The Off-Targets of Clustered Regularly Interspaced Short Palindromic Repeats Gene Editing
Manuel M. Vicente, Miguel Chaves‐Ferreira, João M. P. Jorge, João T. Proença, Vasco M. Barreto
Abstract
The repurposing of the CRISPR/Cas bacterial defense system against bacteriophages as simple and flexible molecular tools has revolutionized the field of gene editing. These tools are now widely used in basic research and clinical trials involving human somatic cells. However, a global moratorium on all clinical uses of human germline editing has been proposed because the technology still lacks the required efficacy and safety. Here we focus on the approaches developed since 2013 to decrease the frequency of unwanted mutations (the off-targets) during CRISPR-based gene editing.
Topics & Concepts
CRISPRGenome editingPalindromeRepurposingComputational biologyBiologyGeneSomatic cellGermlineGeneticsComputer scienceEcologyCRISPR and Genetic EngineeringInnovation and Socioeconomic DevelopmentVirus-based gene therapy research