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Effective and Accurate Gene Silencing by a Recombinant AAV-Compatible MicroRNA Scaffold.


ABSTRACT: Short hairpin RNAs that are delivered by recombinant adeno-associated virus (rAAV) have the potential to elicit long-term RNAi therapy for human disease. However, the discovery that short hairpin sequences can cause truncation of the rAAV genome calls into question the efficiency and gene-silencing specificity of this strategy in humans. Here, we report that embedding the guide strand of a small silencing RNA into an artificial microRNA (miRNA) scaffold derived from mouse miRNA-33 ensures rAAV genomic integrity and reduces off-targeting by 10-fold, while maintaining effective in vivo target gene repression in mice.

SUBMITTER: Xie J 

PROVIDER: S-EPMC7001000 | biostudies-literature |

REPOSITORIES: biostudies-literature

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