Precise transcriptome engineering

Abstract

Biotechnology 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][1]; see also p. [996][2] [1]: /lookup/doi/10.1126/science.aaq0180 [2]: /lookup/doi/10.1126/science.aar2400