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- W2793359586 abstract "Class 2 CRISPR-Cas systems endow microbes with diverse mechanisms for adaptive immunity. Here, we analyzed prokaryotic genome and metagenome sequences to identify an uncharacterized family of RNA-guided, RNA-targeting CRISPR systems that we classify as type VI-D. Biochemical characterization and protein engineering of seven distinct orthologs generated a ribonuclease effector derived from Ruminococcus flavefaciens XPD3002 (CasRx) with robust activity in human cells. CasRx-mediated knockdown exhibits high efficiency and specificity relative to RNA interference across diverse endogenous transcripts. As one of the most compact single-effector Cas enzymes, CasRx can also be flexibly packaged into adeno-associated virus. We target virally encoded, catalytically inactive CasRx to cis elements of pre-mRNA to manipulate alternative splicing, alleviating dysregulated tau isoform ratios in a neuronal model of frontotemporal dementia. Our results present CasRx as a programmable RNA-binding module for efficient targeting of cellular RNA, enabling a general platform for transcriptome engineering and future therapeutic development." @default.
- W2793359586 created "2018-03-29" @default.
- W2793359586 creator A5010848619 @default.
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- W2793359586 date "2018-04-01" @default.
- W2793359586 modified "2023-10-14" @default.
- W2793359586 title "Transcriptome Engineering with RNA-Targeting Type VI-D CRISPR Effectors" @default.
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- W2793359586 doi "https://doi.org/10.1016/j.cell.2018.02.033" @default.
- W2793359586 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/5910255" @default.
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- W2793359586 hasPublicationYear "2018" @default.
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