LATE-a novel sensitive cell-based assay for the study of CRISPR/Cas9-related long-term adverse treatment effects.

ABSTRACT: Since the introduction of clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9), genome editing has been broadly applied in basic research and applied biotechnology, whereas translation into clinical testing has raised safety concerns. Indeed, although frequencies and locations of off-target events have been widely addressed, little is known about their potential biological consequences in large-scale long-term settings. We have developed a long-term adverse treatment effect (LATE) in vitro assay that addresses potential toxicity of designer nucleases by assessing cell transformation events. In small-scale proof-of-principle experiments we reproducibly detected low-frequency (<0.5%) growth-promoting events in primary human newborn fore

SUBMITTER: Glow D 

PROVIDER: S-EPMC8399046 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature