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.996del
Protein Change
A333Pfs*11
Location
Exon 8 (Exon 8 of )
8
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
COSM6934741
Recurrence
2 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 A333Pfs*11 variant is a truncating mutation in the tumor suppressor gene PTEN. Functional evidence indicates that truncating mutations in PTEN result in the loss of phosphatase function, leading to an inability to negatively regulate the PI3K/AKT pathway. Experimental studies have shown that expression of PTEN truncation mutations in mouse embryonic fibroblasts is oncogenic and increases genomic fragility due to impaired chromosomal centromere association. This functional evidence supports a damaging effect of the PTEN A333Pfs*11 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) 0.0 -148 bp
- Donor Loss (DL) 0.01 -171 bp
+ Acceptor Gain (AG) 0.0 -191 bp
+ Donor Gain (DG) 0.0 278 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 Use PTEN PVS1 decision tree." The evidence for this variant shows a frameshift deletion c.996del leading to a premature stop (A333Pfs*11) in a gene where loss of function is a known mechanism. Therefore, this criterion is applied at Very Strong strength because the variant clearly results in loss of function guided by the PTEN-specific PVS1 decision tree.

PS1

PS1 (Not Applied)

According to VCEP guidelines, the rule for PS1 is: "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, where in silico models predict impact equal to or greater than the known pathogenic variant." The evidence shows this variant is a frameshift, 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 Two proven OR four assumed OR one proven + two assumed de novo observations in a patient with the disease and no family history." There are no reported de novo observations for this variant. Therefore, this criterion is not applied.

PS3

PS3 (Strong)

According to PTEN Pre-processing, the finding for PS3 is: "The PTEN A333Pfs*11 variant is a truncating mutation in the tumor suppressor gene PTEN. Functional evidence indicates that truncating mutations in PTEN result in the loss of phosphatase function, leading to an inability to negatively regulate the PI3K/AKT pathway. Experimental studies have shown that expression of PTEN truncation mutations in mouse embryonic fibroblasts is oncogenic and increases genomic fragility due to impaired chromosomal centromere association. This functional evidence supports a damaging effect of the PTEN A333Pfs*11 variant." The evidence directly demonstrates damaging functional impact. Therefore, this criterion is applied at Strong strength.

PS4

PS4 (Not Applied)

According to VCEP guidelines, the rule for PS4 is: "Strong Probands with specificity score 4-15.5 OR prevalence significantly increased in affected vs controls." There are no case-control prevalence data or specificity score reported. Therefore, this criterion is not applied.

PM1

PM1 (Not Applied)

According to VCEP guidelines, the rule for PM1 is: "Moderate Located in a mutational hot spot and/or critical and well-established functional domain, defined to include residues in catalytic motifs: 90-94, 123-130, 166-168." This variant occurs at codon 333 outside those motifs. Therefore, this criterion is not applied.

PM2

PM2 (Supporting)

According to VCEP guidelines, the rule for PM2 is: "Supporting Absent in population Databases present at <0.00001 (0.001%) allele frequency in gnomAD or another large sequenced population." The variant is absent from gnomAD and other control databases. Therefore, this criterion is applied at Supporting strength.

PM3

PM3 (Not Applied)

According to standard ACMG guidelines, the rule for PM3 is: "Detected in trans with a pathogenic variant for a recessive disorder." PTEN-related conditions are autosomal dominant and no trans observations are reported. Therefore, this criterion is not applied.

PM4

PM4 (Not Applied)

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

PM5

PM5 (Not Applied)

According to VCEP guidelines, the rule for PM5 is: "Moderate Missense change at an amino acid residue where a different missense change determined to be pathogenic has been seen before." This is 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 Two proven OR four assumed OR one proven + two assumed de novo observations without confirmation of paternity/maternity." No de novo data exist. Therefore, this criterion is not applied.

PP1

PP1 (Not Applied)

According to VCEP guidelines, the rule for PP1 is: "Supporting/Moderate/Strong 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 guidelines, the rule for PP2 is: "Supporting Missense variant in a gene with a low rate of benign missense variation where missense variants are a common mechanism of disease." This is a loss-of-function frameshift variant. Therefore, this criterion is not applied.

PP3

PP3 (Not Applied)

According to VCEP guidelines, the rule for PP3 is: "Supporting Multiple lines of computational evidence support a deleterious effect on the gene or gene product." A frameshift leading to truncation is already captured by PVS1; computational predictions are not required. Therefore, this criterion is not applied.

PP4

PP4 (Not Applied)

According to standard ACMG guidelines, the rule for PP4 is: "Supporting Patient’s phenotype is highly specific for a disease with a single genetic etiology." No 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 Reputable source classifies variant as pathogenic or likely pathogenic without available evidence." No such classification exists. Therefore, this criterion is not applied.

BA1

BA1 (Not Applied)

According to VCEP guidelines, the rule for BA1 is: "Stand Alone gnomAD Filtering allele frequency >0.00056 (0.056%)." The variant is absent. Therefore, this criterion is not applied.

BS1

BS1 (Not Applied)

According to VCEP guidelines, the rule for BS1 is: "Strong gnomAD Filtering allele frequency from 0.000043 up to 0.00056 (0.0043%-0.056%)." The variant is absent. Therefore, this criterion is not applied.

BS2

BS2 (Not Applied)

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

BS3

BS3 (Not Applied)

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

BS4

BS4 (Not Applied)

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

BP1

BP1 (Not Applied)

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

BP2

BP2 (Not Applied)

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

BP3

BP3 (Not Applied)

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

BP4

BP4 (Not Applied)

According to VCEP guidelines, the rule for BP4 is: "Supporting Multiple lines of computational evidence suggest no impact on gene or gene product." Computational predictions are irrelevant for a clear loss-of-function variant. Therefore, this criterion is not applied.

BP5

BP5 (Not Applied)

According to VCEP guidelines, the rule for BP5 is: "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 Reputable source classifies variant as benign or likely benign without available evidence." No such classification exists. Therefore, this criterion is not applied.

BP7

BP7 (Not Applied)

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