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Article

RNA editing with CRISPR-Cas13

25 Oct 2017

Abstract excerpt

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...

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PMID: 29070703
DOI: 10.1126/science.aaq0180

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