In a new study published in the Cell Research, Chen-Yu Zhang’s group at Nanjing University reports “In vivo self-assembled small RNA is the new generation of RNAi therapeutics.”
The development of RNAi therapy has undergone two major stages, direct injection of synthetic siRNAs and delivery with artificial vehicles; neither have realized the full therapeutic potential of RNAi in clinic. In this study, Chen-Yu Zhang’s group reprogram host liver with genetic circuits to direct the synthesis and self-assembly of siRNAs into secretory exosomes. In vivo assembled siRNAs are systematically distributed to multiple tissues or targeted to specific tissues (e.g., brain), inducing potent target gene silencing in these tissues. The therapeutic value of this strategy is demonstrated in a variety of diseases ranging from cancers to metabolic diseases. Overall, in vivo self-assembled siRNA represents a next generation RNAi therapeutics, which makes RNAi therapy feasible.
The scientific significance of these findings is highlighted below:
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