Alternative Export Pathway for Vector Expressed RNAi
The addition of a short additional sequence targets the RNAi molecules to an alternative nuclear export pathway.
UIRF Case #:07042
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Level of Development
General: Experimental Proof of Concept
Researchers at the University of Iowa have discovered that the general nucleus-to-cytoplasm transport system used by RNAi molecules to enter the cytoplasm, Exportin-V, can be saturated. When this occurs, the therapeutic RNAi molecules are caught in the nucleus and prevented from reaching their target and exerting their silencing effect. In addition, other cellular molecules are also prevented from entering the cytoplasm, which may have additional detrimental effects to the cell. Experiments have shown that the addition of a short hairpin-forming nucleotide sequence targets the RNAi molecule to the cytoplasm via the nxf export pathway, which is less likely to be blocked by saturation. This addition has been utilized on several RNAi molecules and its implementation has be demonstrated to reverse the saturation of Exportin-V and return efficient cytoplasmic transport to the RNAi molecules.
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