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.368del
Protein Change
H123Pfs*11
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
Uncertain Significance (VUS)
1 publications
Clinical Statement

"This variant has been reported in ClinVar as Uncertain significance (1 clinical laboratories)."

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 H123Pfs*11 variant is a truncating mutation that results in the loss of PTEN phosphatase function. This loss of function impairs the negative regulation of the PI3K/AKT pathway, contributing to oncogenic activity. Functional studies have demonstrated that such truncating mutations in PTEN lead to increased genome fragility and an inability to associate with chromosomal centromeres, supporting a damaging effect.

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 -75 bp
- Donor Loss (DL) 0.0 129 bp
+ Acceptor Gain (AG) 0.01 -5 bp
+ Donor Gain (DG) 0.0 125 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: "Very Strong: Null variant (nonsense, frameshift, start codon, etc.) in a gene where LOF is a known mechanism of disease." The evidence shows that c.368del (H123Pfs*11) is a frameshift predicted to result in NMD in PTEN, a gene with haploinsufficiency. Therefore, PVS1 is applied at Very Strong strength because it is a truncating null variant in a gene where LOF causes disease.

PS1

PS1 (Not Applied)

According to VCEP guidelines: "Strong: Same amino acid change as a previously established pathogenic variant regardless of nucleotide change…" The evidence shows this is a frameshift variant, not a missense change recreating a known pathogenic amino acid substitution. Therefore, PS1 is not applied because the rule is not met.

PS2

PS2 (Not Applied)

According to VCEP guidelines: "Very Strong: Two proven or four assumed de novo observations…" There are no data on de novo occurrence for this variant. Therefore, PS2 is not applied due to lack of de novo evidence.

PS3

PS3 (Strong)

According to VCEP guidelines: "Strong: Well-established in vitro or in vivo functional studies supportive of a damaging effect on the gene or gene product." The evidence shows functional studies demonstrate loss of PTEN phosphatase activity, genome instability, and failure to regulate PI3K/AKT pathway. Therefore, PS3 is applied at Strong strength because functional assays support a damaging effect.

PS4

PS4 (Not Applied)

According to VCEP guidelines: "Strong: Prevalence of variant in affected individuals is significantly increased compared with controls or probands with specificity score 4–15.5…" There are no case‐control data or proband specificity scores. Therefore, PS4 is not applied due to absence of evidence.

PM1

PM1 (Not Applied)

According to VCEP guidelines: "Moderate: Located in a mutational hotspot or critical functional domain (residues 90–94, 123–130)." The variant is a frameshift and not a missense change within these residues. Therefore, PM1 is not applied because the rule applies only to missense variants.

PM2

PM2 (Supporting)

According to VCEP guidelines: "Supporting: Absent in population databases present at <0.00001 allele frequency." The variant is absent from gnomAD, ESP, and 1000 Genomes. Therefore, PM2 is applied at Supporting strength because it is absent in controls.

PM3

PM3 (Not Applied)

According to standard ACMG guidelines: "Moderate: For recessive disorders, detected in trans with a pathogenic variant…" PTEN disease is autosomal dominant and no trans observations exist. Therefore, PM3 is not applied due to non‐applicability in a dominant context.

PM4

PM4 (Not Applied)

According to standard ACMG guidelines: "Moderate: Protein length changes due to in-frame indels…" This variant is a frameshift truncation appropriately evaluated under PVS1. Therefore, PM4 is not applied because PVS1 covers null variants.

PM5

PM5 (Not Applied)

According to VCEP guidelines: "Moderate: Missense change at a residue where a different missense change determined to be pathogenic has been seen." The variant is a frameshift, not a missense change. Therefore, PM5 is not applied because it does not meet the missense requirement.

PM6

PM6 (Not Applied)

According to VCEP guidelines: "Strong/Very Strong/Moderate: Assumed de novo occurrences…" No presumed de novo data are available. Therefore, PM6 is not applied due to lack of de novo evidence.

PP1

PP1 (Not Applied)

According to VCEP guidelines: "Supporting: Co-segregation with disease in multiple affected family members (3–4 meioses)…" There are no segregation data. Therefore, PP1 is not applied.

PP2

PP2 (Not Applied)

According to standard ACMG guidelines: "Supporting: Missense variant in a gene with low rate of benign missense variation…" The variant is not a missense change. Therefore, PP2 is not applied.

PP3

PP3 (Not Applied)

According to VCEP guidelines: "Supporting: Multiple lines of computational evidence support a deleterious effect…" The variant is a frameshift null change, and computational splicing predictions show minimal impact on splicing but are irrelevant to its LOF effect. Therefore, PP3 is not applied.

PP4

PP4 (Not Applied)

According to standard ACMG guidelines: "Supporting: Patient’s phenotype is highly specific for a disease with single genetic etiology…" No phenotype data were provided. Therefore, PP4 is not applied.

PP5

PP5 (Not Applied)

According to standard ACMG guidelines: "Supporting: Reputable source reports variant as pathogenic…" ClinVar classifies this variant as VUS, not (likely) pathogenic. Therefore, PP5 is not applied.

BA1

BA1 (Not Applied)

According to VCEP guidelines: "Stand Alone: Allele frequency >0.00056…" The variant is not frequent. Therefore, BA1 is not applied.

BS1

BS1 (Not Applied)

According to VCEP guidelines: "Strong: Allele frequency 0.000043–0.00056…" The variant frequency is zero. Therefore, BS1 is not applied.

BS2

BS2 (Not Applied)

According to VCEP guidelines: "Strong: Observed homozygous in healthy…" No homozygous observations exist. Therefore, BS2 is not applied.

BS3

BS3 (Not Applied)

According to VCEP guidelines: "Strong: Functional studies show no damaging effect…" Functional studies show damaging effect. Therefore, BS3 is not applied.

BS4

BS4 (Not Applied)

According to VCEP guidelines: "Strong: Lack of segregation in affected members…" No segregation data. Therefore, BS4 is not applied.

BP1

BP1 (Not Applied)

According to standard ACMG guidelines: "Supporting: Missense variant in a gene where truncating variants are known mechanism…" The variant is truncating and not missense. Therefore, BP1 is not applied.

BP2

BP2 (Not Applied)

According to VCEP guidelines: "Supporting: Observed in trans with pathogenic variant…" No such observations. Therefore, BP2 is not applied.

BP3

BP3 (Not Applied)

According to standard ACMG guidelines: "Supporting: In-frame indels in repetitive region…" The variant is a frameshift truncation. Therefore, BP3 is not applied.

BP4

BP4 (Not Applied)

According to VCEP guidelines: "Supporting: Multiple lines of computational evidence suggest no impact…" Although splicing algorithms predict minimal impact, the variant causes LOF by frameshift. Therefore, BP4 is not applied.

BP5

BP5 (Not Applied)

According to VCEP guidelines: "Supporting: Variant found with alternate molecular basis…" No alternate molecular basis reported. Therefore, BP5 is not applied.

BP6

BP6 (Not Applied)

According to standard ACMG guidelines: "Supporting: Reputable source labels as benign…" ClinVar lists as VUS, not benign. Therefore, BP6 is not applied.

BP7

BP7 (Not Applied)

According to VCEP guidelines: "Supporting: Synonymous or intronic variant…" The variant is not synonymous or intronic. Therefore, BP7 is not applied.