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Target intelligence / Profile preview
Calsequestrin-1 (CASQ1)
- Target
- CASQ1
- Molecular classification
- Other (Calcium-binding protein), Calcium sensor
01
Overview
Calsequestrin-1 (CASQ1) is a high-capacity, moderate-affinity calcium-binding protein that serves as the major calcium buffer in the sarcoplasmic reticulum (SR) of fast-twitch skeletal muscle fibers. It helps maintain SR calcium concentration, regulates calcium release essential for muscle contraction via interaction with ryanodine receptor 1 (RyR1), and functions as a dynamic calcium sensor, modulating excitation–contraction coupling. CASQ1 plays important structural roles in organizing SR terminal cisternae and influences store-operated calcium entry (SOCE) indirectly. Pathogenic mutations or loss can result in a spectrum of skeletal muscle diseases, and while not currently a direct drug target, it is of significant biomedical interest for understanding muscle physiology and related pathologies.
- Other names
- Calsequestrin-1CASQ1CASQCSQ1CalmitinePDIB1VMCQAcalmitincalsequestrinskeletal muscle isoformcalsequestrin 1fast-twitchskeletal musclecalmitine
02
Mechanism of action
CASQ1 is not a direct pharmacological target for drugs; mechanisms relate to physiological calcium buffering and modulation of RyR1 activity
03
Biological functions
Calcium ion bindingCalcium buffering in the sarcoplasmic reticulumRegulation of calcium release via ryanodine receptor type 1 (RyR1)Regulation of excitation–contraction coupling in skeletal muscleNegative regulation of store-operated calcium entry (SOCE)
04
Disease associations
Skeletal muscle disease (e.g., vacuolar myopathy with CASQ1 aggregates, tubular aggregate myopathy)MyopathyPotentially involved in muscular dystrophies (absent in Duchenne and Becker muscular dystrophy patients)Other
05
Safety considerations
Mutations or dysfunction in CASQ1 are linked to skeletal muscle diseases, including vacuolar myopathy and tubular aggregate myopathyLoss of function or aggregation can lead to muscle weakness, altered excitability, or contractile defects
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