PSEN2

Chr 1

presenilin 2

Also known as: AD3L, AD4, CMD1V, PS2, STM2

Alzheimer's disease (AD) patients with an inherited form of the disease carry mutations in the presenilin proteins (PSEN1 or PSEN2) or the amyloid precursor protein (APP). These disease-linked mutations result in increased production of the longer form of amyloid-beta (main component of amyloid deposits found in AD brains). Presenilins are postulated to regulate APP processing through their effects on gamma-secretase, an enzyme that cleaves APP. Also, it is thought that the presenilins are involved in the cleavage of the Notch receptor such that, they either directly regulate gamma-secretase activity, or themselves act are protease enzymes. Two alternatively spliced transcript variants encoding different isoforms of PSEN2 have been identified. [provided by RefSeq, Jul 2008]

GeneReviewsResearchGenerating clinical summary…
MultiplemechanismLOEUF 0.74
Clinical SummaryPSEN2
🧬
Gene-Disease Validity (ClinGen)
dilated cardiomyopathy 1V · ADLimited

Limited evidence — not for standalone diagnostic reporting

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
11 unique Pathogenic / Likely Pathogenic· 165 VUS of 368 total submissions
💊
Clinical Trials
8 active or recruiting trials — potential therapeutic options may be available
📖
GeneReview available — PSEN2
Authoritative clinical overview · Recommended first read
Open GeneReview ↗
Some data sources returned errors (1)

omim: Error: OMIM fetch failed: 429

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint?
0.74LOEUF
pLI 0.001
Z-score 2.44
OE 0.43 (0.260.74)
Tolerant

Typical tolerance to LoF variation

Missense Constraint?
0.40Z-score
OE missense 0.93 (0.841.03)
254 obs / 272.8 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios?
LoF OE?0.43 (0.260.74)
00.351.4
Missense OE?0.93 (0.841.03)
00.61.4
Synonymous OE?1.21
01.21.6
LoF obs/exp: 9 / 21.1Missense obs/exp: 254 / 272.8Syn Z: -1.77
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
limitedPSEN2-related dilated cardiomyopathyOTHERAD

This gene — mechanism propensity

DN
0.76top 25%
GOF
0.74top 25%
LOF
0.2386th %ile

This gene has evidence for multiple mechanisms of pathogenicity (gain-of-function and dominant-negative). The Badonyi & Marsh model scores dominant-negative highest among its predictions, but genomic evidence (constraint, ClinVar variant spectrum, and literature) most strongly supports gain-of-function. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

GOFprediction above median · 1 literature citation
DNprediction above median

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Literature Evidence

GOFThis gain of function might accelerate the process of neurodegeneration that occurs in Alzheimer's disease, leading to the earlier age of onset characteristic of familial Alzheimer's disease.1

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

References

  1. 1.PMID 8939861

ClinVar Variant Classifications

368 submitted variants in ClinVar

Classification Summary

Pathogenic8
Likely Pathogenic3
VUS165
Likely Benign124
Benign32
Conflicting21
8
Pathogenic
3
Likely Pathogenic
165
VUS
124
Likely Benign
32
Benign
21
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
1
6
1
0
8
Likely Pathogenic
2
1
0
0
3
VUS
8
131
23
3
165
Likely Benign
0
10
39
75
124
Benign
0
0
28
4
32
Conflicting
21
Total111489182353

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

32 pathogenic / likely-pathogenic (of 40) ClinVar copy-number / structural variants overlap PSEN2 — these span large chromosomal regions, not the gene specifically, and are excluded from the counts above. Explore in CNV tools →

Protein Context — Lollipop Plot

PSEN2 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Ataxia Telangiectasia

Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)

RECRUITING
NCT07215416Phase PHASE1, PHASE2Timothy YuStarted 2026-08
Antisense oligonucleotide targeting the ATM gene
Ovarian CancerFallopian TubePrimary Peritoneal Cancer

Study of Carboplatin and Mirvetuximab Soravtansine in First-Line Treatment of Patients Receiving Neoadjuvant Chemotherapy With Advanced-Stage Ovarian, Fallopian Tube or Primary Peritoneal Cancer

RECRUITING
NCT04606914Phase PHASE2University of Alabama at BirminghamStarted 2021-05-27
mirvetuximab soravtansine (MIRV; IMGN853)
Alzheimer DiseaseFamilial Alzheimer Disease (FAD)

The Chinese Familial Alzheimer's Network

RECRUITING
NCT03657732Capital Medical UniversityStarted 2005-01-10
Cerebral Amyloid Aβ Angiopathy

Phenotypic and Molecular Characterisation of Cerebral Amyloid Angiopathy

RECRUITING
NCT06864000University Hospital, RouenStarted 2023-03-31
Breast Atypical HyperplasiaBreast CarcinomaBreast Ductal Carcinoma In Situ

Low Dose Tamoxifen With or Without Omega-3 Fatty Acids for Breast Cancer Risk Reduction

RECRUITING
NCT06195306Phase PHASE2National Cancer Institute (NCI)Started 2025-07-28
Biospecimen CollectionMammographyOmega-3-Acid Ethyl Esters
Neurodegenerative DisordersParkinson DiseaseAlzheimer Disease

An Innovative Method in SAliva Samples for the Early Differential Diagnosis of High-impact NeuroDegenerative Diseases Through Raman Spectroscopy

ENROLLING BY INVITATION
NCT06875739Fondazione Don Carlo Gnocchi OnlusStarted 2025-02-14
Mild Cognitive Impairment(MCI)Alzheimer Disease, Late OnsetFamilial Alzheimer Disease (FAD)

China Cognition and Aging Study

RECRUITING
NCT03653156Capital Medical UniversityStarted 2000-01-01
Subjective Cognitive Decline (SCD)Subjective Cognitive Complaints (SCCs)Subjective Cognitive Impairment

The Signature of Alzheimer's Disease in Subjective Cognitive Decline

RECRUITING
NCT07402161IRCCS Policlinico S. DonatoStarted 2025-10-01