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• W2765351619 endingPage "1027" @default.
• W2765351619 startingPage "1019" @default.
• W2765351619 abstract "Nucleic acid editing holds promise for treating genetic disease, particularly at the RNA level, where disease-relevant sequences can be rescued to yield functional protein products. Type VI CRISPR-Cas systems contain the programmable single-effector RNA-guided ribonuclease Cas13. We profiled type VI systems in order to engineer a Cas13 ortholog capable of robust knockdown and demonstrated RNA editing by using catalytically inactive Cas13 (dCas13) to direct adenosine-to-inosine deaminase activity by ADAR2 (adenosine deaminase acting on RNA type 2) to transcripts in mammalian cells. This system, referred to as RNA Editing for Programmable A to I Replacement (REPAIR), which has no strict sequence constraints, can be used to edit full-length transcripts containing pathogenic mutations. We further engineered this system to create a high-specificity variant and minimized the system to facilitate viral delivery. REPAIR presents a promising RNA-editing platform with broad applicability for research, therapeutics, and biotechnology." @default.
• W2765351619 created "2017-11-10" @default.
• W2765351619 creator A5011678843 @default.
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• W2765351619 date "2017-11-24" @default.
• W2765351619 modified "2023-10-11" @default.
• W2765351619 title "RNA editing with CRISPR-Cas13" @default.
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• W2765351619 doi "https://doi.org/10.1126/science.aaq0180" @default.
• W2765351619 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/5793859" @default.
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