Brief intro:
- Author: Giuseppe Militello, Alyssa Greig, Chongfeng Bi, Ana Vasileva, Maria I. Zavodszky, Shih-Ching Lo, Edward Guilmette, Pete Clarner, Bin Liu, Guruharsha Bhat, Junghae Suh, Lukas Dow, Johannes Zuber, Christof Fellmann & Prem K. Premsrirut
- Journal: Scientific Reports
- Doi: https://www.doi.org/10.1038/s41598-025-07061-y
- Publication Date: 2025/7/1
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 novel amiRNAs with potential therapeutic application as well as a pipeline to generate and validate novel amiRNAs from endogenous pri-miRNAs.
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PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
