Gene therapy: CRISPR therapies — making the grade not the cut

Nature Reviews Drug Discovery 17, 95 (2018). doi:10.1038/nrd.2018.3 Author: Darren J. Burgess CRISPR-based systems for therapeutic purposes have shown promise in numerous mouse models of human diseases, but one concern that is limiting roll-out to human trials is that during genome editing, unintended Cas9-induced DNA double-strand breaks (DSBs) could cause off-target mutations, toxicity and harm to patients.
Source: Nature Reviews Drug Discovery - Category: Drugs & Pharmacology Authors: Tags: Research Highlight Source Type: research