Fig. 2

Ex vivo and in vivo genome editing for clinical therapy. Right: For in ex vivo editing therapy, cells are isolated from a patient to be treated, edited and then re-engrafted back to the patient. To achieve therapeutic success, the target cells must be able to survive in vitro and return to the target tissue after transplantation. Left: For in vivo editing therapy, engineered nucleases are delivered by viral or nonviral approaches and directly injected into the patient for systemic or targeted tissue (such as the eye, brain, or muscle) effect.