Target intelligence / Profile preview

Alpha-actinin-2 (ACTN2)

Target: ACTN2
Molecular classification: Cytoskeletal protein, Actin cross-linking protein, EF-hand domain containing, Spectrin superfamily

Overview

Alpha-actinin-2 is a member of the spectrin superfamily, comprising actin-binding and F-actin cross-linking proteins prominent in the sarcomeric Z-disc of striated muscle, where it scaffolds myofibrillar actin filaments and links other structural proteins (e.g. titin), ensuring mechanical integrity and force transmission[1][2]. This protein also coordinates signaling pathways in muscle and non-muscle cells, interacting with enzymes (e.g. calcineurin), ion channels, and postsynaptic proteins. Pathogenic variants in ACTN2 are associated with inherited cardiac muscle diseases, notably dilated or hypertrophic cardiomyopathy. Alpha-actinin-2 is not commonly a direct target for current drugs; its biomedical relevance is primarily genetic and biomarker-driven rather than pharmacological[1][2].

Other names: Alpha-actinin-2ACTN2Alpha-actinin skeletal muscle isoform 2F-actin cross-linking proteinCMD1AACMH23CMYO8CMYP8MPD6MYOCOZalpha-actinin skeletal muscle

Biological functions

Organization of the contractile apparatus in striated muscleF-actin cross-linkingSarcomeric Z-disc scaffoldingRegulation of signaling complexesModulation of hypertrophic response and excitation–contraction coupling (cardiomyocytes)Stabilization of postsynaptic density (neurons)

Disease associations

Cardiovascular disease (especially dilated and hypertrophic cardiomyopathy due to pathogenic mutations)Muscle disorders (structural myopathies)

Safety considerations

Mutations can lead to cardiac structural and functional defects (e.g. arrhythmias, cardiomyopathy), but direct therapeutic targeting is not practiced; interventions would face challenges due to ubiquitous, essential structural roles in muscle and neuronal cells

Biomarkers

Mutations and protein expression of ACTN2 are used as biomarkers in cardiomyopathy diagnostics and research

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