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Genome editing for gene deletion or gene repair. A nuclease (often CAS9 with a guide RNA as part of the CRISPR system) generates double-strand DNA breaks. If a donor template is not provided, NHEJ will generate mutations that typically lead to a loss-of-function mutation. This strategy is used (for example) to remove repressors of fetal hemoglobin. The other approach requires a donor sequence for repair and relies on HDR. The donor template is often provided by an AAV.
