A crisper look at genome editing: RNA-guided genome modification.

摘要

There is an exciting new player in the ever-expanding field of genome editing. In a study reported in the January 2013 issue of Science, two groups—Cong et al.1 and Mali et al2—explored the limits and adaptability of a prokaryotic RNA-based system for mammalian genome-wide editing. This new method of genome engineering is derived from an adaptive immune system known as CRISPR (Clustered Regulatory Interspaced Short Palindromic Repeats) that bacteria and ar-chaea use as a means to protect themselves against foreign invasive elements. These two studies show that the CRISPR system is an efficient method to alter mammalian genomes. At present, four types of discrete systems have been shown to generate, to different degrees of specificity and efficiency, genome-wide editing: three distinct protein-based nuclease systems,3"5 a chemical-based nuclease system,6 an adeno-associated virus (AAV)-based system,7 and now a protein RNA-based system.1-2