Target intelligence / Profile preview

KH-type splicing regulatory protein (KHSRP)

Target
KHSRP
Molecular classification
RNA-binding protein, Splicing factor, Gene expression regulator, Single-stranded nucleic acid binding protein
01

Overview

KH-type splicing regulatory protein (KHSRP) is a multifunctional RNA-binding protein that modulates gene expression at the levels of pre-mRNA splicing, mRNA decay, and microRNA maturation. It preferentially binds single-stranded nucleic acids via four KH-domains, promoting rapid degradation of labile transcripts containing AU-rich elements and facilitating the maturation of select microRNAs. As a regulator of critical cellular processes—including cell proliferation, differentiation, immune response, and metabolism—KHSRP is implicated in diverse diseases such as cancer, neuromuscular disorders, inflammation, obesity, and diabetes. KHSRP's complex regulatory roles make it a significant potential therapeutic target, although direct drugs have yet to be established[1][2][3][4][5].

Other names
KHSRPKSRPFBP2FUBP2Far upstream element-binding protein 2p75
02

Mechanism of action

Not applicable (no direct drugs known; hypothetical strategies include modulation of RNA-binding, inhibition of mRNA decay function, or alteration of miRNA maturation[1])

03

Biological functions

pre-mRNA splicingmRNA decaymicroRNA biogenesismRNA traffickingmRNA localizationRegulation of gene expressionCell proliferationCell differentiationImmune response modulation
04

Disease associations

CancerNeuromuscular disordersObesityType II diabetesInflammationRenal cell carcinoma (with MIT translocations)Pheochromocytoma
05

Safety considerations

Therapeutic challenges would likely include systemic effects due to KHSRP's broad roles in RNA metabolism and gene regulation, risking off-target effects in multiple tissues
06

Biomarkers

KHSRP expression levels may act as biomarkers for certain tumors or immune conditions (e.g., cancer tissue profiling, muscle disease diagnostics)

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