Figure 1. Genome editors can be used therapeutically in several ways, and both ex vivo and in vivo delivery for somatic genome editing have advanced to clinical trial.

Ex vivo: cells can be extracted from the patient or donor modified in the laboratory and then infused into the patient. In vivo: delivery vehicles, including viral vectors and nanoparticles can be loaded with the genome editor and then injected into the patient either systemically, which results in liver editing primarily, or into the location of interest, for example, the eye.