Target intelligence / Profile preview

FUS RNA binding protein (FUS)

Target: FUS
Molecular classification: RNA binding protein, heterogeneous nuclear ribonucleoprotein (hnRNP) component, DNA binding protein, transcription factor interactor

Overview

FUS RNA binding protein (FUS) is a multifunctional DNA/RNA binding protein critical for RNA metabolism, including transcription regulation, pre-mRNA splicing, mRNA export, and DNA damage repair. It is a component of the hnRNP complex and interacts with various transcriptional machinery and splicing factors. FUS is highly expressed in neurons, influencing synaptic function and neuronal survival. Pathological aggregation or mislocalization of FUS is a hallmark of ALS and FTLD. FUS was also identified as a fusion oncoprotein in myxoid liposarcoma, highlighting its relevance in cancer biology. Its broad molecular interactions make it both a potential therapeutic target and a key biomarker in neurodegenerative and neoplastic conditions.

Other names: TLSFUS/TLSFused in sarcomaTranslocated in liposarcoma

Mechanism of action

Experimental gene therapies aim to correct FUS mislocalization in ALS/FTLD Approaches include inhibition of FUS aggregation, restoration of nuclear localization, and modulation of RNA binding or splicing function

Biological functions

Transcriptional regulation (via interaction with RNA polymerase II and transcription factors)Pre-mRNA splicing (component of the spliceosome; regulates alternative splicing)mRNA transport (exports mRNA from nucleus to cytoplasm)DNA damage repair (rapidly recruited to DNA breaks, involved in homologous recombination)miRNA processingFormation of stress granules (in response to cellular stress)

Disease associations

Neurodegenerative disease (Amyotrophic lateral sclerosis (ALS), frontotemporal lobar degeneration (FTLD))Cancer (fusion oncoprotein in myxoid liposarcoma, regulates gene expression and chromatin structure)Other (immune system defects, depending on genetic background)

Safety considerations

Major concerns are off-target effects in gene therapies, risk of impairing vital RNA/DNA repair mechanisms, and possible broader effects due to FUS’s pleiotropic functionsLoss-of-function can cause neuronal death or immune defects; gain-of-function may induce cytoplasmic aggregates.

Biomarkers

FUS mislocalization or aggregation (cytoplasmic FUS)nuclear FUS levels in neuronspresence of FUS-positive inclusions in ALS/FTLD brain tissue

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