Target intelligence / Profile preview

Alpha-actinin-4 mRNA (ACTN4 mRNA) (ACTN4 mRNA)

Target
ACTN4 mRNA
Molecular classification
Actin-binding protein, Cytoskeletal protein, Transcription co-activator
01

Overview

Alpha-actinin-4 (ACTN4) is a member of the spectrin gene superfamily, primarily known for its role as an actin-binding protein that cross-links F-actin into bundles (UniProt P35609). Beyond its cytoskeletal functions, ACTN4 can translocate to the nucleus where it acts as a transcriptional co-activator for various signaling pathways, including the Wnt/beta-catenin and NF-kappaB pathways (PMID: 25983578). In the context of oncology, ACTN4 mRNA is frequently overexpressed, correlating with increased cell motility, invasion, and poor prognosis in cancers such as pancreatic, breast, and colorectal carcinoma (PMID: 25983578, PMID: 26261558). Additionally, mutations in the ACTN4 gene are a known cause of familial focal segmental glomerulosclerosis (FSGS), a condition characterized by progressive kidney scarring (PMID: 10746727). Therapeutic strategies targeting ACTN4 mRNA, such as small interfering RNAs (siRNAs) or antisense oligonucleotides (ASOs), aim to downregulate protein expression to inhibit metastatic progression or manage podocyte dysfunction (PMID: 25983578). While currently primarily in the preclinical stage, ACTN4 mRNA represents a promising target for precision medicine in both oncology and nephrology.

Other names
ACTN4Alpha-actinin-4FSN1Non-muscle alpha-actinin 4
02

Mechanism of action

RNA interference (siRNA) or antisense oligonucleotide (ASO) mediated degradation of mRNA to reduce protein expression.

03

Biological functions

Actin filament bundlingCell migrationTranscription regulationCell adhesion
04

Disease associations

CancerFocal segmental glomerulosclerosisMetastasis
05

Safety considerations

Potential for off-target effectsImpact on normal cytoskeletal function in healthy cellsRenal toxicity if over-inhibited
06

Interacting drugs

Experimental siRNAs

1 more in the full profile.

07

Biomarkers

ACTN4 mRNA expression levelsACTN4 protein levelsNuclear ACTN4 localization

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