846–2057
NM_000314.7 RefSeq Select 8514 nt | 845–2056
NM_000314.5 Alternative 8719 nt | 1032–2243
NM_000314.4 Alternative 5572 nt | 1032–2243
NM_000314.3 Alternative 3416 nt | 1032–2243
NM_000314.6 Alternative 8718 nt | 1032–2243

Variant Details

HGVS Notation
NM_000314.8:c.37A>T
Protein Change
K13*
Location
Exon 1 (Exon 1 of )
1
5'Exon Structure3'
Functional Consequence
Loss of Function
Alternate Identifiers

Clinical & Population Data

Population Frequency

gnomAD
Global Frequency
0.0 in 100,000
Extremely Rare
ACMG Criteria Applied PM2
This variant is absent or extremely rare in population databases (PM2 criteria applies).

ClinVar

Open
Classification
Pathogenic
1 publications
Publications List
PMID: 9467011

For these reasons, this variant has been classified as Pathogenic. While this particular variant has not been reported in the literature, loss-of-function variants in PTEN are known to be pathogenic (PMID: 9467011, 21194675). This sequence change creates a premature translational stop signal at codon 13 (p.Lys13*) of the PTEN gene. It is expected to result in an absent or disrupted protein product.

Clinical Statement

"This variant has been reported in ClinVar as Pathogenic (2 clinical laboratories)."

COSMIC Somatic Evidence

Open
COSMIC ID
COSM86063
Recurrence
6 occurrences
PM1 Criteria
Not Applied
COSMIC Database Preview
COSMIC Preview
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Functional Impact & Domains

Functional Domain

Hotspot Status
Not a hotspot
Domain Summary
This variant is not located in a mutational hotspot or critical domain.
Related Variants in This Domain
No evidence of other pathogenic variants at this position in gene PTEN.

Functional Studies & Therapeutic Relevance

Functional Summary

The PTEN K13* variant is a truncating mutation that results in a premature stop codon at amino acid 13, leading to a loss of PTEN phosphatase function. Functional studies indicate that this truncation impairs the protein's ability to negatively regulate the PI3K/AKT pathway and increases genomic instability due to disrupted chromosomal centromere association. Additionally, yeast assays demonstrate reduced phosphatase activity, supporting the conclusion that the PTEN K13* variant is functionally damaging and likely oncogenic.

Database Previews
OncoKB
OncoKB Preview
JAX-CKB
JAX-CKB Preview

Click on previews to view full database entries. External databases may require institutional access.

Computational Analysis

Pathogenicity Predictions

SpliceAI
Predictor Consensus
Mixed/VUS
PP3 Applied
No
REVEL Score
0.0
Threshold: ≥0.75 = PP3 applied

SpliceAI Scores

Window: ±500bp
Effect Type Score Position
- Acceptor Loss (AL) 0.0 131 bp
- Donor Loss (DL) 0.05 42 bp
+ Acceptor Gain (AG) 0.08 -52 bp
+ Donor Gain (DG) 0.06 131 bp
High impact (≥0.5) Medium impact (0.2-0.49) Low impact (<0.2)

VCEP Guidelines

Applied ACMG/AMP Criteria (VCEP Specific)

Filter Criteria:
PVS1

PVS1 (Very Strong)

According to VCEP guidelines: "Null Variant (nonsense, fs, start codon, splicing +1/2, startgain, single or multi-exon deletion) in a gene where LOF is a known mechanism of disease". The evidence shows this variant is a nonsense change (K13*) introducing a premature stop codon in PTEN, not in the last exon, leading to loss of function. Therefore, this criterion is applied at Very Strong strength because it meets the VCEP PVS1 decision tree for PTEN.

PS1

PS1 (Not Applied)

According to VCEP guidelines: "Same amino acid change as a previously established pathogenic variant regardless of nucleotide change". There is no known variant causing the same amino acid change at codon 13. Therefore, this criterion is not applied.

PS2

PS2 (Not Applied)

According to VCEP guidelines: "Very Strong: two proven de novo or four assumed de novo, etc., in a patient with the disease and no family history". No de novo data are available for this variant. Therefore, this criterion is not applied.

PS3

PS3 (Moderate)

According to PTEN Pre-processing: "Applied PS3_Moderate evidence added based on high-confidence functional score (-3.9912) < threshold (-1.11)". The evidence shows phosphatase activity of –3.9912, which is below the VCEP PTEN threshold of –1.11, indicating damaging effect. Therefore, this criterion is applied at Moderate strength.

PS4

PS4 (Not Applied)

According to VCEP guidelines: "Moderate: probands with specificity score 2–3.5 or increased prevalence in cases versus controls". No case/control or proband specificity data are available. Therefore, this criterion is not applied.

PM1

PM1 (Not Applied)

According to VCEP guidelines: "Located in a mutational hot spot and/or critical and well-established functional domain (residues 90–94, 123–130, 166–168)". Codon 13 is outside these PTEN functional domains. Therefore, this criterion is not applied.

PM2

PM2 (Supporting)

According to VCEP guidelines: "Supporting: Absent in population Databases present at <0.00001 (0.001%) allele frequency in gnomAD". The variant is not found in gnomAD or other large population datasets. Therefore, this criterion is applied at Supporting strength.

PM3

PM3 (Not Applied)

According to VCEP guidelines: "Detected in trans with a pathogenic variant in recessive disorders". PTEN disease is dominant and no trans observations are relevant. Therefore, this criterion is not applied.

PM4

PM4 (Not Applied)

According to VCEP guidelines: "Protein length changes due to in-frame indels or stop-loss variants". This is a nonsense variant, not an in-frame alteration or stop-loss. Therefore, this criterion is not applied.

PM5

PM5 (Not Applied)

According to VCEP guidelines: "Missense change at an amino acid residue where a different missense change determined to be pathogenic". This is a nonsense variant, not missense. Therefore, this criterion is not applied.

PM6

PM6 (Not Applied)

According to VCEP guidelines: "Assumed de novo without paternity/maternity confirmation". No de novo data exist. Therefore, this criterion is not applied.

PP1

PP1 (Not Applied)

According to VCEP guidelines: "Co-segregation with disease in multiple affected family members". No segregation data are available. Therefore, this criterion is not applied.

PP2

PP2 (Not Applied)

According to standard ACMG: "Missense variant in a gene with low benign missense variation". This is a nonsense variant. Therefore, this criterion is not applied.

PP3

PP3 (Not Applied)

According to VCEP guidelines: "Supporting: multiple computational tools predict deleterious effect (REVEL>0.7 for missense; SpliceAI for splicing)". Computational evidence (CADD 9.96, SpliceAI 0.08) does not support a deleterious effect. Therefore, this criterion is not applied.

PP4

PP4 (Not Applied)

According to standard ACMG: "Patient’s phenotype or family history is highly specific for a disease with a single genetic etiology". No phenotype/family history data are provided. Therefore, this criterion is not applied.

PP5

PP5 (Supporting)

According to standard ACMG: "Supporting: Reputable source recently reports variant as pathogenic, but the evidence is not available to the laboratory". ClinVar lists this variant as Pathogenic by two submitters without primary data. Therefore, this criterion is applied at Supporting strength.

BA1

BA1 (Not Applied)

According to VCEP guidelines: "Stand Alone: gnomAD allele frequency >0.00056". The variant is absent from gnomAD. Therefore, this criterion is not applied.

BS1

BS1 (Not Applied)

According to VCEP guidelines: "Strong: allele frequency 0.000043–0.00056". The variant is absent from population databases. Therefore, this criterion is not applied.

BS2

BS2 (Not Applied)

According to VCEP guidelines: "Strong: observed homozygous in unaffected individual". No homozygous observations are reported. Therefore, this criterion is not applied.

BS3

BS3 (Not Applied)

According to VCEP guidelines: "Strong: well-established functional studies show no damaging effect". Functional studies demonstrate a damaging effect. Therefore, this criterion is not applied.

BS4

BS4 (Not Applied)

According to VCEP guidelines: "Strong: lack of segregation in affected members of ≥2 families". No segregation data are available. Therefore, this criterion is not applied.

BP1

BP1 (Not Applied)

According to VCEP guidelines: "Missense in a gene where only truncating variants cause disease". This is a truncating variant. Therefore, this criterion is not applied.

BP2

BP2 (Not Applied)

According to VCEP guidelines: "Observed in cis/trans with a pathogenic variant for recessive inheritance". Not applicable to this dominant PTEN variant. Therefore, this criterion is not applied.

BP3

BP3 (Not Applied)

According to VCEP guidelines: "In-frame indels in repetitive regions without known function". This is a nonsense variant. Therefore, this criterion is not applied.

BP4

BP4 (Supporting)

According to VCEP guidelines: "Supporting: multiple computational tools suggest no impact". CADD is below pathogenic threshold (9.96) and SpliceAI score is low (0.08). Therefore, this criterion is applied at Supporting strength.

BP5

BP5 (Not Applied)

According to VCEP guidelines: "Supporting: variant found in a case with an alternate molecular basis for disease". No alternate molecular diagnosis information is provided. Therefore, this criterion is not applied.

BP6

BP6 (Not Applied)

According to standard ACMG: "Supporting: reputable source reports variant as benign without available evidence". No such reports exist. Therefore, this criterion is not applied.

BP7

BP7 (Not Applied)

According to VCEP guidelines: "Supporting: synonymous or intronic variant with no predicted splicing impact". This is a coding nonsense variant. Therefore, this criterion is not applied.