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the disease is a genetic disease or disorder

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[00603] In this example, a translatable molecule can be made and used for expressing human ornithine carbamoyltransferase (OTC) in vivo

assay for DNA cleavage or mutation at the target sequence, or assay for altered gene expression activity affected by CRISPR complex formation and/or CRISPR enzyme activity), as compared to a control no exposed to the CRISPR enzyme or complex, or exposed to a CRISPR enzyme lacking the one or more NLSs