Self-assembled primary microRNA mimics for simultaneous multimodal gene regulation
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Here we report self-assembled primary microRNA mimics for achieving simultaneous multimodal gene regulation. Primary microRNA mimics were designed as substrate RNAs to recruit and interact with Drosha/DGCR8 microprocessor. Incorporation of functional RNA motifs and site-specific chemical modification on the primary microRNA mimics have supported the biogenesis of two individual gene regulating oligonucleotides. Once they were cleaved by the endogenous microprocessor, basal strands and pre-miRNA would be generated inside of cells. Primary microRNA mimics with the basal single strands modified with Antisense oligonucleotides(ASOs) targeting survivor motor nuron2(SMN2-ASO) or miR21(anti-miR21) showed an improved in vitro activity and resulted simultaneous multimodal gene regulation in cells. Combinatorial use of different class of oligonucleotide therapeutics using artificial pri-miRNA may show synergies in therapeutic effect and broad range of applications for gene therapy.