DES

Chr 2ADAR

desmin

Also known as: CDCD3, CSM1, CSM2, LGMD1D, LGMD1E, LGMD2R

Desmin encodes a muscle-specific intermediate filament protein that forms filamentous networks connecting myofibrils to each other and to the plasma membrane in cardiac and skeletal muscle. Mutations cause desmin-related myopathy and myofibrillar myopathy type 1, presenting as familial cardiac and skeletal myopathy, dilated cardiomyopathy, distal myopathies, or scapuloperoneal syndrome through dominant-negative mechanisms. Inheritance is autosomal dominant or autosomal recessive.

OMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
MultiplemechanismAD/ARLOEUF 0.603 OMIM phenotypes
Clinical SummaryDES
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Gene-Disease Validity (ClinGen)
arrhythmogenic right ventricular cardiomyopathy · ADModerate

Moderate evidence — consider for supplementary testing

2 total gene-disease associations curated

Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.33) despite low pLI — interpret in context.
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Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.60LOEUF
pLI 0.009
Z-score 3.05
OE 0.33 (0.190.60)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
1.77Z-score
OE missense 0.71 (0.630.79)
206 obs / 291.1 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.33 (0.190.60)
00.351.4
Missense OE0.71 (0.630.79)
00.61.4
Synonymous OE0.87
01.21.6
LoF obs/exp: 8 / 24.2Missense obs/exp: 206 / 291.1Syn Z: 1.11
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveDES-related myofibrillar myopathyOTHERAR
definitiveDES-related myofibrillar myopathyOTHERAD
definitiveDES-related dilated cardiomyopathyOTHERAD
DN
0.96top 5%
GOF
0.87top 5%
LOF
0.1598th %ile

This gene has evidence for multiple mechanisms of pathogenicity (dominant-negative and gain-of-function). Both the Badonyi & Marsh prediction and the broader genomic evidence point to dominant-negative as the predominant mechanism. 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

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

DNWe conclude that the L345P desmin missense mutation causes myopathy by interfering in a dominant-negative manner with the dimerization-polymerization process of intermediate filament assembly.PMID:10545598

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

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

DES · 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

Stage IB Breast CancerStage II Breast Cancer

Standard or Comprehensive Radiation Therapy in Treating Patients With Early-Stage Breast Cancer Previously Treated With Chemotherapy and Surgery

ACTIVE NOT RECRUITING
NCT01872975Phase NANSABP Foundation IncStarted 2013-08
regional nodal XRTchestwall XRTWBI
Breast CancerDepression

E-Mindfulness Approaches for Living After Breast Cancer

RECRUITING
NCT06748222Phase PHASE3NRG OncologyStarted 2025-06-27
Mindfulness (MAPs) Live OnlineMindfulness (MAPs) Digital AppMeditation Only Control Group
Breast CarcinomaMastectomy Patient

Testing the Application of Mepitel Film During Radiation Therapy to Reduce Radiation Related Redness and Peeling in Breast Cancer Patients Following Mastectomy

ACTIVE NOT RECRUITING
NCT04989504Phase PHASE3Alliance for Clinical Trials in OncologyStarted 2022-07-26
Best PracticeWound Dressing MaterialQuality-of-Life Assessment
X-Linked Retinitis Pigmentosa

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

ACTIVE NOT RECRUITING
NCT04794101Phase PHASE3Janssen Research & Development, LLCStarted 2020-12-04
Genetic: AAV5-hRKp.RPGR Intermediate DoseGenetic: AAV5-hRKp.RPGR Low Dose
Pancreatic Acinar Cell CarcinomaPancreatic AdenocarcinomaPancreatic Adenosquamous Carcinoma

The PLATINUM Trial: Optimizing Chemotherapy for the Second-Line Treatment of Metastatic BRCA1/2 or PALB2-Associated Metastatic Pancreatic Cancer

ACTIVE NOT RECRUITING
NCT06115499Phase PHASE2, PHASE3Alliance for Clinical Trials in OncologyStarted 2024-08-13
Nab paclitaxelGemcitabineCisplatin
Recurrent Lung Non-Small Cell CarcinomaStage IV Lung Cancer AJCC v8

Ramucirumab Plus Pembrolizumab vs Usual Care for Treatment of Stage IV or Recurrent Non-Small Cell Lung Cancer Following Immunotherapy, Pragmatica-Lung Study

ACTIVE NOT RECRUITING
NCT05633602Phase PHASE3SWOG Cancer Research NetworkStarted 2023-03-14
ChemotherapyPembrolizumabRamucirumab
Opioid Use Disorder

Biopsychosocial Markers of Addiction in Opioid Users: an Integrated Approach

ACTIVE NOT RECRUITING
NCT06021548University Hospital, Strasbourg, FranceStarted 2024-02-07
Blood sampleSaliva sampleHair sample
Breast AdenocarcinomaHormone Receptor PositiveStage IA Breast Cancer AJCC v7

Hormone Therapy With or Without Combination Chemotherapy in Treating Women Who Have Undergone Surgery for Node-Negative Breast Cancer (The TAILORx Trial)

ACTIVE NOT RECRUITING
NCT00310180Phase PHASE3National Cancer Institute (NCI)Started 2006-04-07
AnastrozoleExemestaneLaboratory Biomarker Analysis
Uveal Melanoma

Identification of New Candidate Genes for Hereditary Predisposition to Uveal Melanoma

RECRUITING
NCT06550674Phase NACentre Jean PerrinStarted 2024-10-29
Constitutional exome analysis
Breast Carcinoma In SituInvasive Breast CarcinomaStage I Breast Cancer AJCC v7

Testing MK-3475 (Pembrolizumab) as Adjuvant Therapy for Triple Receptor-Negative Breast Cancer

ACTIVE NOT RECRUITING
NCT02954874Phase PHASE3National Cancer Institute (NCI)Started 2017-01-18
Biospecimen CollectionLaboratory Biomarker AnalysisPatient Observation
Advanced (Stage IIIB/IV) Non-small Cell Lung Cancer (NSCLC) With MET Exon 14 (METex14) Skipping Alterations or MET AmplificationLung Adenocarcinoma Stage IIIB/IV

Tepotinib Phase II in NSCLC Harboring MET Alterations (VISION)

ACTIVE NOT RECRUITING
NCT02864992Phase PHASE2EMD Serono Research & Development Institute, Inc.Started 2016-09-13
Tepotinib
Infiltrating Bladder Urothelial CarcinomaStage II Bladder Urothelial CarcinomaStage III Bladder Urothelial Carcinoma

Gemcitabine and Cisplatin Without Cystectomy for Patients With Muscle Invasive Bladder Urothelial Cancer and Select Genetic Alterations

ACTIVE NOT RECRUITING
NCT03609216Phase PHASE2Alliance for Clinical Trials in OncologyStarted 2018-12-10
Gemcitabine HydrochlorideCisplatinPegfilgrastim
Clinical Literature
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Full-Text Mentions
NLP-detected gene mentions in article bodies · via PubTator3
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