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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 15 days ago
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MYBPC1
myosin binding protein C1
Chromosome 12 Β· 12q23.2
NCBI Gene: 4604Ensembl: ENSG00000196091.16HGNC: HGNC:7549UniProt: A8KAB1
56PubMed Papers
23Diseases
0Drugs
14Pathogenic Variants
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
protein bindingmyosin bindingtitin bindingM bandlethal congenital contracture syndrome 4Disorder of bilirubin metabolism and excretiondigitotalar dysmorphismLethal congenital contracture syndrome type 3
✦AI Summary

MYBPC1 encodes myosin-binding protein C, slow type (sMyBP-C), a sarcomeric thick filament-associated protein localized at the C-zone of the A-band 1. The protein binds to both myosin and actin filaments, modulating actomyosin crossbridge cycling and regulating skeletal muscle contractility 1. sMyBP-C undergoes extensive exon shuffling to generate multiple variants co-expressed in slow and fast skeletal muscles, with phosphorylation sites modified by PKA and PKC 1. Beyond its regulatory role, MYBPC1 appears to serve a primarily structural function, as sarcomeric disorganization accompanies contractile dysfunction in affected muscles 2. Pathogenic missense and nonsense mutations in MYBPC1 cause Myotrem (congenital myopathy 16), an early-onset disorder characterized by muscle weakness, hypotonia, skeletal deformities, and myogenic tremor 123. Clinical features overlap with spinal muscular atrophy, including hypotonia and involuntary tongue/limb movements, complicating differential diagnosis 4. MYBPC1 mutations are also associated with arthrogryposis distal 1B and lethal congenital contracture syndrome 4. Phenotypic manifestation shows muscle-specific, age-dependent, and sex-dependent progression, with soleus muscle consistently affected across sexes while tibialis anterior shows male-specific decline at 24 months 2. Interestingly, MYBPC1 is downregulated in laryngeal carcinoma, functioning as a tumor suppressor 5.

Sources cited
1
MYBPC1 localization, function in regulating actomyosin crossbridges, exon shuffling generating variants, phosphorylation sites, and association with distal arthrogryposis and muscle tremors
PMID: 27683561
2
MYBPC1 E248K mutation causes Myotrem with muscle weakness and tremor; sMyBP-C serves primarily structural role; muscle-specific, sex-dependent, and age-dependent progression of phenotype
PMID: 40569690
3
MYBPC1 variants cause congenital tremor and myopathy (MYOTREM) with postural and intention tremor; muscle MRI findings show atrophy and fatty infiltration
PMID: 38185014
4
MYBPC1 mutations present in early childhood with hypotonia, involuntary tongue/limb movements, and normal nerve conduction, overlapping SMA features
PMID: 37392669
5
MYBPC1 is downregulated in laryngeal carcinoma and functions as a tumor suppressor through miR-451a/ATF2 signaling
PMID: 36539363
Disease Associationsβ“˜23
lethal congenital contracture syndrome 4Open Targets
0.71Strong
Disorder of bilirubin metabolism and excretionOpen Targets
0.69Moderate
digitotalar dysmorphismOpen Targets
0.69Moderate
Lethal congenital contracture syndrome type 3Open Targets
0.63Moderate
myopathy, congenital, with tremorOpen Targets
0.62Moderate
distal arthrogryposisOpen Targets
0.45Moderate
genetic disorderOpen Targets
0.42Moderate
MYBPC1-related autosomal recessive non-lethal arthrogryposis multiplex congenita syndromeOpen Targets
0.40Weak
cervical carcinomaOpen Targets
0.39Weak
lethal congenital contracture syndromeOpen Targets
0.37Weak
liver diseaseOpen Targets
0.30Weak
placental retentionOpen Targets
0.29Weak
Abnormality of the musculatureOpen Targets
0.27Weak
obesityOpen Targets
0.22Weak
cardiomyopathyOpen Targets
0.22Weak
Abnormality of the skeletal systemOpen Targets
0.21Weak
biliary tract diseaseOpen Targets
0.21Weak
musculoskeletal system diseaseOpen Targets
0.17Weak
preeclampsiaOpen Targets
0.15Weak
Dental malocclusionOpen Targets
0.13Weak
Arthrogryposis, distal, 1BUniProt
Congenital myopathy 16UniProt
Lethal congenital contracture syndrome 4UniProt
Pathogenic Variants14
NM_002465.4(MYBPC1):c.788T>G (p.Leu263Arg)Pathogenic
MYBPC1-related disorder|Myopathy, congenital, with tremor|not provided|Inborn genetic diseases|Arthrogryposis, distal, type 1B;Myopathy, congenital, with tremor
β˜…β˜…β˜†β˜†2025β†’ Residue 263
NM_002465.4(MYBPC1):c.793C>G (p.Arg265Gly)Likely pathogenic
Arthrogryposis, distal, type 1B|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 265
NM_002465.4(MYBPC1):c.776T>C (p.Leu259Pro)Pathogenic
MYBPC1-related disorder|not provided|Myopathy, congenital, with tremor
β˜…β˜…β˜†β˜†2024β†’ Residue 259
NM_002465.4(MYBPC1):c.742G>A (p.Glu248Lys)Pathogenic
not provided|Myopathy, congenital, with tremor
β˜…β˜…β˜†β˜†2023β†’ Residue 248
NM_002465.4(MYBPC1):c.795_803dup (p.Arg268_Met269insLeuLysArg)Likely pathogenic
Myopathy, congenital, with tremor
β˜…β˜†β˜†β˜†2025β†’ Residue 268
NM_002465.4(MYBPC1):c.788T>C (p.Leu263Pro)Pathogenic
not provided
β˜…β˜†β˜†β˜†2025β†’ Residue 263
NM_002465.4(MYBPC1):c.832+1G>CLikely pathogenic
not provided
β˜…β˜†β˜†β˜†2022
NM_002465.4(MYBPC1):c.122del (p.Pro41fs)Likely pathogenic
Abnormality of the musculature
β˜…β˜†β˜†β˜†2021β†’ Residue 41
NM_002465.4(MYBPC1):c.1242C>A (p.Tyr414Ter)Likely pathogenic
not provided
β˜…β˜†β˜†β˜†2019β†’ Residue 414
NM_002465.4(MYBPC1):c.1678G>C (p.Val560Leu)Likely pathogenic
Distal arthrogryposis
β˜…β˜†β˜†β˜†2017β†’ Residue 560
NM_002465.4(MYBPC1):c.1253T>G (p.Val418Gly)Likely pathogenic
not provided
β˜…β˜†β˜†β˜†2016β†’ Residue 418
NM_002465.4(MYBPC1):c.739T>C (p.Tyr247His)Pathogenic
Myopathy, congenital, with tremor
β˜†β˜†β˜†β˜†2024β†’ Residue 247
NM_002465.4(MYBPC1):c.2566T>C (p.Tyr856His)Pathogenic
Arthrogryposis, distal, type 1B
β˜†β˜†β˜†β˜†2010β†’ Residue 856
NM_002465.4(MYBPC1):c.706T>C (p.Trp236Arg)Pathogenic
Arthrogryposis, distal, type 1B
β˜†β˜†β˜†β˜†2010β†’ Residue 236
View on ClinVar β†—
Related Genes
MYOM3Shared pathway100%MYBPC2Shared pathway100%MYL3Protein interaction100%MYH6Protein interaction99%MYL1Protein interaction97%TTNProtein interaction94%
Tissue Expression6 tissues
Brain
100%
Heart
8%
Lung
5%
Liver
4%
Ovary
2%
Bone Marrow
0%
Gene Interaction Network
Click a node to explore
MYBPC1MYOM3MYBPC2MYL3MYH6MYL1TTN
PROTEIN STRUCTURE
Preparing viewer…
PDB2YXM Β· 1.51 Γ… Β· X-ray
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
0.43Moderately Constrained
pLIβ“˜
1.00Intolerant
Observed/Expected LoF0.34 [0.27–0.43]
RankingsWhere MYBPC1 stands among ~20K protein-coding genes
  • #8,110of 20,598
    Most Researched56
  • #2,528of 5,498
    Most Pathogenic Variants14
  • #2,338of 17,882
    Most Constrained (LOEUF)0.43 Β· top quartile
Genes detectedMYBPC1
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
MYBPC1, an Emerging Myopathic Gene: What We Know and What We Need to Learn.
PMID: 27683561
Front Physiol Β· 2016
0.90
3
Gene expression profiling reveals upregulated FUT1 and MYBPC1 in children with pancreaticobiliary maljunction.
PMID: 31365696
Braz J Med Biol Res Β· 2019
0.80
4
Peripherally-induced Movement Disorders: An Update.
PMID: 37008994
Tremor Other Hyperkinet Mov (N Y) Β· 2023
0.70
5
Sex-dependent and muscle-specific progression of the MYBPC1 E248K Myotrem myopathy in response to aging.
PMID: 40569690
JCI Insight Β· 2025
0.60