ISSN 1662-4009 (online)

ESPE Yearbook of Paediatric Endocrinology (2018) 15 14.11 | DOI: 10.1530/ey.15.14.11


To read the full abstract: Science 2017;358:1019-1027

The gene-editing system CRISPR-Cas9 continues to surge through molecular biology labs and article titles. This article provides a new lab shelf, manipulating RNA, not DNA. This RNA editor could be used to treat conditions that are short term in nature, such as local inflammation. The RNA tool focuses on single-letter mutations in the DNA, A to I mutations that account for more than 40% of all the mutations. The authors named the new system REPAIR: RNA Editing for Programmable A to I Replacement. REPAIR successfully converted A to I at target sites at an average rate of 20-40%. With DNA editing, once you make a permanent change it is hard to undo it. Whereas with RNA, once you stop the RNA editing REPAIR system, then those changes will revert as new RNA is produced.

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