Integrative Analysis of CRISPR/Cas9 Target Sites in the Human HBB Gene.

ABSTRACT: Recently, the clustered regularly interspaced short palindromic repeats (CRISPR) system has emerged as a powerful customizable artificial nuclease to facilitate precise genetic correction for tissue regeneration and isogenic disease modeling. However, previous studies reported substantial off-target activities of CRISPR system in human cells, and the enormous putative off-target sites are labor-intensive to be validated experimentally, thus motivating bioinformatics methods for rational design of CRISPR system and prediction of its potential off-target effects. Here, we describe an integrative analytical process to identify specific CRISPR target sites in the human β-globin gene (HBB) and predict their off-target effects. Our method includes off-target analysis in both coding and noncoding

SUBMITTER: Luo Y 

PROVIDER: S-EPMC4396065 | biostudies-literature | 2015

REPOSITORIES: biostudies-literature