1. LATE–a novel sensitive cell-based assay for the study of CRISPR/Cas9-related long-term adverse treatment effects
- Author
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Dawid Głów, Simon Meyer, Irene García Roldán, Lara Marie Akingunsade, Kristoffer Riecken, and Boris Fehse
- Subjects
CRISPR/Cas ,genome editing ,off-targets ,side effects ,gene therapy ,cellular assay ,Genetics ,QH426-470 ,Cytology ,QH573-671 - 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 (
- Published
- 2021
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