Exportin-5 (XPO5) is a karyopherin family transporter responsible for exporting pre-miRNA and other structured RNAs from the nucleus to the cytoplasm in a RanGTP-dependent manner, ensuring efficient miRNA maturation by Dicer[1][2][3][4]. It also facilitates export of additional double-stranded RNAs, including tRNA and certain viral RNAs[2][3]. Genetic or functional disruption of XPO5 impairs miRNA processing and can contribute to developmental defects and disease, particularly cancer[1][3].
Other names
Exportin-5XPO5KIAA1291RANBP21Exp5Ran-binding protein 21
02
Mechanism of action
For hypothetical or investigational drugs: inhibition of nuclear export of pre-miRNA, impairment of miRNA processing, modulation of RNA metabolism and gene regulation[1][3]
03
Biological functions
Nuclear export of pre-miRNA (pre-microRNA)[1][2][3][4]Nuclear export of structured double-stranded RNA (e.g., tRNA and viral RNAs)[2][3][4]Contributes to maturation/processing of miRNA and some primary miRNAs[1]
04
Disease associations
Cancer (dysregulation or mutation impairs miRNA maturation and has been implicated in various cancers, such as colorectal cancer)[1][3]Other (essential for embryonic development and tissue morphogenesis)[1]
05
Safety considerations
Essential for development: genetic knockout/complete inhibition is embryonic lethal in mice[1]Suppression or mutation may globally impair miRNA biogenesis, risking widespread deregulation of gene expression—possibly leading to toxicity, developmental defects, or adverse cancer phenotypes[1][3]
06
Interacting drugs
No approved drugs currently directly target Exportin-5 in clinical use; research into modulators or miRNA-based therapeutics involving this pathway is ongoing[2]
07
Biomarkers
Levels of pre-miRNA, mature miRNA, XPO5 expression (may be used as research biomarkers for miRNA pathway activity and select cancers)[1][3]
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