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GeneE
25 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
β“˜GeneE is for informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment.
RYR1
ryanodine receptor 1
Chromosome 19 Β· 19q13.2
NCBI Gene: 6261Ensembl: ENSG00000196218.15HGNC: HGNC:10483UniProt: A0A8I5KSR1
345PubMed Papers
24Diseases
2Drugs
772Pathogenic Variants
FUNCTIONAL ROLE
Hub GeneIon ChannelReceptorTransporter
RESEARCH IMPACT
Highly StudiedTrendingVariant-Rich
CLINICAL
FDA Approved TargetOMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
extracellular exosomeI bandcell cortexcellular response to caffeinemalignant hyperthermia, susceptibility to, 1congenital multicore myopathy with external ophthalmoplegiacentral core myopathyCentral core disease
✦AI Summary

RYR1 encodes the ryanodine receptor 1 (RyR1), a calcium-activated calcium channel located on the skeletal muscle sarcoplasmic reticulum that mediates calcium release into the cytoplasm, playing a critical role in excitation-contraction coupling and muscle contraction 1. The protein comprises 5,038 amino acids and is fundamental to skeletal muscle calcium homeostasis 1. Pathogenic RYR1 mutations destabilize the channel, leading to pathologic calcium leak from the sarcoplasmic reticulum, which causes oxidative stress and muscle dysfunction 2. This calcium leak increases activity of calcium-activated proteases and contributes to muscle damage 2. RYR1 mutations are associated with multiple disorders including malignant hyperthermia susceptibility, central core disease, multi-minicore disease, and other RYR1-related myopathies 134. These conditions exhibit highly heterogeneous clinical phenotypes ranging from asymptomatic hyperCKemia to severe muscle weakness and respiratory impairment 43. Recent therapeutic approaches target calcium leak stabilization, with RyR1-stabilizing compounds like Rycals showing promise in clinical trials for treating RYR1-related myopathies 52. The genetic basis shows both autosomal dominant and recessive inheritance patterns depending on the specific mutation and associated phenotype 6.

Sources cited
1
RYR1 gene structure, protein function in calcium homeostasis and excitation-contraction coupling
PMID: 16917943
2
Pathogenic mutations cause calcium leak, oxidative stress, and increased protease activity
PMID: 32236737
3
Multi-minicore disease phenotypes and clinical features associated with RYR1 mutations
PMID: 17631035
4
Clinical heterogeneity of RYR1-related myopathies ranging from mild to severe phenotypes
PMID: 35428369
5
Recent therapeutic developments including Rycal compounds for RYR1-related myopathies
PMID: 38994695
6
Genetic epidemiology and inheritance patterns of RYR1 mutations in malignant hyperthermia
PMID: 30236257
Disease Associationsβ“˜24
malignant hyperthermia, susceptibility to, 1Open Targets
0.85Strong
congenital multicore myopathy with external ophthalmoplegiaOpen Targets
0.84Strong
central core myopathyOpen Targets
0.83Strong
Central core diseaseOpen Targets
0.81Strong
King-Denborough syndromeOpen Targets
0.78Strong
RYR1-related myopathyOpen Targets
0.70Moderate
malignant hyperthermia of anesthesiaOpen Targets
0.67Moderate
neuromuscular disease, congenital, with uniform type 1 fiberOpen Targets
0.62Moderate
ryr1-related disordersOpen Targets
0.58Moderate
Minicore myopathyOpen Targets
0.58Moderate
congenital fiber-type disproportion myopathyOpen Targets
0.57Moderate
centronuclear myopathyOpen Targets
0.55Moderate
myopathyOpen Targets
0.55Moderate
genetic disorderOpen Targets
0.55Moderate
arthrogryposis multiplex congenitaOpen Targets
0.54Moderate
congenital myopathyOpen Targets
0.53Moderate
fetal akinesia deformation sequence 1Open Targets
0.53Moderate
fetal akinesia deformation sequenceOpen Targets
0.53Moderate
FeverOpen Targets
0.52Moderate
Abnormality of the musculatureOpen Targets
0.51Moderate
Congenital myopathy 1A, autosomal dominant, with susceptibility to malignant hyperthermiaUniProt
Congenital myopathy 1B, autosomal recessiveUniProt
King-Denborough syndromeUniProt
Malignant hyperthermia 1UniProt
Pathogenic Variants772
NM_000540.3(RYR1):c.14918C>T (p.Pro4973Leu)Likely pathogenic
not provided|Malignant hyperthermia, susceptibility to, 1|RYR1-related disorder|Malignant hyperthermia of anesthesia|Congenital multicore myopathy with external ophthalmoplegia;Central core myopathy;Malignant hyperthermia, susceptibility to, 1|Central core myopathy|Inborn genetic diseases|Congenital multicore myopathy with external ophthalmoplegia;King Denborough syndrome;Central core myopathy;Malignant hyperthermia, susceptibility to, 1|RYR1-related myopathy
β˜…β˜…β˜…β˜†2026β†’ Residue 4973
NM_000540.3(RYR1):c.12700G>T (p.Val4234Leu)Likely pathogenic
RYR1-related disorder|Malignant hyperthermia of anesthesia|not provided
β˜…β˜…β˜…β˜†2026β†’ Residue 4234
NM_000540.3(RYR1):c.12700G>C (p.Val4234Leu)Pathogenic
not provided|Malignant hyperthermia of anesthesia|RYR1-related disorder|Malignant hyperthermia, susceptibility to, 1
β˜…β˜…β˜…β˜†2026β†’ Residue 4234
NM_000540.3(RYR1):c.8189A>G (p.Asp2730Gly)Likely pathogenic
not provided|Malignant hyperthermia of anesthesia
β˜…β˜…β˜…β˜†2025β†’ Residue 2730
NM_000540.3(RYR1):c.6757C>T (p.His2253Tyr)Likely pathogenic
not provided|RYR1-related disorder|Malignant hyperthermia, susceptibility to, 1
β˜…β˜…β˜…β˜†2025β†’ Residue 2253
NM_000540.3(RYR1):c.7354C>T (p.Arg2452Trp)Pathogenic
Central core myopathy|not provided|RYR1-related disorder|King Denborough syndrome|desflurane response - Toxicity|halothane response - Toxicity|enflurane response - Toxicity|isoflurane response - Toxicity|methoxyflurane response - Toxicity|sevoflurane response - Toxicity|succinylcholine response - Toxicity|RYR1-related myopathy|Malignant hyperthermia, susceptibility to, 1
β˜…β˜…β˜…β˜†2025β†’ Residue 2452
NM_000540.3(RYR1):c.1925+1G>CLikely pathogenic
Malignant hyperthermia, susceptibility to, 1|RYR1-related myopathy
β˜…β˜…β˜…β˜†2025
NM_000540.3(RYR1):c.14551C>G (p.Leu4851Val)Likely pathogenic
RYR1-related myopathy
β˜…β˜…β˜…β˜†2025β†’ Residue 4851
NM_000540.3(RYR1):c.10347+1G>APathogenic
Inborn genetic diseases|not provided|RYR1-related disorder|Congenital multicore myopathy with external ophthalmoplegia;Central core myopathy;Malignant hyperthermia, susceptibility to, 1;Congenital myopathy with fiber type disproportion|Congenital multicore myopathy with external ophthalmoplegia|Malignant hyperthermia, susceptibility to, 1|Congenital multicore myopathy with external ophthalmoplegia;Central core myopathy;Malignant hyperthermia, susceptibility to, 1;King Denborough syndrome|RYR1-related myopathy
β˜…β˜…β˜…β˜†2025
NM_000540.3(RYR1):c.6387C>G (p.Asp2129Glu)Likely pathogenic
not provided|Malignant hyperthermia, susceptibility to, 1
β˜…β˜…β˜…β˜†2025β†’ Residue 2129
NM_000540.3(RYR1):c.7043A>G (p.Glu2348Gly)Likely pathogenic
not provided|Malignant hyperthermia, susceptibility to, 1
β˜…β˜…β˜…β˜†2025β†’ Residue 2348
NM_000540.3(RYR1):c.7076G>A (p.Arg2359Gln)Likely pathogenic
RYR1-related disorder|Malignant hyperthermia, susceptibility to, 1|not provided|Malignant hyperthermia of anesthesia
β˜…β˜…β˜…β˜†2025β†’ Residue 2359
NM_000540.3(RYR1):c.1615T>C (p.Phe539Leu)Likely pathogenic
not provided|RYR1-related disorder|Malignant hyperthermia, susceptibility to, 1
β˜…β˜…β˜…β˜†2025β†’ Residue 539
NM_000540.3(RYR1):c.8026C>T (p.Arg2676Trp)Likely pathogenic
not provided|RYR1-related disorder|Malignant hyperthermia of anesthesia|Malignant hyperthermia, susceptibility to, 1
β˜…β˜…β˜…β˜†2025β†’ Residue 2676
NM_000540.3(RYR1):c.7123G>A (p.Gly2375Arg)Likely pathogenic
not provided|Malignant hyperthermia, susceptibility to, 1
β˜…β˜…β˜…β˜†2025β†’ Residue 2375
NM_000540.3(RYR1):c.1202G>T (p.Arg401Leu)Likely pathogenic
Malignant hyperthermia, susceptibility to, 1|Congenital multicore myopathy with external ophthalmoplegia
β˜…β˜…β˜…β˜†2025β†’ Residue 401
NM_000540.3(RYR1):c.7060G>A (p.Val2354Met)Likely pathogenic
Malignant hyperthermia, susceptibility to, 1|RYR1-related disorder
β˜…β˜…β˜…β˜†2025β†’ Residue 2354
NM_000540.3(RYR1):c.7310C>T (p.Ala2437Val)Likely pathogenic
not provided|Malignant hyperthermia, susceptibility to, 1
β˜…β˜…β˜…β˜†2025β†’ Residue 2437
NM_000540.3(RYR1):c.1654C>T (p.Arg552Trp)Likely pathogenic
not provided|RYR1-related disorder|desflurane response - Toxicity|halothane response - Toxicity|methoxyflurane response - Toxicity|sevoflurane response - Toxicity|succinylcholine response - Toxicity|enflurane response - Toxicity|isoflurane response - Toxicity|Malignant hyperthermia, susceptibility to, 1|Central core myopathy
β˜…β˜…β˜…β˜†2025β†’ Residue 552
NM_000540.3(RYR1):c.7291G>A (p.Asp2431Asn)Likely pathogenic
not provided|Malignant hyperthermia, susceptibility to, 1|RYR1-related disorder|Malignant hyperthermia of anesthesia|Autosomal dominant and autosomal recessive RYR1-related disorders|Congenital multicore myopathy with external ophthalmoplegia;King Denborough syndrome;Central core myopathy;Malignant hyperthermia, susceptibility to, 1
β˜…β˜…β˜…β˜†2025β†’ Residue 2431
View on ClinVar β†—
Drug Targets2
DANTROLENEApproved
Ryanodine receptor 3 antagonist
Fever
DANTROLENE SODIUMApproved
Ryanodine receptor 3 antagonist
Related Genes
DMDProtein interaction100%CALML6Protein interaction100%CALML5Protein interaction100%CALML4Protein interaction100%QDPRProtein interaction99%CACNA2D4Protein interaction96%
Tissue Expression6 tissues
Brain
100%
Lung
18%
Ovary
9%
Heart
5%
Liver
5%
Bone Marrow
3%
Gene Interaction Network
Click a node to explore
RYR1DMDCALML6CALML5CALML4QDPRCACNA2D4
PROTEIN STRUCTURE
Preparing viewer…
PDB6UHS Β· 2.46 Γ… Β· X-ray
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
0.64LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.59 [0.54–0.64]
RankingsWhere RYR1 stands among ~20K protein-coding genes
  • #926of 20,598
    Most Researched345 Β· top 5%
  • #529of 1,025
    FDA-Approved Drug Targets2
  • #58of 5,498
    Most Pathogenic Variants772 Β· top 5%
  • #4,618of 17,882
    Most Constrained (LOEUF)0.64
Genes detectedRYR1
Sources retrieved25 papers
Response timeβ€”
πŸ“„ Sources
25β–Ό
1
Update on RYR1-related myopathies.
PMID: 38994695
Curr Opin Neurol Β· 2024
1.00
2
Genetic epidemiology of malignant hyperthermia in the UK.
PMID: 30236257
Br J Anaesth Β· 2018
0.90
3
Intracellular calcium leak as a therapeutic target for RYR1-related myopathies.
PMID: 32236737
Acta Neuropathol Β· 2020
0.80
4
Mutations in RYR1 in malignant hyperthermia and central core disease.
PMID: 16917943
Hum Mutat Β· 2006
0.70
5
[Congenital myopathies].
PMID: 14593641
Rev Neurol Β· 2003
0.68