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In all 3 studies, ALC was also found to be safe and well tolerated which has been shown in other clinical trials for dementia, depression, and pain disorders (Evans et al., 2008

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Editing efficiency was determined on bulk-edited cell populations by targeted PCR of the Cas9-targeted region and secondary PCR using overhang sequences (Table S1), followed by Illumina MiSeq, as previously described (Aubrey et al, 2015)