ELN

Chr 7AD

elastin

Also known as: ADCL1, SVAS, WBS, WS

This gene encodes a protein that is one of the two components of elastic fibers. Elastic fibers comprise part of the extracellular matrix and confer elasticity to organs and tissues including the heart, skin, lungs, ligaments, and blood vessels. The encoded protein is rich in hydrophobic amino acids such as glycine and proline, which form mobile hydrophobic regions bounded by crosslinks between lysine residues. Degradation products of the encoded protein, known as elastin-derived peptides or elastokines, bind the elastin receptor complex and other receptors and stimulate migration and proliferation of monocytes and skin fibroblasts. Elastokines can also contribute to cancer progression. Deletions and mutations in this gene are associated with supravalvular aortic stenosis (SVAS) and autosomal dominant cutis laxa. [provided by RefSeq, Aug 2017]

GeneReviewsOMIMResearchGenerating clinical summary…
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
221 unique Pathogenic / Likely Pathogenic· 528 VUS of 1341 total submissions
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Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available
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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 OE?0.64 (0.470.87)
00.351.4
Missense OE?0.99 (0.921.08)
00.61.4
Synonymous OE?1.09
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

This gene — mechanism propensity

DN
0.7325th %ile
GOF
0.74top 25%
LOF
0.3163th %ile

This gene has evidence for multiple mechanisms of pathogenicity (loss-of-function, dominant-negative and gain-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 loss-of-function (haploinsufficiency). Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

LOF1 literature citation · 84% of P/LP variants are LoF · ClinGen HI: Sufficient evidence for dosage pathogenicity
DNprediction above median · 1 literature citation
GOFprediction 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

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.1
LOFSpectrum of findings in a family with nonsyndromic autosomal dominant supravalvular aortic stenosis: a Doppler echocardiographic study2

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

1341 submitted variants in ClinVar

Classification Summary

Pathogenic147
Likely Pathogenic74
VUS528
Likely Benign435
Benign57
Conflicting75
147
Pathogenic
74
Likely Pathogenic
528
VUS
435
Likely Benign
57
Benign
75
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
120
5
22
0
147
Likely Pathogenic
66
5
3
0
74
VUS
18
407
88
15
528
Likely Benign
1
26
204
204
435
Benign
0
2
50
5
57
Conflicting
75
Total2054453672241,316

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

View in ClinVar →

169 pathogenic / likely-pathogenic (of 177) ClinVar copy-number / structural variants overlap ELN — these span large chromosomal regions, not the gene specifically, and are excluded from the counts above. Explore in CNV tools →

Protein Context — Lollipop Plot

ELN · 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

Acute Myeloid Leukemia

Sulfasalazine in AML Treated by Intensive Chemotherapy: Elderly Patients-first Line Treatment

RECRUITING
NCT05580861Phase PHASE1, PHASE2Assistance Publique - Hôpitaux de ParisStarted 2023-05-17
Sulfasalazine
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
Chronic Myeloid Leukemia in Chronic Phase

Real-world Study of Scemblix in the Treatment of Chronic Myeloid Leukemia in China

RECRUITING
NCT07375355Novartis PharmaceuticalsStarted 2026-02-12
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
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
Chronic Myeloid Leukemia, Chronic PhaseAdult CMLLeukemia, Myeloid

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

RECRUITING
NCT05143840Phase PHASE2University of Alabama at BirminghamStarted 2022-04-22
Single Agent AsciminibLow TKIElective Free Treatment
Acute Myeloid Leukemia

Comparing Cytarabine + Daunorubicin Therapy Versus Cytarabine + Daunorubicin + Venetoclax Versus Venetoclax + Azacitidine in Younger Patients With Intermediate Risk AML (A MyeloMATCH Treatment Trial)

RECRUITING
NCT05554393Phase PHASE2National Cancer Institute (NCI)Started 2024-09-13
AzacitidineBiospecimen CollectionBone Marrow Aspiration
Acute Myeloid LeukemiaMixed Lineage Leukemia Gene MutationRefractory AML

A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML

RECRUITING
NCT05735184Phase PHASE1Kura Oncology, Inc.Started 2023-07-18
ZiftomenibVenetoclaxAzacitidine
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
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
Leukemia

Talazoparib for Cohesin-Mutated AML and MDS With Excess Blasts

ACTIVE NOT RECRUITING
NCT03974217Phase PHASE1Dana-Farber Cancer InstituteStarted 2019-08-01
TalazoparibDecitabine
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