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- W3136169552 abstract "Abstract CRISPR-mediated gene activation (CRISPRa) is a promising therapeutic gene editing strategy without inducing DNA double-strand breaks (DSBs). However, in vivo implementation of these CRISPRa systems remains a challenge. Here, we report a compact and robust miniCas9 activator (termed miniCAFE) for in vivo activation of endogenous target genes. The system relies on recruitment of an engineered minimal nuclease-null Cas9 from Campylobacter jejuni and potent transcriptional activators to a target locus by a single guide RNA. It enables robust gene activation in human cells even with a single DNA copy and is able to promote lifespan of Caenorhabditis elegans through activation of longevity-regulating genes. As proof-of-concept, delivered within an all-in-one adeno-associated virus (AAV), miniCAFE can activate Fgf21 expression in the liver and regulate energy metabolism in adult mice. Thus, miniCAFE holds great therapeutic potential against human diseases." @default.
- W3136169552 created "2021-03-29" @default.
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- W3136169552 date "2021-03-22" @default.
- W3136169552 modified "2023-10-15" @default.
- W3136169552 title "MiniCAFE, a CRISPR/Cas9-based compact and potent transcriptional activator, elicits gene expression <i>in vivo</i>" @default.
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