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Schematic overview of exosome delivery of siRNA targeting huntingtin mRNA. The plasmid administered by venous injection contains a CMV promoter, neuron-steering RVG and huntingtin siRNA.2 When the plasmid reaches the liver the transcription of huntingtin siRNA and RVG fused to Lamp2b results in the production of RVG-labelled exosomes containing huntingtin siRNA. RVG-tagged exosomes are targeted to the brain where siRNA reduces expression of mHTT mRNA.
