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.159_160dup
Protein Change
V54Efs*2
Location
Exon 2 (Exon 2 of )
2
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 V54Efs*2 variant is a truncating mutation that results in the loss of PTEN phosphatase function, leading to an inability to negatively regulate the PI3K/AKT pathway. Functional studies have demonstrated that such truncating mutations are oncogenic, increasing genome fragility and disrupting chromosomal centromere association.

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) 0.02 -107 bp
- Donor Loss (DL) 0.0 221 bp
+ Acceptor Gain (AG) 0.03 -85 bp
+ Donor Gain (DG) 0.02 6 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 it is a frameshift (c.159_160dup, V54Efs*2) causing a null allele in PTEN, a gene where loss of function is a known disease mechanism and the variant does not occur in the last exon. Therefore, this criterion is applied at Very Strong strength because it meets the PTEN-specific truncating variant rule.

PS1

PS1 (Not Applied)

According to VCEP guidelines, the rule for PS1 is: "Same amino acid change as a previously established pathogenic variant regardless of nucleotide change." The evidence for this variant shows a novel frameshift change with no matching known pathogenic amino acid change. Therefore, this criterion is not applied.

PS2

PS2 (Not Applied)

According to VCEP guidelines, the rule for PS2 is: "De novo occurrence confirmed by paternity and maternity." There is no information on de novo status for this variant. Therefore, this criterion is not applied.

PS3

PS3 (Strong)

According to PTEN Pre-processing, the finding for PS3 is: "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 functional studies demonstrating loss of PTEN phosphatase activity and oncogenic effect. Therefore, this criterion is applied at Strong strength because it meets the PTEN-specific functional evidence requirement.

PS4

PS4 (Not Applied)

According to VCEP guidelines, the rule for PS4 is: "Prevalence of the variant in affected individuals is significantly increased compared with controls or probands with specificity score ≥4." There are no case-control or proband specificity data. Therefore, this criterion is not applied.

PM1

PM1 (Not Applied)

According to VCEP guidelines, the rule for PM1 is: "Located in a mutational hotspot or critical functional domain (residues 90-94, 123-130, 166-168)." The variant at residue 54 is outside these motifs. Therefore, this criterion is not applied.

PM2

PM2 (Supporting)

According to VCEP guidelines, the rule for PM2 is: "Supporting Absent in population databases (allele frequency <0.00001 in gnomAD)." 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 controls.

PM3

PM3 (Not Applied)

According to standard ACMG guidelines, PM3 applies to recessive disorders with trans observations. PTEN-related disease is autosomal dominant and no trans data are relevant. Therefore, this criterion is not applied.

PM4

PM4 (Not Applied)

According to VCEP guidelines, PM4 applies to in-frame indels causing protein length changes. This variant is a frameshift leading to truncation. Therefore, this criterion is not applied.

PM5

PM5 (Not Applied)

According to VCEP guidelines, PM5 applies to novel missense changes at residues with known pathogenic missense variants. This is a frameshift, not a missense variant. Therefore, this criterion is not applied.

PM6

PM6 (Not Applied)

According to VCEP guidelines, PM6 applies to assumed de novo occurrences without confirmation. No de novo data are available. Therefore, this criterion is not applied.

PP1

PP1 (Not Applied)

According to VCEP guidelines, PP1 applies to co-segregation with disease in multiple affected family members. No segregation data are provided. Therefore, this criterion is not applied.

PP2

PP2 (Not Applied)

According to standard ACMG guidelines, PP2 applies to missense variants in genes with low benign missense rate. This is a frameshift variant. Therefore, this criterion is not applied.

PP3

PP3 (Not Applied)

According to VCEP guidelines, PP3 requires multiple lines of computational evidence supporting deleterious effect. Computational analyses show minimal splicing impact and no high REVEL score. Therefore, this criterion is not applied.

PP4

PP4 (Not Applied)

According to standard ACMG guidelines, PP4 applies when the phenotype is highly specific for a single gene disorder. No phenotype data are provided. Therefore, this criterion is not applied.

PP5

PP5 (Not Applied)

According to standard ACMG guidelines, PP5 applies when reputable sources report variant as pathogenic without evidence. The variant is not in ClinVar or other databases. Therefore, this criterion is not applied.

BA1

BA1 (Not Applied)

According to VCEP guidelines, BA1 applies to allele frequency >0.056%. The variant frequency is 0%. Therefore, this criterion is not applied.

BS1

BS1 (Not Applied)

According to VCEP guidelines, BS1 applies to allele frequency 0.0043–0.056%. The variant frequency is 0%. Therefore, this criterion is not applied.

BS2

BS2 (Not Applied)

According to VCEP guidelines, BS2 applies to observation in homozygous state in unaffected individuals. No such data are available. Therefore, this criterion is not applied.

BS3

BS3 (Not Applied)

According to VCEP guidelines, BS3 applies to functional studies showing no damaging effect. Functional studies for this variant show damaging effect. Therefore, this criterion is not applied.

BS4

BS4 (Not Applied)

According to VCEP guidelines, BS4 applies to lack of segregation in multiple families. No segregation data are provided. Therefore, this criterion is not applied.

BP1

BP1 (Not Applied)

According to standard ACMG guidelines, BP1 applies to missense variants in genes where only truncating variants cause disease. This variant is truncating. Therefore, this criterion is not applied.

BP2

BP2 (Not Applied)

According to VCEP guidelines, BP2 applies to observations in trans with pathogenic PTEN variants. No such data are available. Therefore, this criterion is not applied.

BP3

BP3 (Not Applied)

According to standard ACMG guidelines, BP3 applies to in-frame indels in repetitive regions. This variant is a frameshift. Therefore, this criterion is not applied.

BP4

BP4 (Supporting)

According to standard ACMG guidelines, BP4 is: "Multiple lines of computational evidence suggest no impact on gene or gene product." Computational analysis shows minimal predicted splicing impact (SpliceAI 0.03). Therefore, this criterion is applied at Supporting strength because computational data predict no impact.

BP5

BP5 (Not Applied)

According to standard ACMG guidelines, BP5 applies when an alternate molecular basis for disease is present. No such alternate basis is reported. Therefore, this criterion is not applied.

BP6

BP6 (Not Applied)

According to standard ACMG guidelines, BP6 applies when a variant is reported benign by a reputable source without evidence. No such report exists. Therefore, this criterion is not applied.

BP7

BP7 (Not Applied)

According to standard ACMG guidelines, BP7 applies to synonymous or intronic variants with no splicing impact. This variant is coding and frameshifting. Therefore, this criterion is not applied.