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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 15 days ago
β“˜GeneE is for informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment.
CASQ1
calsequestrin 1
Chromosome 1 Β· 1q23.2
NCBI Gene: 844Ensembl: ENSG00000143318.14HGNC: HGNC:1512UniProt: P31415
39PubMed Papers
22Diseases
0Drugs
5Pathogenic Variants
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
regulation of store-operated calcium entrypositive regulation of release of sequestered calcium ion into cytosolpositive regulation of store-operated calcium channel activityendoplasmic reticulummyopathy due to calsequestrin and SERCA1 protein overloadmyopathy, tubular aggregate, 1tubular aggregate myopathygenetic disorder
✦AI Summary

CASQ1 encodes calsequestrin-1, a high-capacity calcium-binding protein that serves as the primary calcium buffer in the sarcoplasmic reticulum (SR) of skeletal muscle fibers 1. The protein can bind approximately 80 calcium ions and regulates calcium release through ryanodine receptor type-1 (RyR1), playing a crucial role in excitation-contraction coupling and muscle contraction 12. CASQ1 functions through calcium-dependent polymerization and physically interacts with RyR1 to modulate its activity 3. Additionally, CASQ1 negatively regulates store-operated calcium entry (SOCE), preventing excessive calcium influx when SR stores are depleted 42. Mutations in CASQ1 cause tubular aggregate myopathy (TAM), characterized by muscle weakness and accumulation of SR tubules 14. Disease-associated mutations impair calcium-dependent aggregation, reduce calcium storage capacity, and diminish SOCE inhibition 14. CASQ1 deficiency is also associated with malignant hyperthermia susceptibility and heat stroke, as CASQ1-knockout mice exhibit excessive SR calcium leak and are prone to sudden death under heat or anesthetic exposure 52. The protein is also expressed in cardiac muscle, where its deficiency contributes to arrhythmias 3.

Sources cited
1
CASQ1 binds approximately 80 calcium ions and causes tubular aggregate myopathy when mutated
PMID: 28895244
2
CASQ1 is expressed in cardiac muscle and interacts with RyR1 to regulate calcium release
PMID: 34162222
3
CASQ1 mutations are associated with heat stroke susceptibility
PMID: 24887214
4
TAM-associated CASQ1 mutations diminish calcium storage capacity and lose SOCE inhibition ability
PMID: 39126637
5
CASQ1-knockout mice show excessive SR calcium leak and sudden death under stress conditions
PMID: 36311237
Disease Associationsβ“˜22
myopathy due to calsequestrin and SERCA1 protein overloadOpen Targets
0.63Moderate
myopathy, tubular aggregate, 1Open Targets
0.60Moderate
tubular aggregate myopathyOpen Targets
0.50Moderate
genetic disorderOpen Targets
0.19Weak
Decreased total leukocyte countOpen Targets
0.18Weak
neutropeniaOpen Targets
0.18Weak
myopathyOpen Targets
0.12Weak
Distal myopathy, Nonaka typeOpen Targets
0.05Suggestive
paramyotonia congenita of Von EulenburgOpen Targets
0.05Suggestive
myopathy, distal, 5Open Targets
0.05Suggestive
autosomal recessive limb-girdle muscular dystrophy type 2LOpen Targets
0.05Suggestive
GNE myopathyOpen Targets
0.05Suggestive
Autosomal dominant limb-girdle muscular dystrophy type 1DOpen Targets
0.04Suggestive
distal myopathyOpen Targets
0.04Suggestive
tibial muscular dystrophyOpen Targets
0.04Suggestive
X-linked myopathy with excessive autophagyOpen Targets
0.04Suggestive
Autosomal recessive limb-girdle muscular dystrophy due to ISPD deficiencyOpen Targets
0.04Suggestive
myopathy, distal, 6, adult-onset, autosomal dominantOpen Targets
0.04Suggestive
Thomsen and Becker diseaseOpen Targets
0.04Suggestive
polyglucosan body myopathy type 2Open Targets
0.04Suggestive
Myopathy, tubular aggregate, 1UniProt
Myopathy, vacuolar, with CASQ1 aggregatesUniProt
Pathogenic Variants5
NM_001231.5(CASQ1):c.1154T>C (p.Ile385Thr)Likely pathogenic
not provided
β˜…β˜†β˜†β˜†2025β†’ Residue 385
NM_001231.5(CASQ1):c.166A>T (p.Asn56Tyr)Pathogenic
Myopathy with tubular aggregates
β˜…β˜†β˜†β˜†2024β†’ Residue 56
NM_001231.5(CASQ1):c.984+1G>ALikely pathogenic
Myopathy due to calsequestrin and SERCA1 protein overload
β˜…β˜†β˜†β˜†2024
NM_001231.5(CASQ1):c.731A>G (p.Asp244Gly)Pathogenic
Myopathy due to calsequestrin and SERCA1 protein overload|not provided
β˜…β˜†β˜†β˜†2022β†’ Residue 244
NM_001231.5(CASQ1):c.790del (p.Thr263_Leu264insTer)Likely pathogenic
not provided
β˜…β˜†β˜†β˜†2016β†’ Residue 263
View on ClinVar β†—
Related Genes
TRDNProtein interaction100%MYH6Protein interaction99%MYL1Protein interaction97%RYR1Protein interaction96%RYR3Protein interaction95%JPH2Protein interaction95%
Tissue Expression6 tissues
Heart
100%
Brain
26%
Lung
3%
Liver
1%
Ovary
1%
Bone Marrow
0%
Gene Interaction Network
Click a node to explore
CASQ1TRDNMYH6MYL1RYR1RYR3JPH2
PROTEIN STRUCTURE
Preparing viewer…
PDB5CRG Β· 1.97 Γ… Β· X-ray
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
0.96LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.68 [0.48–0.96]
RankingsWhere CASQ1 stands among ~20K protein-coding genes
  • #10,287of 20,598
    Most Researched39
  • #3,599of 5,498
    Most Pathogenic Variants5
  • #9,137of 17,882
    Most Constrained (LOEUF)0.96
Genes detectedCASQ1
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
A 20-year Clinical and Genetic Neuromuscular Cohort Analysis in Lebanon: An International Effort.
PMID: 34602496
J Neuromuscul Dis Β· 2022
1.00
2
Functional Calsequestrin-1 Is Expressed in the Heart and Its Deficiency Is Causally Related to Malignant Hyperthermia-Like Arrhythmia.
PMID: 34162222
Circulation Β· 2021
0.90
3
An association study of CASQ1 gene polymorphisms and heat stroke.
PMID: 24887214
Genomics Proteomics Bioinformatics Β· 2014
0.80
4
Identification and characterization of three novel mutations in the CASQ1 gene in four patients with tubular aggregate myopathy.
PMID: 28895244
Hum Mutat Β· 2017
0.70
5
TRIM24 regulates chromatin remodeling and calcium dynamics in cardiomyocytes.
PMID: 40598158
Cell Commun Signal Β· 2025
0.60