ELN

Chr 7AD

elastin

Also known as: ADCL1, SVAS, WBS, WS

Elastin is a major structural protein that provides elasticity to tissues including blood vessels, skin, lungs, and ligaments, and plays a critical role in late arterial morphogenesis by stabilizing arterial structure and regulating vascular smooth muscle organization. Mutations cause autosomal dominant supravalvular aortic stenosis and cutis laxa, affecting cardiovascular and connective tissue systems. This gene is not highly constrained against loss-of-function variants.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismADLOEUF 0.872 OMIM phenotypes
Clinical SummaryELN
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Gene-Disease Validity (ClinGen)
cutis laxa, autosomal dominant 1 · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
51 unique Pathogenic / Likely Pathogenic· 166 VUS of 400 total submissions
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Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available
📖
GeneReview available — ELN
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.87LOEUF
pLI 0.000
Z-score 2.26
OE 0.64 (0.470.87)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
0.05Z-score
OE missense 0.99 (0.921.08)
427 obs / 429.8 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.64 (0.470.87)
00.351.4
Missense OE0.99 (0.921.08)
00.61.4
Synonymous OE1.09
01.21.6
LoF obs/exp: 29 / 45.4Missense obs/exp: 427 / 429.8Syn Z: -0.95
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveELN-related cutis laxaLOFAD
DN
0.7325th %ile
GOF
0.74top 25%
LOF
0.3163th %ile

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

DNprediction above median · 1 literature citation
GOFprediction above median
LOF1 literature citation · 80% of P/LP variants are LoF

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

DNInterestingly, molecular analysis on patient fibroblasts showed that the c.2044+5G>C mutant allele encodes for an aberrant shorter form of the elastin polypeptide that may hamper the normal assembly of elastin fibers in a dominant-negative manner.PMID:19844261
LOFSpectrum of findings in a family with nonsyndromic autosomal dominant supravalvular aortic stenosis: a Doppler echocardiographic studyPMID:2913119

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

400 submitted variants in ClinVar

Classification Summary

Pathogenic31
Likely Pathogenic20
VUS166
Likely Benign149
Benign5
Conflicting4
31
Pathogenic
20
Likely Pathogenic
166
VUS
149
Likely Benign
5
Benign
4
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
21
1
9
0
31
Likely Pathogenic
20
0
0
0
20
VUS
7
125
30
4
166
Likely Benign
1
7
74
67
149
Benign
0
0
4
1
5
Conflicting
4
Total4913311772375

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

View in ClinVar →

Protein Context — Lollipop Plot

ELN · protein map & ClinVar variants

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

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Acute Myeloid LeukemiaRecurrent Acute Myeloid LeukemiaRefractory Acute Myeloid Leukemia

Entrectinib in Combination With ASTX727 for the Treatment of Relapsed/Refractory TP53 Mutated Acute Myeloid Leukemia

ACTIVE NOT RECRUITING
NCT05396859Phase PHASE1OHSU Knight Cancer InstituteStarted 2022-10-28
Decitabine and CedazuridineEntrectinibLaboratory Biomarker Analysis
Chronic Myeloid Leukemia, Chronic PhaseAdult CMLLeukemia, Myeloid

Asciminib as Initial Therapy for Patients With Chronic Myeloid Leukemia in Chronic Phase

RECRUITING
NCT05143840Phase PHASE2Augusta UniversityStarted 2022-04-22
Single Agent AsciminibLow TKIElective Free Treatment
Acute Myeloid LeukemiaAML, AdultAML With Gene Mutations

Revumenib in Combination With 7+3 + Midostaurin in AML

RECRUITING
NCT06313437Phase PHASE1Richard Stone, MDStarted 2024-12-06
RevumenibMidostaurinCytarabine
Acute Myeloblastic LeukaemiaLeukemia, T-Cell

Safety and Efficacy of GYA01 (CART84) in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) and Acute Lymphoblastic T Leukemia Patients (T-ALL).

RECRUITING
NCT07471789Phase PHASE1, PHASE2Gyala TherapeuticsStarted 2026-02-04
CART84
Acute Myeloid LeukemiaMyelodysplastic NeoplasmChronic Myelomonocytic Leukemia

Oral-ATO for TP53-mutated Myeloid Malignancies

RECRUITING
NCT06778187Phase PHASE2The University of Hong KongStarted 2025-02-07
Oral arsenic trioxide
AML, Adult

OBServatory of Compassionate Use of IVOsidenib in France for Patients With Acute Myeloid Leukemia

RECRUITING
NCT06377579French Innovative Leukemia OrganisationStarted 2024-07-31
Leukemia

Talazoparib for Cohesin-Mutated AML and MDS With Excess Blasts

ACTIVE NOT RECRUITING
NCT03974217Phase PHASE1Dana-Farber Cancer InstituteStarted 2019-08-01
TalazoparibDecitabine
Leukemia, Myeloid, Acute

Toxicity Genetic Determinants and Response to Azacitidine and Venetoclax in AML

RECRUITING
NCT06580106Wake Forest University Health SciencesStarted 2025-04-09
Biospecimen samples
Philadelphia Chromosome-Positive Chronic Myeloid Leukemia

A Study to Investigate Tolerability and Efficacy of Asciminib (Oral) Versus Nilotinib (Oral) in Adult Participants (≥18 Years of Age) With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP)

ACTIVE NOT RECRUITING
NCT05456191Phase PHASE3Novartis PharmaceuticalsStarted 2022-11-21
AsciminibNilotinib
MucopolysaccharidosesCarotid DiseaseCardiac Disease

Cardiovascular Structure and Function in the Mucopolysaccharidoses

ACTIVE NOT RECRUITING
NCT05063435Children's Hospital of Orange CountyStarted 2021-10-13
Carotid ultrasonographyEchocardiography, transthoracicVenipuncture
7q11.23 Microduplication Syndrome (7DUP)Autism Spectrum Disorder (ASD)Neurodevelopmental Disorders (NDD)

Characterization of the Natural History of Microduplication Syndrome 7q11.23

NOT YET RECRUITING
NCT07469566Phase NAHospices Civils de LyonStarted 2026-03-15
clinical assessmentParental questionnairesCognitive assessment
Acute Myeloid Leukemia

Chimeric Antigen Receptor T Cell Redirected to Target CD4 Positive Relapsed Refractory Acute Myeloid Leukemia (AML ) as a Bridge to Allogeneic Stem Cell Transplant

RECRUITING
NCT06197672Phase PHASE1Huda SalmanStarted 2024-03-19
CD4CAR
Clinical Literature
Open Research Assistant →
Full-Text Mentions
NLP-detected gene mentions in article bodies · via PubTator3
PubTator3
Top 5 full-text resultsSearch PubTator3 ↗
Key Publications
Landmark & review papers · by relevance
PubMed
Top 5 results · since 2015Search PubMed ↗