Illuminating the genome-wide activity of genome editors for safe and effective therapeutics.

Abstract:

:Genome editing holds remarkable promise to transform human medicine as new therapies that can directly address the genetic causes of disease. However, concerns remain about possible undesired biological consequences of genome editors, particularly the introduction of unintended 'off-target' mutations. Here, we discuss both important considerations for therapeutic genome editing and our understanding of the functional impact of undesired off-target mutations. An important challenge for the future will be the development of new approaches for predicting and defining the probable function of unintended genome-editing mutations, which will inspire confidence in the next generation of promising genome-editing therapies.

journal_name

Genome Biol

journal_title

Genome biology

authors

Cheng Y,Tsai SQ

doi

10.1186/s13059-018-1610-2

subject

Has Abstract

pub_date

2018-12-22 00:00:00

pages

226

issue

1

eissn

1474-7596

issn

1474-760X

pii

10.1186/s13059-018-1610-2

journal_volume

19

pub_type

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