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.344dup
Protein Change
D115Efs*2
Location
Exon 5 (Exon 5 of )
5
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
Unknown
0 publications
Clinical Statement

""

COSMIC Somatic Evidence

Open
COSMIC ID
Recurrence
0 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 D115Efs*2 variant is a truncating mutation that results in the loss of PTEN phosphatase function. Functional studies have demonstrated that such truncating mutations lead to an inability to negatively regulate the PI3K/AKT pathway, contributing to oncogenic activity. Additionally, expression of PTEN truncation mutations in mouse embryonic fibroblasts has shown increased genome fragility due to impaired chromosomal centromere association. These findings support a damaging effect of the PTEN D115Efs*2 variant.

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
Unknown
PP3 Applied
No
REVEL Score
0.0
Threshold: ≥0.75 = PP3 applied

SpliceAI Scores

Window: ±500bp
Effect Type Score Position
- Acceptor Loss (AL) 0.01 393 bp
- Donor Loss (DL) 0.0 149 bp
+ Acceptor Gain (AG) 0.02 -107 bp
+ Donor Gain (DG) 0.0 51 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, the rule for PVS1 is: "Very Strong Strength: Very Strong Use PTEN PVS1 decision tree. Modification Type: Disease-specific". The evidence for this variant shows a frameshift duplication (NM_000314.8:c.344dupA) resulting in a premature stop (D115Efs*2) predicted to cause loss of function. Therefore, this criterion is applied at Very Strong strength because it is a null variant in a gene where loss of function is a known mechanism of disease.

PS1

PS1 (Not Applied)

According to VCEP guidelines, the rule for PS1 is: "Strong Strength: Strong Same amino acid change as a previously established pathogenic variant regardless of nucleotide change OR different variant at same nucleotide position as a pathogenic splicing variant...". The evidence for this variant shows a frameshift alteration, not the same amino acid change as any known pathogenic variant. Therefore, this criterion is not applied.

PS2

PS2 (Not Applied)

According to VCEP guidelines, the rule for PS2 is: "Very Strong Strength: Two proven OR four assumed OR one proven + two assumed de novo observations... Strong Strength: De novo (both maternity and paternity confirmed)...". There are no data regarding de novo occurrence for this variant. Therefore, this criterion is not applied.

PS3

PS3 (Strong)

According to VCEP guidelines, the rule for PS3 is: "Strong Strength: Strong Well-established in vitro or in vivo functional studies supportive of a damaging effect on the gene or gene product.". The evidence for this variant shows multiple functional studies demonstrating that truncating PTEN variants abolish phosphatase activity, disrupt regulation of the PI3K/AKT pathway, and increase genomic instability. Therefore, this criterion is applied at Strong strength because well-established functional assays support a damaging effect.

PS4

PS4 (Not Applied)

According to VCEP guidelines, the rule for PS4 is: "Strong Strength: Probands with specificity score 4-15.5 OR prevalence significantly increased in affected vs controls...". No case-level or cohort data are available for this variant. Therefore, this criterion is not applied.

PM1

PM1 (Not Applied)

According to VCEP guidelines, the rule for PM1 is: "Moderate Strength: Moderate Located in a mutational hot spot and/or critical and well-established functional domain: residues 90-94, 123-130, 166-168.". The variant affects codon 115, which is outside the defined critical functional domains. Therefore, this criterion is not applied.

PM2

PM2 (Supporting)

According to VCEP guidelines, the rule for PM2 is: "Supporting Strength: Supporting Absent in population Databases present at <0.001% allele frequency in gnomAD or another large sequenced population.". The evidence for this variant shows it is not present in gnomAD (MAF=0). Therefore, this criterion is applied at Supporting strength because the variant is absent from large population databases.

PM3

PM3 (Not Applied)

According to VCEP guidelines, the rule for PM3 is: "Moderate Strength: Moderate Detected in trans with a pathogenic variant in recessive disorders...". PTEN-related conditions are autosomal dominant and there is no evidence of a trans configuration with another pathogenic PTEN variant. Therefore, this criterion is not applied.

PM4

PM4 (Not Applied)

According to VCEP guidelines, the rule for PM4 is: "Moderate Strength: Moderate Protein length changes due to in-frame deletions/insertions in a non-repeat region or stop-loss variants...". The variant is a frameshift leading to a premature stop, not an in-frame change. Therefore, this criterion is not applied.

PM5

PM5 (Not Applied)

According to VCEP guidelines, the rule for PM5 is: "Moderate Strength: Moderate Missense change at an amino acid residue where a different missense change determined to be pathogenic has been seen before...". The variant is a frameshift, not a missense change. Therefore, this criterion is not applied.

PM6

PM6 (Not Applied)

According to VCEP guidelines, the rule for PM6 is: "Very Strong Strength: Two proven OR four assumed OR one proven + two assumed de novo observations... Strong Strength: Two probands with presumed de novo occurrence... Moderate Strength: Assumed de novo without confirmation...". No data on assumed de novo occurrence are available. Therefore, this criterion is not applied.

PP1

PP1 (Not Applied)

According to VCEP guidelines, the rule for PP1 is: "Supporting Strength: Supporting Co-segregation with disease in multiple affected family members...". No family segregation data are available. Therefore, this criterion is not applied.

PP2

PP2 (Not Applied)

According to standard ACMG guidelines, the rule for PP2 is: "Supporting Strength: Supporting Missense variant in a gene that has a low rate of benign missense variation and where missense variants are a common mechanism of disease.". The variant is a frameshift, not a missense. Therefore, this criterion is not applied.

PP3

PP3 (Not Applied)

According to VCEP guidelines, the rule for PP3 is: "Supporting Strength: Supporting Multiple lines of computational evidence support a deleterious effect on the gene or gene product...". In silico predictions, including SpliceAI (max score 0.02), do not support a deleterious effect. Therefore, this criterion is not applied.

PP4

PP4 (Not Applied)

According to standard ACMG guidelines, the rule for PP4 is: "Supporting Strength: Supporting Patient's phenotype or family history is highly specific for a disease with a single genetic etiology.". No clinical phenotype data are provided. Therefore, this criterion is not applied.

PP5

PP5 (Not Applied)

According to standard ACMG guidelines, the rule for PP5 is: "Supporting Strength: Supporting Reputable source recently reports variant as pathogenic...". There is no ClinVar or other reputable report. Therefore, this criterion is not applied.

BA1

BA1 (Not Applied)

According to VCEP guidelines, the rule for BA1 is: "Stand Alone Strength: Stand Alone gnomAD Filtering allele frequency >0.056%.". The allele frequency for this variant is 0%. Therefore, this criterion is not applied.

BS1

BS1 (Not Applied)

According to VCEP guidelines, the rule for BS1 is: "Strong Strength: Strong gnomAD Filtering allele frequency from 0.0043% up to 0.056%.". The allele frequency for this variant is 0%. Therefore, this criterion is not applied.

BS2

BS2 (Not Applied)

According to VCEP guidelines, the rule for BS2 is: "Strong Strength: Strong Observed in the homozygous state in a healthy or PHTS-unaffected individual...". No homozygous observations are reported. Therefore, this criterion is not applied.

BS3

BS3 (Not Applied)

According to VCEP guidelines, the rule for BS3 is: "Strong Strength: Strong Well-established in vitro or in vivo functional studies shows no damaging effect on protein function.". Functional studies for this variant demonstrate a damaging effect. Therefore, this criterion is not applied.

BS4

BS4 (Not Applied)

According to VCEP guidelines, the rule for BS4 is: "Strong Strength: Strong Lack of segregation in affected members of two or more families.". No segregation data exist. Therefore, this criterion is not applied.

BP1

BP1 (Not Applied)

According to standard ACMG guidelines, the rule for BP1 is: "Supporting Strength: Supporting Missense variant in a gene for which primarily truncating variants are known to cause disease.". This variant is truncating, not missense. Therefore, this criterion is not applied.

BP2

BP2 (Not Applied)

According to VCEP guidelines, the rule for BP2 is: "Supporting Strength: Supporting Observed in trans with a pathogenic or likely pathogenic PTEN variant...". No such observations are reported. Therefore, this criterion is not applied.

BP3

BP3 (Not Applied)

According to standard ACMG guidelines, the rule for BP3 is: "Supporting Strength: Supporting In-frame deletions/insertions in a repetitive region without a known function.". The variant is a frameshift, not in-frame. Therefore, this criterion is not applied.

BP4

BP4 (Not Applied)

According to VCEP guidelines, the rule for BP4 is: "Supporting Strength: Supporting Multiple lines of computational evidence suggest no impact on gene or gene product.". Computational evidence does not indicate benign effect for a truncating variant. Therefore, this criterion is not applied.

BP5

BP5 (Not Applied)

According to VCEP guidelines, the rule for BP5 is: "Supporting Strength: Supporting Variant found in a case with an alternate molecular basis for disease.". No alternate molecular basis is reported. Therefore, this criterion is not applied.

BP6

BP6 (Not Applied)

According to standard ACMG guidelines, the rule for BP6 is: "Supporting Strength: Supporting Reputable source reports variant as benign.". No such report exists. Therefore, this criterion is not applied.

BP7

BP7 (Not Applied)

According to VCEP guidelines, the rule for BP7 is: "Supporting Strength: Supporting A synonymous or intronic variant at or beyond +7/-21 for which splicing prediction algorithms predict no impact.". This variant is not synonymous or intronic. Therefore, this criterion is not applied.