Engineered microRNA scaffolds for potent gene silencing in vivo.

ABSTRACT: RNA interference (RNAi) is emerging as a powerful strategy for therapeutic targeting of "undruggable" targets. However, efficacy of currently used siRNA-based therapies is often hindered by transient effects and limited modeling possibilities. Artificial microRNAs (amiRNAs or miRNA scaffolds) present a durable and precise approach to gene silencing, opening new avenues for developing long lasting targeted therapies. In this study, we engineered highly expressed primary miRNAs (pri-miRNAs) with sequence determinants known to enhance processing efficacy and precision. The resulting amiRNAs were extensively tested both in vitro and in vivo and proved to efficiently silence a target gene when virally delivered via adeno-associated virus (AAV) into mice brains. This study provides a set of nove

SUBMITTER: Militello G 

PROVIDER: S-EPMC12218229 | biostudies-literature | 2025 Jul

REPOSITORIES: biostudies-literature