Genetic Information

Gene & Transcript Details

Gene
PALB2
Transcript
NM_024675.3 MANE Select
Total Exons
Reference Sequence
NC_000016.9
Alternative Transcripts
IDStatusDetails
NM_024675.4 MANE Select 4008 nt | 154–3714
NM_024675.3 RefSeq Select 4069 nt | 201–3761

Variant Details

HGVS Notation
NM_024675.3:c.49-2A>T
Protein Change
Splice
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
Likely Pathogenic
2 publications
Publications List
PMID: 25452441

The c.49-2A>T intronic variant results from an A to T substitution two nucleotides upstream from coding exon 2 in the PALB2 gene. This alteration was identified in 1/1824 triple-negative breast cancer patients unselected for family history of breast or ovarian cancer (Couch FJ et al. J. Clin. Oncol. 2015 Feb; 33(4):304-11). This nucleotide position is highly conserved in available vertebrate species. In silico splice site analysis predicts that this alteration will weaken the native splice acceptor site and will result in the creation or strengthening of a novel splice acceptor site. Usage of this novel acceptor site would result in an in-frame transcript lacking two highly conserved amino acids in the coiled-coiled domain and is predicted to significantly disrupt a protein-protein interface (Ambry internal data). RNA studies have demonstrated that this alteration results in abnormal splicing in the set of samples tested (Ambry internal data; Valenzuela-Palomo A et al. J Pathol, 2022 Mar;256:321-334). This variant is considered to be rare based on population cohorts in the Genome Aggregation Database (gnomAD). Based on the majority of available evidence to date, this variant is likely to be pathogenic.

PMID: 25452441

This submission and the accompanying classification are no longer maintained by the submitter. For more information on current observations and classification, please contact variantquestions@myriad.com.

PMID: 25452441

This sequence change affects an acceptor splice site in intron 1 of the PALB2 gene. RNA analysis indicates that disruption of this splice site induces altered splicing and likely results in a shortened protein product. This variant is not present in population databases (gnomAD no frequency). Disruption of this splice site has been observed in individual(s) with PALB2-related conditions (PMID: 25452441, 29478780). ClinVar contains an entry for this variant (Variation ID: 186820). Studies have shown disruption of this splice site is associated with skipping of 2 amino acids, but one or more of the resulting mRNA isoform(s) may be naturally occurring (PMID: 30890586, 34846068). In summary, the available evidence is currently insufficient to determine the role of this variant in disease. Therefore, it has been classified as a Variant of Uncertain Significance.

PMID: 25452441

Curators: Marc Tischkowitz, Arleen D. Auerbach. Submitter to LOVD: Marc Tischkowitz.

Clinical Statement

"This variant has been reported in ClinVar as Likely pathogenic (2 clinical laboratories) and as Uncertain significance (3 clinical laboratories) and as Uncertain Significance by ClinGen Hereditary Breast, Ovarian and Pancreatic Cancer Variant Curation Expert Panel, ClinGen expert panel."

COSMIC Somatic Evidence

Open
COSMIC ID
Recurrence
0 occurrences
PM1 Criteria
Not Applied
COSMIC Database 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 PALB2.

Functional Studies & Therapeutic Relevance

Functional Summary

Error in OpenAI Consolidation. OncoKB: PALB2SplicePALB2SpliceSomaticNCBI Gene:79728|Show additional gene information Variant OverviewPALB2, a scaffolding protein involved in DNA repair, is altered in various cancers.The PALB2 splice alteration has not specifically been reviewed by the OncoKB team, and therefore its biological significance is unknown. JAX-CKB: No results found

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
Yes
REVEL Score
0.0
Threshold: ≥0.75 = PP3 applied

SpliceAI Scores

Window: ±500bp
Effect Type Score Position
- Acceptor Loss (AL) 0.97 -2 bp
- Donor Loss (DL) 0.2 -61 bp
+ Acceptor Gain (AG) 0.71 -8 bp
+ Donor Gain (DG) 0.0 342 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 (Unknown (Pre-LLM))

From pre-LLM assessment (LLM Failed)

PM2

PM2 (Unknown (Pre-LLM))

From pre-LLM assessment (LLM Failed)

PP3

PP3 (Unknown (Pre-LLM))

From pre-LLM assessment (LLM Failed)

PP5

PP5 (Unknown (Pre-LLM))

From pre-LLM assessment (LLM Failed)