RNA editing with CRISPR-Cas13

American Association for the Advancement of Science (AAAS) - Tập 358 Số 6366 - Trang 1019-1027 - 2017
David Cox1,2,3,4,5,6, Jonathan S. Gootenberg1,2,4,7,6, Omar O. Abudayyeh1,2,4,5,6, Brian Franklin1,2,4,6, Max J. Kellner1,2,4,6, Julia Joung1,2,4,6, Feng Zhang1,2,4,6
1Broad Institute of Massachusetts Institute of Technology (MIT) and Harvard, Cambridge, MA 02142, USA
2Department of Biological Engineering, MIT, Cambridge, MA 02139, USA
3Department of Biology, MIT, Cambridge, MA 02139, USA
4Department of Brain and Cognitive Science, MIT, Cambridge, MA 02139, USA
5Harvard-MIT Division of Health Sciences and Technology, MIT, Cambridge, MA 02139, USA
6McGovern Institute for Brain Research, MIT, Cambridge, MA 02139, USA
7Department of Systems Biology, Harvard Medical School, Boston, MA 02115, USA

Tóm tắt

Precise transcriptome engineering Efficient and precise RNA editing to correct disease-relevant transcripts holds great promise for treating genetic disease. Cox et al. took advantage of the ability of Cas13b, an effector from a type VI CRISPR-Cas system, to target specific RNAs directly (see the Perspective by Yang and Chen). They fused Cas13b with the ADAR2 adenosine deaminase domain and used rational protein engineering to improve the resultant enzyme. These approaches yielded an RNA knockdown and editing platform that allowed efficient and specific RNA depletion and correction in mammalian cells. Science , this issue p. 1019 ; see also p. 996

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American Association for the Advancement of Science (AAAS)

Tập 358 Số 6366

1019-1027

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